Archive for the ‘Gene Therapy Research’ Category

Oncogenuity, Inc., a Fortress partner company, enters into an agreement with Columbia University to develop a broad platform technology using oligonucleotides

Initial target is KRAS-driven cancers, often considered un-druggable

Platform being explored as a treatment for coronaviruses, including COVID-19

NEW YORK, May 08, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (Fortress), an innovative biopharmaceutical company, today announced that Oncogenuity, Inc. (Oncogenuity), a new Fortress partner company, has entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers. The proprietary platform produces oligomers, now known as ONCOlogues, that are capable of binding gene sequences 1,000 times more effectively than complementary native DNA. The technology comes from the labs of Gary Schwartz, M.D., Division Chief, Hematology/Oncology, and Jeffrey Rothman, M.D., Ph.D., Assistant Professor of Medicine.

ONCOlogues are sensitive to a single base pair mismatch, resistant to degradation and use a proprietary delivery sequence to enter cells. ONCOlogues selectivity enables Oncogenuity to target genetically driven cancers caused by mutations without impacting wild-type (WT) DNA sequences, potentially limiting off-target toxicity. In addition, this allows ONCOlogues to target mutations that have historically been considered un-druggable.

Oncogenuity has established proof-of-concept in a pre-clinical setting for various cancer types. The companys most advanced program is targeting the KRAS mutation G12D, which was previously considered un-druggable and plays a significant role in various cancer types with substantial unmet need, including pancreatic and colorectal. Given the platforms ability to target any mutation, Oncogenuity will continue to evaluate other mutations simultaneously. The company anticipates additional data publications in the coming 12 months.

Additionally, Oncogenuity is exploring the platforms potential to treat coronaviruses. Coronaviruses have single-stranded RNA genomes, making them strong targets for ONCOlogues. The company is studying replacement sequences, which could help combat COVID-19 and provide proof-of-concept as a treatment for coronaviruses. These ongoing experiments would validate ONCOlogues as a possible treatment for COVID-19, as well as potentially expedite the discovery of treatments for future coronavirus outbreaks.

Lindsay A. Rosenwald, M.D., Fortress Chairman, President and Chief Executive Officer, said, We are excited to work with the excellent scientists and physicians at Columbia University again. Our last joint effort with Columbia University led to the formation of our partner company, Caelum Biosciences, Inc. (Caelum). Since formation, Caelum has raised approximately $60 million in development funding from a number of sources, with additional amounts available upon the satisfaction of certain milestones and will be initiating two registration clinical trials in the next several weeks. Building upon our success with Caelum, we are grateful to Columbia University for entrusting us to develop this highly innovative technology using oligonucleotides to target genetically driven cancers and coronaviruses. Using a targeted genetic approach to treat cancer has become essential to limiting toxicity and treating patients effectively. This technology has the potential to target mutations that have previously been considered un-druggable. Oncogenuity will aggressively pursue the development of ONCOlogues to ultimately provide patients with new, safe and effective treatment options.

Scientific Co-Founder Jeffrey Rothman, M.D., Ph.D., said, Through rigorous statistical, mechanical and molecular modeling, combined with gene sequence data, we are able to create sequence-specific, targeted therapeutics against oncogenes, which are the cause of and specific to tumor cells. Until now, achieving this goal had been considered nearly impossible. However, with these novel design features, we now have the ability to target cancer while potentially avoiding side effects, which are the main cause of dose-limitation, by design. There is much potential because we are able to target multiple genes and therefore, multiple cancers. Moreover, due to their single-strand format, application toward viral targets such as in COVID are even more facile given their easier accessibility. We are excited and determined to pursue this endeavor with Fortress Biotech and very much welcome their continued support.

About Oncogenuity, Inc.Oncogenuity, Inc. is a biopharmaceutical company focused on the development and commercialization of ONCOlogues for the treatment of genetically driven cancers and coronaviruses. Oncogenuitys lead asset targets a KRAS mutation, G12D. Oncogenuity is located in New York City and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO).

About Fortress Biotech Fortress Biotech, Inc. (Fortress) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloittes 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value while mitigating risk for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the companys portfolio of product opportunities. Fortress has established partnerships with some of the worlds leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Childrens Research Hospital and Nationwide Childrens Hospital. For more information, visit http://www.fortressbiotech.com.

Forward-Looking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words we, us and our may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; risks relating to the COVID-19 outbreak and its potential impact on our employees and consultants ability to complete work in a timely manner and on our ability to obtain additional financing on favorable terms or at all; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contacts:Jaclyn Jaffe and William BegienFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Fortress Biotech Announces Exclusive Worldwide License Agreement With Columbia University to Develop Novel Oligonucleotide Platform for the Treatment...

Orchard Therapeutics plc (NASDAQ:ORTX) Q1 2020 Earnings Conference Call May 7, 2020 8:00 AM ET

Company Participants

Renee Leck Director-Investor Relations

Bobby Gaspar Chief Executive Officer

Frank Thomas Chief Operating Officer

Conference Call Participants

Whitney Ijem Guggenheim

Esther Rajavelu Oppenheimer

Yaron Werber Cowen

Peter Kim Barclays

Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call. [Operator Instructions] I would now like to hand off the conference over to your speaker today, Renee Leck, Director of Investor Relations. Please go ahead, maam.

Renee Leck

Thanks, Sonia. Good morning, everyone, and welcome to Orchards first quarter 2020 investor call. You can access the slides for todays call by going to the Investors section of our website, orchardtx.com.

Before we get started, Id like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, and including those set forth in our annual 10-K filed with the SEC and any other filings we may make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

And with that, Ill turn the call over to our CEO, Bobby Gaspar.

Bobby Gaspar

Thanks, Renee. Hello, everyone, and welcome. Id like to start by first acknowledging the tremendous efforts of our organization and our partners in the health care field to ensure patients in need continue to receive care during this difficult time. Thank you, everyone.

The last few weeks have been an important period for Orchard. Since taking on the leadership, Frank and I, together with the executive team, have thought very carefully about what the new Orchard can become, how we can ensure that Orchard can fulfill its true potential and what we need to do to make that happen. When we think about our strategic vision as a company, its really all based on the potential of the hematopoietic stem cell gene therapy platform, where it can take us and the benefit it can provide for many patient populations even beyond our current portfolio of ultra-rare diseases. We have taken some bold and decisive actions that we believe will allow Orchard to achieve long-term growth and focus the company on sustainable value creation. This vision is supported by a new strategic plan that we have developed and which is built around four pillars. Each of these forms a chapter in our remarks this morning.

First, operating efficiencies. We have made a series of important changes to our operations that will enable us to sharpen our focus and more efficiently execute our strategy, which I will detail in a moment. Second is our commercial build. We are focused on establishing the right model for the diagnosis and treatment of patients undergoing HSC gene therapy, and see the true value of this approach over a series of ultra-rare products.

Third, one of the most exciting areas in gene therapy right now is the innovation taking place in manufacturing technologies that have the potential to deliver economies of scale. We want to be leaders and invest in this space, knowing that our near-term capacity needs are covered by our experienced CDMO network.

Finally, central to this strategy is prioritizing our portfolio to enable the expansion of Orchards pipeline beyond ultra-rare to less-rare indications. We are disclosing two new research programs for the first time today, and these are a genetic subset of frontotemporal dementia or FTD, and a genetic subset of Crohns disease. We believe that the biological and clinical validation that has already been shown in our ultra-rare indications allow us to expand with confidence to these larger indications.

Turning to the first chapter in our new strategic plan. We are focused on improving the operational efficiency throughout the organization. This started with an extensive evaluation over the past six weeks of each program in our portfolio using several criteria that are shown here on the left-hand side of Slide 5. We undertook an objective analysis that involved both financial metrics and strategic considerations in identifying those programs where there was high need for patients and high-value creation for shareholders. As you can imagine, these were difficult decisions given the potentially transformative nature of many of these programs. Each has value, and we intend to realize that in different ways and over different time horizons.

Today, however, we believe our resources are best focused on Metachromatic Leukodystrophy, Wiskott-Aldrich syndrome, the MPS programs and our research programs. This also means that we have a balanced portfolio with late, mid and early stage programs. The programs I havent mentioned such as OTL-101 and ADA-SCID and the transfusion-dependent, beta-thalassemia program, OTL-300, will have a reduced investment moving forward. We will look for alternative ways to realize value with those programs, including through partnerships.

So Slide 6 brings together a summary of the operational changes that weve announced today. We believe these changes were important and necessary to enable Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business. As summarized here, we expect to realize cash savings of approximately $15 million from the prioritization of our portfolio. Another $60 million in savings results from the decision to consolidate our R&D teams to one site and defer the investment in the manufacturing facility.

Finally, the more staged approach to the commercial build-out and 25% reduction to our existing workforce and future headcount planning will each yield another $25 million in savings. All of these cash savings are expected to be realized over 2020 and 2021, and result in total expected savings of $125 million over that period. With the revised plan, we now have cash runway into 2022 and no near term need to finance.

Its worth briefly mentioning that this $125 million savings is after making investments in the following key areas to support our new strategy, shown on Slide 7. In commercial, diagnostic and screening initiatives, including no-charge testing programs to help identify patients with MLD and other neurodegenerative conditions in time for treatment. In manufacturing, the technology, process innovations and efficiencies to drive scalability.

In R&D, initiatives in less-rare diseases that have the potential to fuel the companys future growth in a substantial way. This wasnt just an exercise to reduce expenses, but important decision-making to ensure our capital is deployed in a disciplined manner, while building a pipeline that can leverage our success across all phases of our business.

Now let me turn the call over to Frank to discuss additional key elements of the new plan.

Frank Thomas

Thanks, Bobby, and good morning, everyone. As you can tell from this mornings press release, we have carefully examined each aspect of our business. You heard that a moment ago from Bobby, with the way we are creating operational efficiencies, and I think you will see additional evidence in the next two sections as we summarize our latest thinking around commercial deployment and manufacturing.

Starting with commercial. We understand the importance of developing a commercial model that will demonstrate our ability to execute and bring these therapies to the market successfully. This model and the infrastructure that we build will also be leveraged for any future product launches.

As youll note from the bottom of Slide 9, each rare disease has certain dynamics that will impact the launch trajectory and speed with which we can penetrate the market. In fact, we anticipate our first two potential launches in WAS and MLD having distinct but complementary launch curves, as you can see from the illustrative diagrams.

Let me start with MLD on the left, where we expect to launch first in the EU, followed by the U.S. and then other countries around the world. We think an important inflection point on the revenue curve with MLD will come later when newborn screening is established, providing an opportunity for an acceleration in growth rate. Disease progression is a second important dynamic that will affect market penetration. Because MLD advances so rapidly, it will be important to diagnose patients early and get them treated.

For Wiskott-Aldrich syndrome, the dynamics are very different, and its reflected in the shape of the curve on the right. Unlike MLD, this disease is slower progressing and more readily diagnosed. We believe that WAS will provide an opportunity to treat a number of prevalent patients from the outset and also give us additional long-term revenue stream. This program, the BLA and MAA filings are on track for 2021.

Turning back to MLD for an update on the regulatory time line. We are on track to get a decision from the European Medicines Agency later this year, and if approved, launch in the EU in the first half of 2021. In the U.S., we recently engaged with the FDA on our planned BLA submission of OTL-200 for the treatment of MLD. The FDA has provided written feedback on the sufficiency of the companys data package, including the clinical endpoint, the natural history comparator and the CMC data package.

As a result of this feedback, we intend to file an IND later this year and also seek RMAT designation, both of which we believe will facilitate a more comprehensive dialogue to discuss the data more fully and resolve the open matters before submitting a BLA. We are committed to working closely with the agency, and well provide updated guidance on the new filing time lines for the BLA after further regulatory interaction.

On Slide 10, you can see that were tracking nicely for the launch of OTL-200 in the EU in the first half of 2021, if approved, with Germany being the first country where we expect to treat commercial patients. Many of the prelaunch activities are underway, and the team has been able to keep up momentum during the pandemic to work with key centers and progress with site qualifications. We intend to set up a network of treatment centers where MLD patients are often referred and who also have transplant expertise. These same centers can be leveraged in future launches, especially for programs in the neurometabolic franchise.

I previously mentioned the importance of diagnosis in MLD to identify patients at early stages of disease, and we are taking the necessary steps to achieve long-term success. Beyond typical disease awareness efforts, we are also looking at initiatives such as no-charge diagnostic testing with partners such as Invitae, and we are looking to facilitate newborn screening for MLD with funding of upcoming pilots in New York State and Italy that are designed to validate the assay and provide the data for wider implementation. Success in these key initiatives will support early MLD patient identification.

Coming up quickly behind MLD and the neurometabolic franchise, our two proof-of-concept programs in MPS disorders, where we have made recent progress even during this challenging period with COVID-19. For MPS-I, over the past year, weve shown promising preliminary proof-of-concept data with positive engraftment, biomarker correction and encouraging early clinical outcomes, and we are excited to announce our plan to begin a registrational trial next year, bringing this program one step closer to commercialization.

For MPS-IIIA, we announced late last month that the first patient was treated in a proof-of-concept trial at Royal Manchester Childrens Hospital, with enrollment planned to continue this year and interim data to be released in 2021.

You can see graphically on Slide 12 how the aggregation of these commercial markets lead to sustainable revenue growth. In addition, the infrastructure build is designed to provide the necessary commercial capabilities to realize the potential of the portfolio. On this slide, weve included the incidence figures for MLD and the incidence and prevalence figures for WAS to help you understand each opportunity as we see it today.

Given the dynamics at play for MLD that I described on Slide 9, we believe this opportunity should largely be tied to the incident patient population, which we believe ranges from 200 to 600 patients per year in countries where rare diseases are often reimbursed. Weve taken a more conservative view than previously on the addressable patient and market opportunity in countries such as those in the Middle East and Turkey, where the literature has a wide range of differing incident figures. Also, over time, with improved disease awareness, there may be prevalent patients identified who also could benefit from therapy. Our commercial strategy has always been and continues to be based not only on 1 product, but rather the aggregation of multiple potential products launching off one HSC gene therapy platform and infrastructure.

Turning to manufacturing. Weve also made some key changes to our approach in manufacturing and how we allocate capital in the short and mid-term. On Slide 14, youll see the main tenets of our new manufacturing strategy. First, in the near term, we plan to focus on innovative technologies to enable commercial scalability.

Second, to ensure the appropriate focus on those technologies, weve made a decision to consolidate R&D to a single site in London, which brings together our organization in a more efficient way. This will allow efforts made to improve our manufacturing processes to be quickly and easily shared and then scaled commercially to transfer to our third party manufacturers, all of whom are currently located in Europe. As part of this consolidation, we will close our California site, including the termination of the Fremont project and associated capital spend.

Third, we have strong relationships with CDMOs that will ensure supply of clinical and commercial product to satisfy near-term requirements. And longer term, we intend to identify a new site in the U.S. to eventually bring manufacturing capabilities in-house with a facility that is appropriately sized and fitted for future techniques and operations.

Slide 15 shows the three phases of our approach in manufacturing: invest, partner and build. Today, we are investing, and well continue to invest in technologies such as transduction enhancers, stable producer cell line and closed automated processing of the drug product. This will potentially reduce the amount of vector needed, drive down COGS and potentially change the way products are manufactured, making it less labor-intensive, less expensive and more consistent. In the near and mid-term, we will continue to rely on our manufacturing partners for the early planned launches in MLD and WAS. For example, MolMed has been with these programs since the beginning, and theyve been a reliable commercial partner with Strimvelis.

In addition to our existing CDMO network, we have begun to search for a drug product partner in the U.S. to complete a tech transfer and serve the U.S. market, thereby reducing scheduling challenges and creating some redundancy. And finally, over time, we plan to build in-house manufacturing capabilities closer to when there is a need for additional capacity. This enables us to explore options that are more aligned with our business in terms of scale and timing.

And with that, Ill turn the call back over to Bobby.

Bobby Gaspar

Thanks, Frank. In this section, Im going to briefly highlight the potential of HSC gene therapy to correct not only blood lineage cells, but also how through natural mechanisms, specific cell types may allow correction of disease in specific organ systems and enable expansion of our portfolio into new research indications.

As many of you know, and as shown on Slide 17, through HSC gene therapy, we are able to insert a working copy of the gene permanently into the genome of HSCs, and these genetically modified cells can lead to multiple corrected cell types in the bloodstream, including immune cells, red blood cells and platelets. In addition, HSCs can differentiate into cells of the monocyte macrophage lineage that naturally migrate into various organ systems, and thus gives us an opportunity to deliver genes and proteins directly to those organs, including the brain and the GI tract.

Within the neurometabolic space, in particular, we have understood through our preclinical and clinical programs in MLD, MPS-I and MPS-IIIA how HSC gene therapy can deliver genes and proteins to the CNS to correct neurodegeneration. Here is an example of this natural mechanism at work in Slide 18.

Data shows that there are a population of gene-modified HSCs that can naturally cross the blood-brain barrier, distribute throughout the brain, engraft as microglia and express enzyme that is taken up by neurons. We have seen this approach results in clinical benefits for patients with MLD, and we are also using the same approach for MPS-I and MPS-IIIA. Beyond this, we see that the HSC gene therapy approach could be used to deliver specific genes and proteins for other larger neurodegenerative conditions which have high unmet need.

One of the conditions we are disclosing today, and shown on Slide 19, is a specific genetic subset of frontotemporal dementia, where the underlying pathogenesis has a number of parallels with the neurometabolic conditions that we are already addressing. This program involves a broad strategic alliance with Dr. Alessandra Biffi, Boston Childrens Hospital and Padua University in Italy, to further explore the potential of ex vivo HSC gene therapy in neurometabolic and neurodegenerative conditions.

In other organ systems, such as the GI tract, there are similar mechanisms at work which are illustrated on Slide 20. Tissue resident macrophages in the gut wall are required to respond to bacterial invasion from the gut lumen and prevent infection. In certain disorders, such as X-linked chronic granulomatous disease or XCGD, defects in macrophage function results in an abnormal immune response and severe colitis.

Moving on to Slide 21. We have already seen in our XCGD program the modification of HSCs and migration of gene-modified cells into the gut can lead to resolution of colitis through presumed reconstitution of the immune response. Certain subsets of Crohns disease are also associated with mutations in genes that affect the response of macrophages to infection, and so our clinical observations that HSC gene therapy for XCGD suggest that the same approach may be applicable to this genetic subset of Crohns disease. This preclinical work is ongoing in our Orchard research laboratories.

As we advance our work in FTD and Crohns disease, and assuming we show preclinical proof-of-concept, these will become exciting opportunities for us to expand and address larger patient populations, either alone or in partnership. We believe we have truly just begun to explore the potential for HSC gene therapy in diseases such as these and others, and are excited to share more about the preclinical development of these programs later this year.

So to summarize our path forward on Slide 22, the next 12 to 18 months offers many important milestones as we continue our evolution to a commercial stage company and advance our next wave of clinical stage therapies. We anticipate approval and launch of OTL-200 for MLD in the EU, additional regulatory filings in Wiskott-Aldrich syndrome and MLD, a new registrational study next year in MPS-I, multiple clinical data readouts from our neurometabolic franchise and further detail and progress on our research programs in FTD and Crohns disease.

To wrap up our prepared remarks, we are confident that our new strategic plan and operational decisions announced today will set us on the right path to achieve long-term growth, build sustainable value and serve an even larger number of patients who could benefit from hematopoietic stem cell gene therapy.

Thank you very much. And now well use the rest of the time to answer your questions. So lets have the operator open up the line.

Question-and-Answer Session

Operator

Thank you. [Operator Instructions] And our first question comes from Whitney Ijem from Guggenheim. Your line is now open. Please go ahead.

Whitney Ijem

Hi, guys. Thanks for the question. So first, just wondering, can you give us some more color maybe on the discussions youre having with the FDA in MLD? Kind of what are they looking for? And I guess is the IND just sort of a tool to get RMAT? Or is there additional kind of clinical work you plan you think youll need to do?

Bobby Gaspar

Whitney, Bobby here. Thanks for that. In general, we cant go into all of the details, obviously, of the discussions with the FDA. But I think in the release and in the script, weve talked about the fact that theyve commented on certain endpoints, the natural history, the CMC package, et cetera. Now I think Id just like to say this is a and obviously, a very complex disease, a very ultra-rare population, we have extensive data set, and we have already filed with the EMA. Now for historical reasons, there hasnt been an IND in the U.S., and so we havent had the opportunity to discuss that data in full with the FDA.

What I can say is that we do have an extensive body of data. We want to be able to talk to the FDA and have a comprehensive dialogue to be able to explain that full data set. We feel confident that we have the endpoints that they are looking for and the data that they are asking for. But we need to have that conversation with them in order to be explain to be able to do that fully. So thats why were filing an IND filing, filing the RMAT, so we can have that dialogue. And once we can clarify those issues, then we can go ahead with submission of the BLA.

Whitney Ijem

Okay. Got it. And then just one quick follow-up on MLD. Can you remind us where you are with newborn screening, I guess, both in Europe and then in the U.S.?

Bobby Gaspar

Yes, sure. So newborn screening for MLD, I think, is an important, a very important issue, because, obviously, that means that well be able to get earlier diagnosis and have more patients be able to access therapy. So its a very important part of our kind of diagnostic initiatives in this disease.

What we have so far is that we have worked with a key scientist, where an assay has been developed, thats been published to show that there is an assay that weve done on a dry blood spot to understand the decrease in the enzyme activity and also the increase in the sulfur-type levels. And that assay is now going to be put into pilots, and we are funding a pilot in New York State, and that will start later this year. And were also looking at pilots in other states as well. Were also transferring that assay to Italy and that and were funding a program in Tuscany and in Italy where that will be rolled out. And were also looking for opportunities in other EU states as well. So Id say, there are already two that are going to start, we are looking to fund other pilots as well.

And together, that data will allow us to validate the assay but also allow wider implementation of newborn screening, and also for nomination, for example, onto the WAS panel for implementation in states in the U.S. So I say theres a lot of work going on in order to make sure that happens.

Whitney Ijem

Great. Thanks.

Operator

Thank you. And your next question comes from Esther Rajavelu from Oppenheimer. Your line is now open. Please go ahead.

Esther Rajavelu

Hey, guys. Congrats on all the changes. I guess, my first question again on MLD is Im trying to understand the duration between EU approval and NBS. I dont know if that math or if that graph was drawn to scale, but it looks like its almost a four-year lag from first approval to newborn screening. Can you help us understand the time line there?

Frank Thomas

You mean between EU and U.S. or around newborn screening or both?

Esther Rajavelu

Around newborn screening, generally, between EU approval and newborn screening.

Frank Thomas

Yes, sure. As Bobby mentioned, theres a pretty active program planned around newborn screening that I think we will expect will come over time in order to even apply for the Ross Panel, there are certain requirements that need to be met in terms of the number of patients or a number of children that have to be screened, identifying the positive patients and then you can apply on the Ross Panel.

And then from there, theres a process that you go through in the U.S., at least, on a state-by-state basis to get it added. So I think there are a number of steps along the way. We havent guided specifically on the time line, but I think there are other precedents out there that suggest that this could take years. Once we screen the once we apply for the Ross Panel to get sort of full reimbursement, but obviously, well focus on states initially after that approval that have the largest populations.

Esther Rajavelu

And my Yes, go ahead.

Bobby Gaspar

Esther for I was just going to say for the EU, obviously, were looking for approval for MLD later this year. As far as people screening in the EU is concerned, thats on a country-by-country basis, and sometimes its even certain states. But Ive worked on newborn screening for SCID, for example, in the EU. And now there are numerous countries in the EU that are screening for SCID with a number of pilots also in the pipeline as well. And so with that kind of experience, and we would be looking to kind of really facilitate that uptake in the EU and as in and in the U.S., as Frank has already mentioned.

Esther Rajavelu

Understood. And then the decision to defer CapEx, is that related to some of the time lines for U.S. versus EU approvals and the newborn screening? Or what really kind of went into that delay, given you already have some cost into that facility?

Frank Thomas

Yes. I can start, and Bobby can add on that again. I think, obviously, we continue to believe in-house manufacturing is an important capability that were going to want to have over some period of time. It really comes down to sort of when is the need for that capacity and capability relative to the various programs we have. Working with the CDMOs that we have today, we know that we have capacity for the MLD and WAS launches and for a period beyond the launch. So theres not an imminent need to secure the capacity today, and we think that deferring it makes the most sense. Well continue to work with CDMOs on those launches. We will look at bringing on a U.S. supplier for drug product to be able to more easily service the U.S. market.

And then longer term, look at, potentially, in-house manufacturing at a site and location that we think is more fitted to what the capacity needs will be. So I wouldnt say its tied to any sort of launch time lines because the plan always was to utilize CDMOs for WAS and MLD. But certainly, as those launches roll out and demand grows, our capacity needs will grow and that will be the appropriate time, we think, to make the investment.

Esther Rajavelu

Understood. Thank you very much.

Operator

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Orchard Therapeutics' (ORTX) CEO Bobby Gaspar on Q1 2020 Results - Earnings Call Transcript - Seeking Alpha

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The report covers various areas such as Gene Editing Technologies market size, segmental analysis, regional growth opportunities, drivers and constraints, major vendors in the market, as well as the competitive landscape.

The main aim of this report is to present various updates and data pertaining to the Gene Editing Technologies market and to list out the growth opportunities prevalent for market expansion. A detailed market synopsis, as well as market definitions and an overview of the Gene Editing Technologies market, have been provided in the report.

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The segmentation included in the report is beneficial for readers to capitalize on the selection of appropriate segments for the Gene Editing Technologies sector and can help companies in deciphering the optimum business move to reach their desired business goals.

In market segmentation by types of Gene Editing Technologies, the report covers-

Double Strand Break RepairEngineered Nucleases

In market segmentation by applications of the Gene Editing Technologies, the report covers the following uses-

Gene TherapyEradicating Diseases

The report is inclusive of all the factors that act as drivers and restraints of the market and result in the functioning and changing of trends of the industry. The supply chain and raw material chain are also vividly elucidated in the explanation for the factors of production. The market and consumer behavior that play a greater role in shaping future demand are offered for increased comprehensibility.

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The report contains information on which region brings in the biggest demand and the other regions of the world which contribute significantly to the demand of the products of the Gene Editing Technologies market. The region which are considered in the report for deriving the demand for the Gene Editing Technologies industry are given below:

Key Highlights

An in-depth evaluation of the vendor matrix as well as leading companies that would help understand the competitive scenario in the global Gene Editing Technologies market

Insights about the regulatory as well as investment scenarios of the global Gene Editing Technologies market

An analysis of the factors fuelling the market growth as well as their influence on the projection and dynamics of the global Gene Editing Technologies market

A detailed roadmap presenting the growth opportunities in the global Gene Editing Technologies market alongside the identification of key factors influencing market growth

An exhaustive evaluation of the numerous trends prevailing in the global Gene Editing Technologies market that would help identify market developments

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To summarize, the global Gene Editing Technologies market report studies the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and the trends observed in the market that can either propel or curtail the growth rate of the industry. The market factors impacting the global sector also include provincial trade policies, international trade disputes, entry barriers, and other regulatory restrictions.

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Gene Editing Technologies Market What Factors will drive the Gene Editing Technologies Market in Upcoming Years and How it is Going to Impact on...

The Medical Research Council (MRC) and independent medical research charity LifeArc are streaming 16 million into establishing a network of gene therapy innovation hubs.

The centres will offer clinical grade viral vectors as well as translational and regulatory guidance to support academic-led patient trials of new gene therapies.

Operating as 'centrally coordinated facilities', the hubs aim to address challenges faced by academics as they seek to advance novel gene therapy research into early stage clinical trials, such as a shortage of viral vector production capacity and a complex and evolving translational pathway for gene therapies.

LifeArc and the MRC said they will create the network by providing UK-based research organisations with grants for up to five years, to support the costs associated with expanding or repurposing existing viral vector production centres.

The selected centres, or hubs, will also have access to LifeArcs translation advice and support.

We hope that through this unique collaboration with the MRC, LifeArc can offer its funding and expertise in technology transfer and translational science to support the progression of promising gene therapies, said Dr Melanie Lee, the charity's chief executive. Translation of advanced therapies will be a core focus of LifeArcs future strategy for delivering significant new patient benefits.

MRC executive chair Professor Fiona Watt added: Through this partnership, we aim to support clinical development of the most exciting gene therapy projects from the UKs world-leading academic researchers. This investment will streamline and accelerate progress towards a new generation of genetic medicines for patients.

Read more:
LifeArc, MRC create 16m fund to set up gene therapy hubs - PharmaTimes

SAN RAFAEL, Calif., May 3, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR will receive an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales. The company did not disclose financial terms. BioMarin management reiterated its 2020 GAAP net income guidance of $20 to $80 million, inclusive of this collaboration.

The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM). Additionally, the companies will collaborate on several of DiNAQOR's other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms. Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR.

"With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer at BioMarin. "This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies."

"We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyophathies," said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin. "We believe there is tremendous potential in combining our experience in gene therapy research and development with DiNAQOR's in-depth knowledge of genetic heart diseases."

DiNAQOR was founded and is led by several leading pharmaceutical and biotechnology executives and academics with deep cardiology and gene therapy expertise. The company's holistic approach to gene therapy is focused on gene therapies for the heart that deliver a medical solution that can safely deliver gene therapies to the heart muscle, ensure transduction of the cardiac cells, and limit the exposure of the therapy to other organs.

"BioMarin is a global leader in rare disease research, development and commercialization, and their commitment to DiNA-001 is a powerful validation of DiNAQOR's gene therapy platform," said Dr. Johannes Holzmeister, Co-Founder, Chairman and CEO at DiNAQOR. "We believe our platform has many potential applications and this milestone agreement will enable us to invest in expanding our genetic medicine pipeline."

"Momentum for gene therapies continues to build, and BioMarin has demonstrated tremendous scientific, clinical, and manufacturing leadership and expertise in the space," said Thomas Voit, M.D., Ph.D., Co-Founder and Chief Scientific Officer at DiNAQOR and Director of the Biomedical Research Centre at the Great Ormond Street Hospital and the UCL Institute of Child Health, University College London. "We are looking forward to combining our strengths to expand the promise of gene therapy treatments by targeting the heart muscle to treat rare genetic cardiomyopathies."

About HCM and MYPBC3

Hypertrophic cardiomyopathy (HCM) is one of the most common genetic heart diseases, with about 500,000 patients diagnosed with HCM worldwide. Up to 60% of HCM cases have a genetic origin, and it is estimated that 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C (MyBP-C).

HCM affects the heart muscle, causing the muscle to enlarge. HCM patients have an increased risk of developing heart failure and life-threatening arrhythmias. There are no approved pharmacological treatment options available that address the underlying disease biology of HCM and invasive surgery or heart transplantation may be the only options available for patients with advanced disease.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

About DiNAQOR

Founded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001 is focused on the treatment of MYBPC3-linked cardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

Forward Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: BioMarin's expectations regarding the announced collaboration, the prospects for the lead and follow on pipeline products and it's 2020 GAAP profitability. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning the programs; the ability to manufacture the product candidates, BioMarin's revenue for 2020, especially with the possible impact of COVID-19, and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

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Company Codes: NASDAQ-NMS:BMRN

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BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic...

When the axons that extend from neurons break during a spinal cord injury, the result is often a lifelong loss of motor functioning, because vital connections from the brain to other body parts cannot be restored. Now, researchers from Temple Universitys Lewis Katz School of Medicine say they may have found a way to recover some functions lost to axon breaks.

The researchers discovered that boosting levels of a protein called Lin28 in injured spinal cords of mice prompts the regrowth of axons and repairs communication between the brain and body. Lin28 also helped repair injured optic nerves in the animals, they reported in the journal Molecular Therapy.

The Temple team zeroed in on Lin28 because its a known regulator of stem cells, meaning it controls their ability to differentiate into various cells in the body. The researchers examined the effects of Lin28 on spinal cord and optic nerve injuries using two mouse models: one that was engineered to express extra Lin28 and another that was normal and was given the protein after injury via a viral vector.

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All of the mice experienced axon regeneration, the researchers reported. But they found that the best results occurred in the normal mice that received Lin28 injections post-injury. In fact, in animals with optic nerve injuries, the axons regrew to the point where they filled the entire tract of the nerve.

Lin28 treatment after injury improved coordination and sensation in the mice, the researchers reported.

"We observed a lot of axon regrowth, which could be very significant clinically, since there currently are no regenerative treatments for spinal cord injury or optic nerve injury," said senior author Shuxin Li, M.D., Ph.D., professor of anatomy and cell biology at the Lewis Katz School of Medicine, in a statement.

RELATED: Gene therapy with 'off switch' restores hand movement in rats with spinal cord injury

Lin28 is already a target of interest, though it has garnered the most attention so far in cancer research. Startup Twentyeight-Seven Therapeutics is developing a small molecule that inhibits the protein in the hopes that doing so will boost Let-7, a cancer-suppressing microRNA. The company raised more than $82 million in a series A financing last year.

Several new approaches for repairing spinal cord injuries are under investigation, most notably gene therapy. King's College researchers are working on a gene therapy that repairs axons by prompting the production of the enzyme chondroitinase. A UT Southwestern team is targeting the gene LZK to increase levels of supportive nervous system cells called astrocytes in response to spinal injuries.

The Temple team has a two-pronged approach to further developing their Lin28-directed treatment. They hope to develop a vector that can be safely delivered by injection and that would deliver the therapy directly to damaged neurons. They also plan to study other molecules in the Lin28 signaling pathway.

"Lin28 associates closely with other growth signaling molecules, and we suspect it uses multiple pathways to regulate cell growth," Li said, potentially revealing other therapeutic molecules that could further boost neuron repair.

Originally posted here:
Repairing spinal cord injuries with a protein that regulates axon regeneration - FierceBiotech

Hours after Gilead announced that an NIH trial testing their antiviral drug remdesivir in Covid-19 patients had succeeded, NIAID director Anthony Fauci sat on a couch in the Oval Office and gave the world the top-line readout.

The drug induced a 31% improvement on the primary endpoint of time to recovery: 11 days in the drug arm compared to 15 days in the placebo arm, he said, adding that patients taking the drug appeared less likely to die, with an 8% mortality rate in the drug arm compared to 11% in patients given the placebo.

The mortality data were not yet statistically significant, he cautioned but were trending in the right direction. Fauci, surrounded by President Trump, Vice President Mike Pence and several other advisors, said the news was a very optimistic sign in the hunt for treatments to fight the virus.

Although a 31% improvement doesnt seem like a knockout 100%, it is a very important proof of concept, he said. Because what it has proven, is that a drug has blocked this virus.

Fauci said more details would come and that the study would be submitted to a peer-reviewed journal. Trump, who deferred to Fauci in giving the readout, echoed Faucis commentary.

Its a beginning, that means you build on it, Trump said. But its a very positive event.

Shortly after the briefing, the New York Times reported that the FDA was preparing to issue an emergency use authorization for the drugs use in Covid-19. In an email to Endpoints News, the FDA did not confirm or deny the Times report, but a spokesperson said the agency has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate.

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BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases - Endpoints News

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Summary

Its an understatement to say that Facebook has changed the world its created an ability for people to be transparent about their experiences, lives, and opinions, for better or worse. In the case of Homology, its for the latter. Homology, a gene therapy company whose technology has already been scrutinized by scientists as untrue, has just one product in clinical trials, HMI-102, for the rare disease phenylketonuria ("PKU"). As Med Genie reported back in January, FIXXs trial update showed interim phenylalanine (Phe) results that suggested HMI-102 was not efficacious for low and mid-dose patients. Our note today highlights a data point the company would very much like you not to know the only patient in FIXXs high dose cohort posted her results on Facebook, and they show that HMI-102 is unlikely to reach trial endpoints even at a high dose. We believe this patient was forced to take her posts down as a result, and management selectively disclosed the issue to the sell side, providing comments about the drugs safety, and conveniently ignoring the implications to efficacy and the business. Maybe they were hoping to raise capital before officially announcing trial results. Who knows? We believe that the HMI-102 program is dead in the water, and since its progress is the major driver of FIXXs value, we believe that the stock should trade to cash value, or $5.80, down 56% from the April 27th close.

Background

The best shorts are often one-trick ponies, but rarely do we find one where a single data point completely upends the long investment case. Homology Medicines, FIXX, is one of those rare finds.

Homology is a gene therapy company which has their first and lead product candidate, HMI-102, in a dose-escalation Phase 1/2 trial (pheNIX) for phenylketonuria. FIXX also has 2 IND-enabling programs and some discovery stage programs, but the HMI-102 trial is the companys main shot at viability.

PKU is a relatively rare disease, with a U.S. incidence of approximately 350 cases per year and a prevalence of just 16,500, per FIXXs 10-k. PKU is tied to mutations in the gene that control PAH, an enzyme that metabolizes phenylalanine, or Phe. The condition results in a deficiency in the enzymatic activity of PAH, causing an excess in Phe in the bloodstream that can result in intellectual disability. PKU patients are identified soon after birth, and are primarily treated with a Phe-restricted diet. FIXXs HMI-102 seeks to modify the underlying genetic cause of PKU, effectively curing the disease and allowing patients to eat normally and not experience the cognitive and metabolic issues from higher than normal Phe.

FIXXs technology, which does not use the CRISPR approach to gene therapy, has already been subject to scrutiny, with David Russell, a researcher at the University of Washington, saying, Whats surprising is this company raised so much money on something thought to be untrue in the scientific community, in a piece in MIT Technology Review.

We believe that recent revelations about the efficacy of HMI-102 support this skepticism and show that the drug is not efficacious, kicking out the one leg holding up FIXXs business, and that FIXX should trade to cash value, or $5.80 per share, down 56% from the April 27th closing price.

The pheNIX trial

The pheNIX trial for HMI-102 launched in June 2019, and its primary efficacy endpoint is two plasma Phe measurements below 360 umol/L (or 6 mg/dL) between 16 and 24 weeks after dosing. Following evaluation of the first two patients in a cohort, a decision can be made to either escalate to the next dose level, add a third patient or expand the cohort at the selected dose level.

Now before we review whats new, its helpful to note that a mouse study presented at the 21st Annual Meeting of the American Society of Gene & Cell Therapy titled Sustained Correction of Phenylketonuria by a Single Dose of AAVHSC Packaging a Human Phenylalanine Hydroxylase Transgene showed that HMI-102 showed an effect to mouse Phe levels just one week after dosing in fact, mouse Phe levels remained relatively flat after that first drop.

This would imply that the therapy shows its effect soon after dosing and the longer timelines contemplated in the study endpoints are to indicate that the effect is long lasting. Sure enough, we see a similar dynamic in the pheNIX trial data released by FIXX in December 2019. There were 3 patients examined here from the 10-k: (n=2 patients in the low-dose Cohort 1 and n=1 patient in the mid-dose Cohort 2) as of the data cutoff of December 2, 2019. A fourth patient was dosed in Cohort 2 subsequent to the data cutoff date and was therefore not included in the analysis.

In this release, we see the two patients in the low-dose cohort experienced no improvement in fasting Phe after dosing or even 12 weeks later. The cohort 2 patient, getting a mid-dose, showed an improvement in Phe level immediately after dosing, but their Phe level did not fall below the 360 umol/L threshold defined as the primary endpoint (it stayed around 500) calling into question the efficacy of the treatment, which Med Genie mentions in their piece (from the 10-k):

Damning revelations

On March 5, 2020, a woman we will call Miss A started a Facebook group (since made private or taken down on April 15, 2020) to discuss her experience getting gene therapy for PKU:

On March 9, 2020, Miss A, who lives in Normal, IL tells us shes going to Chicago, which happens to be one of the pheNIX trial sites:

The next day, Miss A provides us with enough to data to know that she is Patient 5, part of the high dose cohort 3 in FIXXs HMI-102 trial (cohort 3, mentioned by Miss A, would be the next dose up from the cohort 2, the mid-dose cohort):

Dr. Burton appears to be Dr. Barbara K. Burton, a physician focused on PKU at the Ann & Robert H. Lurie Childrens Hospital of Chicago, a trial site mentioned in the pheNIX trial description on clinicaltrials.gov:

This, taken together with the fact that Biomarins own PKU trial was in too early a stage to enroll a Cohort 3 patient in March 2020, its probably safe to say that Miss A is taking part in FIXXs pheNIX trial.

On March 11, Miss A receives her infusion:

Miss A then shares a series of updates on how she is feeling and the progress of her weekly visits post-infusion. Six days post infusion, she says she hasnt gotten any test results back, but that it may take 2 or 3 weeks to get a Phe level:

27 days post-infusion, Miss A tells a FB commenter that she wont get Phe levels till six weeks post-infusion:

And then 35 days post-infusion, on April 15, 2020, a bombshell Miss A gets her Phe levels, and at 25 mg/dL or 1497uMol/L, they are well above the 360 uMol/L endpoint threshold after 5 weeks (and after the drug typically takes effect), suggesting that HMI-102 is not efficacious even for a high dose patient:

Just a few hours after her post, Miss As entire group is either taken down or made private, perhaps at the demands of FIXX itself.

Management's disclosure problem

We believe that management was aware of this post and tried to manage the perception of it by talking to the sell side and to select investors. In fact, Oppenheimers equity sales desk was sharing the below email conversation between their analyst Matt Biegler and FIXXs Theresa McNeely to explain the price action on April 15th:

Who was Theresa planning to speak to? We know that Bairds Madhu Kumar got a call:

But when we asked Theresa ourselves, she was much less forthcoming:

It appears to us that FIXX chose to inform the sell side and potential larger investors, who appear to be selling the stock (it has dramatically underperformed biotech broadly), but not the average investor. This is a significant red flag that investors should be aware of.

Why all this matters

Because the mouse study and the Cohort 2 data showed an effect to Phe levels one week after dosing rather than a gradual reduction, we can conclude that Miss As level, at 1497 uMol/L, is probably not going to get better, unfortunately. Further, her levels several weeks into the trial are still much higher than the threshold level specified in the primary endpoint of 360 uMol/L. This means that the therapy is showing zero efficacy even for a high dose patient. This data point is damning given the size of the trial and importance of the high dose patient in light of the lack of efficacy in the low and mid-dose cohorts.

Now the sell side may parrot management and say that there is no way to know whether Miss A is who she says she is, but the evidence is certainly strong supporting her case. They may say that there was no way for her to know her Phe level, but her posts show that she expected to receive them. They may also say that the drug is safe, and that liver enzyme elevation should be expected in a therapy like HMI-102, but thats all beside the point. The point is that the Phe level Miss A received 5 weeks after infusion show that HMI-102 is not efficacious.

These results support the skepticism around FIXXs use of the AAVHSC vector in liver directed gene therapy, which, based on the results thus far, and in particular Miss As results, appear to show zero efficacy and thus makes it inferior to Biomarins AAV5 vector.

Furthermore, if management thought this information was material enough to talk to the sell side analysts covering the stock, why not put it in an 8-k or even a press release for the benefit of their entire shareholder base? Given the materiality of the information, wouldnt all shareholders have benefited from the same level of disclosure rather than be kept in the dark? This is behavior consistent with that of MDXG and ALLK, both companies with executives formerly from reputed companies who have seen their stock prices demolished.

For FIXX, we believe that this is a huge problem the HMI-102 pheNIX trial is the ONLY program in their portfolio that is in the Phase 1/2 stage, and thus the primary path to viability for the company. With this piece of data showing that HMI-102 is not efficacious, we believe that the program is likely worthless and unlikely to proceed to commercialization. While FIXX may try to apply HMI-102 to other indications, we believe that doing so would essentially restart the trial and approval clock without making up for the lost time to market from a failed PKU trial.

Furthermore, social media matters. While FIXX management may be dismissive of people posting on Facebook, these posts have value. In the case of Allakos (ALLK), Seligman Research put together a barn burner of a report which included numerous Facebook posts questioning the efficacy, safety, and trial design of ALLKs drug candidate. Since that report, ALLK stock is down approximately 51%, and ALLK actually has several later stage trials.

In the case of FIXX, we believe that HMI-102 in PKU is the ONLY path to viability given the lack of efficacy of HMI-102 at high dose, we believe that the HMI-102 program is dead, and that the stock should trade to its cash value per share, or $5.80 per share, down 56% from the April 27th close price.

Disclosure: I am/we are short FIXX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: This piece is our opinion and not an offer to buy/sell any securities. We're biased, you're biased, so do your own work and make your own decisions.

Link:
Can't FIXX This - We Believe Homology's HMI-102 Is In Trouble - Seeking Alpha

After a comprehensive analysis, Mrinsights.bizhas published a new research study titled GlobalHemophilia Gene Therapy Market Growth 2020-2025 that covers the latest and upcoming industry trends and offers a global spectrum of the Hemophilia Gene Therapy market, and future forecast from 2020 to 2025 years. The market is bifurcated into product type, application, key manufacturers and key regions and countries. The research assists users to achieve competitive leverage with acquiring and preserving market position as key aims of the program. The report expands on details pertaining to contributions by key players, demand and supply analysis as well as market share growth of the industry.

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It covers the leading manufacturers profiles involving market entry strategies, production analysis, market share, revenue forecast. In addition, the regional analysis of the industry is offered where the report delivers analytical information on regional segmentation. Top leadingcompaniesof global Hemophilia Gene Therapy market are:Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

Industry Trends And Opportunities:

The report provides an investigation into the global Hemophilia Gene Therapy market status, shares, supply-demand, market drivers, challenges and opportunities, and geological areas. Key trends and development opportunities are covered in this analysis report. The report then serves information on sales and market share estimates by-product as well as a profile of the companys business.

Regional Analysis:

This research report consists of the worlds crucial region Hemophilia Gene Therapy market share, size (volume), trends including the product profit, price, value, production, capacity, capability utilization, supply, and demand and industry growth rate. It helps readers to understand strategies to make sound investments. The regions are extensively analyzed with respect to every parameter of the geographies in question, comprising: Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries).

The Report Addresses The Following Queries Related To The Market:

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Furthermore in this report, external as well as internal factors that are supposed to affect the business positively or negatively have been investigated. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors by region on the global Hemophilia Gene Therapy market is given in the report. A further section of the report discusses expansion plans of companies, key mergers and acquisitions, funding and investment analysis, company establishment dates, revenues of manufacturers, and their areas served and manufacturing bases.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Global Hemophilia Gene Therapy Market 2020 Top Companies, Industry Demand, Business Review and Regional Analysis by 2025 Cole Reports - Cole of Duty

The Gene Therapy Market report market intelligence study intended to offer complete understanding of global market scenario with the Impact of COVID-19 (Corona Virus). It attempts to analyze the major components of the Market which have greater influence on it. This includes various elements of significant nature including market overview, segmentation, competition landscape, Market chain analysis, key players stratergyand more. Also, the report provides a 360-degree overview of global market on the basis of various analysis techniques including SWOT and Porters Five Forces. Approximations associated with the market values over the forecast period are based on empirical research and data collected through both primary and secondary sources. This might help readers to understand the strengths, opportunities, challenges and perceived threats of the market.

Based on Classification, each type is studied as Sales, Market Share (%), Revenue (Million USD), Price, Gross Margin and more similar information. The report can help to realize the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Gene Therapy industry.

The Gene Therapy Market report wraps:

There are 13 Chapters to thoroughly display the Gene Therapy market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

In the end, The objective of the market research report is the current status of the market and in accordance classifies it into a few objects. The report takes into consideration the first market players in every area from over the globe.

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

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Gene Therapy Market to witness impressive Growth in Production-Consumption Ratio through 2026 - Latest Herald

CAMBRIDGE, Mass., May 04, 2020 (GLOBE NEWSWIRE) -- TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, today announced the appointment of Stephen Webster to its Board of Directors. With nearly 30 years of biotechnology industry experience in raising capital, business development transactions and operations, Mr. Webster has played important roles as the Chief Financial Officer of Spark Therapeutics, Optimer Pharmacuticals and Adolor Corporation. In connection with Mr. Websters arrival, Mitchell Finer, Ph.D., will be stepping down from the Board of Directors effective May 4, 2020 but will continue to serve TCR2 in an advisory capacity.

"We are delighted to welcome in another successful cell and gene therapy executive as Stephen Webster joins our Board of Directors. His distinguished track record of leading companies through periods of growth will prove invaluable at this moment in time as we prepare to present clinical data for our two lead programs, TC-210 and TC-110, and advance a third mono TRuC-T cell therapy towards the clinic," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "His business development transaction expertise will be particularly useful in helping us strike the right partnerships in pursuing our goal of developing innovative T cell therapies for patients suffering from cancer.

Mr. Webster served as the Chief Financial Officer of Spark Therapeutics, a publicly traded gene therapy biotechnology company, from July 2014 until its acquisition by Roche for $4.3 billion in December 2019. He was previously Senior Vice President (SVP) and Chief Financial Officer of Optimer Pharmaceuticals, a publicly traded biotechnology company, from July 2012 until its acquisition by Cubist Pharmaceuticals in October 2013. Prior to joining Optimer, Mr. Webster served as SVP and Chief Financial Officer of Adolor Corporation, a biopharmaceutical company, from 2008 until its acquisition by Cubist Pharmaceuticals in 2011. Mr. Webster also served in leadership positions in the investment banking healthcare groups of Broadpoint Capital and PaineWebber Incorporated.

Mr. Webster has served as a director of Nabriva Therapeutics AG (formerly Nabriva Therapeutics plc), a publicly traded biopharmaceutical company, since August 2016 and Viking Therapeutics, a publicly traded biopharmaceutical company, since May 2014. Mr. Webster received an A.B. in Economics from Dartmouth College and an M.B.A. in Finance from The Wharton School of the University of Pennsylvania.

I am thrilled to become a director of TCR2 Therapeutics, where there is a great opportunity to turn a distinctive TRuC-T cell platform into a series of novel treatments of cancer, said Mr. Webster. I look forward to working with the TCR2 leadership team and Board of Directors to add my business development expertise in helping the Company achieve its goal of bringing transformational therapies to people living with serious solid tumors and hematologic malignancies.

On behalf of TCR2 and the Board of Directors, I would like to thank Mitchell Finer for his many contributions to the rapid growth of our company, added Dr. Menzel. Our strategy to begin with an automated cell therapy manufacturing process benefited from working very closely with Dr. Finer, whose three decades of cell therapy manufacturing leadership provided us a significant competitive advantage in the cell therapy landscape. We look forward to continuing to benefit from his insights as he transitions from a Board member to a consultant.

About TCR2 Therapeutics

TCR2Therapeutics Inc.is a clinical-stage immunotherapy company developing the next generation of novel Tcell therapies for patients suffering from cancer.TCR2sproprietary T cell receptor (TCR) Fusion Construct Tcells (TRuC-T cells) specifically recognize and kill cancer cells by harnessing signaling from the entire TCR, independent ofhuman leukocyte antigens (HLA). In preclinical studies, TRuC-T cells have demonstrated superior anti-tumor activity compared to chimeric antigen receptor T cells (CAR-T cells), while exhibiting lower levels of cytokine release. The Companys lead TRuC-T cell product candidate targeting solid tumors, TC-210, is currently being studied in a Phase 1/2 clinical trial to treat patients with mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. The Companys lead TRuC-T cell product candidate targeting hematological malignancies, TC-110, is currently being studied in a Phase 1/2 clinical trial to treat patients with CD19-positive adult acute lymphoblastic leukemia (aALL) and with aggressive or indolent non-Hodgkin lymphoma (NHL). For more information about TCR2, please visitwww.tcr2.com.

Forward-looking Statements

This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include, but are not limited to, express or implied statements regarding the development of the Companys product candidates, future business plans and the therapeutic potential of its product candidates and platform.

The expressed or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities, including review under accelerated approval processes; orphan drug designation eligibility; regulatory approvals to conduct trials or to market products; TCR2s ability to maintain sufficient manufacturing capabilities to support its research, development and commercialization efforts, whether TCR2's cash resources will be sufficient to fund TCR2's foreseeable and unforeseeable operating expenses and capital expenditure requirements, the impact of the COVID-19 pandemic on TCR2s ongoing operations; and other risks set forth under the caption "Risk Factors" in TCR2s most recent Annual Report on Form 10-K, most recent Quarterly Report on Form 10-Q and its other filings with theSecurities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although TCR2believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur.

Moreover, except as required by law, neither TCR2nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investor and Media Contact:

Carl MauchDirector, Investor Relations and Corporate CommunicationsTCR2 Therapeutics Inc.(617) 949-5667carl.mauch@tcr2.com

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TCR Therapeutics Announces Veteran Finance Executive Stephen Webster Joins its Board of Directors - GlobeNewswire

The Global CRISPR technology market is expected to reach USD 1,715 million by 2023 from USD 562 million in 2018, at a CAGR of 25%

The rising funding from government and private organizations and the high adoption of CRISPR technology are major factors driving the growth of CRISPR technology market002E

How much is the CRISPR Technology Market worth?

MarketsandMarkets forecasts the CRISPR technology market is expected to reach USD 1,715 million by 2023 from USD 562 million in 2018, at a CAGR of 25% during the forecast period. The global CRISPR services market is segmented into four major regions, namely, North America, Europe, the Asia Pacific, and the Rest of the World.

In 2018, North America accounted for the largest share of this market majorly due to the rising government and private funding, presence of major pharma and gene therapy companies, and the adoption of CRISPR in a number of applications.

The CRISPR products segment is expected to command the largest share of the CRISPR Products market during the forecast period.

The CRISPR Products market, by product and service, is estimated to be dominated by the products segment in 2018. This is attributed to the fact that the CRISPR Products is being adopted quickly by academics and researchers, pharma and biotech companies.

The enzymes segment is expected to account for the largest share of the products market, being one of the key ingredients in the CRISPR process. Companies like Merck KGaA and Thermo Fisher Scientific are providing hands-on training to researchers, which will increase the demand for CRISPR products in the future.

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Biomedical applications to occupy the majority of the market, by application, and grow at the fastest rate during the forecast period.

The biomedical applications segment is projected to be the fastest-growing segment of the CRISPR services market, by application, during the forecast period. Developments in gene therapy, drug discovery, and diagnostics, due to the application of CRISPR, are driving the growth of this biomedical segment.

Many companies have also invested in drug discovery and gene therapy companies that are using CRISPR technology.

North America is expected to account for the largest market share during the forecast period.

North America is estimated to account for the largest share of the CRISPR services market in 2018. This is majorly attributed to the rising government and private funding, presence of major pharma and gene therapy companies, and the adoption of CRISPR in several applications.

Furthermore, crops that are treated with CRISPR-based gene editing are not considered as GMOs in US; this has attracted a number of agricultural companies to focus on the commercialization of CRISPR-edited crops.

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Who are the leading vendors operating in CRISPR Services Market?

Cellecta, Inc. (US), Thermo Fisher (US), GeneCopoeia, Inc. (US), Applied StemCell (US), Synthego Corporation (US), OriGene Technologies (US), Horizon Discovery (UK), Merck (Germany), and GenScript (US).

Thermo Fisher Scientific has established its presence in diversified life sciences markets; this has helped it to minimize risks and dependency on any business segment. The company has a strong product portfolio and brand image, which enables it to strengthen its position in the market.

The company has initiated a promotional campaign in which it conducts workshops and provides hands-on training to researchers in academic and research institutes working on CRISPR. This campaign is being organized to spread awareness on and promote CRISPR technology.

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CRISPR Technology Market worth USD 1715 million by 2023 according to a new research report - WhaTech Technology and Markets News

Cancer Gene Therapy Marketis expected to reach 5075 million by 2026 from XX million in 2018 at CAGR of XX %.

The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.

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Cancer Gene Therapy Research Report is a method of therapeutic delivery of genetic material into a patients cells as a drug to treat disease and compensate for abnormal genes or to make a beneficial protein. Cancer cells modify themselves (called faults or mutations) in several of their genes which make them divide very often and form a tumor. Gene therapy provides various methods by which doctors can cure cancer like:

Inactivation of a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Replacement of mutated gene that causes disease with a healthy copy of the gene.Cancer caused 9.02 million deaths in 2017, and is expected to reach 10 million by 2026; the increased no of cancer prevalence is because of increased body mass index, low fruit and vegetable intake, lack of physical activity, increased tobacco and alcohol consumption. Also, Factors like increasing ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines, High success rate during the preclinical and clinical trial, Increase in funding for R&D in cancer gene therapy, Increase in geriatric population, favorable government regulations will fuel the global Cancer Gene therapy market. However, the high cost of gene therapy treatment and unwanted immune responses will restrain market growth.The highest revenue-generating region is North America in 2017 followed by Europe; reasons behind this increased growth rate are well-established health care facilities, high per capita health care expenditure, and extensive R&D activities for the gene therapy in the region. However, Asia Pacific is projected to expand at a moderate growth rate during the forecast period.

Key Highlights:

Assessment of market definition along with the identification of key players and an analysis of their strategies to determine the competitive outlook of the market, opportunities, drivers, restraints, and challenges for this market during the forecast period Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, growth strategies that can In-depth analysis of the industry on the basis of market segments, market dynamics, market size, competition & companies involved value chain Cancer Gene Therapy market analysis and comprehensive segmentation with respect to the therapy and geography to assist in strategic business planning Cancer Gene Therapy market Research Report analysis and forecast for five major geographies North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key countries Complete quantitative analysis of the industry from 2017 to 2026 to enable the stakeholders to capitalize on the prevailing market opportunities.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

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Research Methodology:

An objective of the study is to estimate the size of the Cancer Gene Therapy market for 2017 and projects its demand till 2026 with quantitative and qualitative analysis of Cancer Gene Therapy market. Industry experts have studied various industry journals, directories, have referred information available with various associations to identify, collect information and to put it in articulated format to make useful for all stake holders in the industry. Primary research has been done and various industry experts and suppliers from worlds wide have given their inputs to make the study more accurate.Key Players in the Cancer Gene Therapy Market Are:

Shenzhen Sibiono Genetech Adaptimmune Glaxosmithkline Oncogenex Pharmaceuticals Bluebird Bio, Inc. Synergene Therapeutics Shanghai Sunway Biotech Biocancell Celgene MerckKey Target Audience:

Cancer Gene Therapy Market Investors Cancer Gene Therapy Marketing Players Pharmaceutical and Biotechnology Companies Healthcare Institutions (Individual Surgeons, Medical Schools, Group Practices, Hospitals, and Governing Bodies) Diabetes Drugs Market Research Associations Diabetes drug Manufacturers & DistributorsScope of the Cancer Gene Therapy Market

Research report categorizes the Cancer Gene Therapy market based on Therapy and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Cancer Gene Therapy market with key developments in companies and market trendsCancer Gene Therapy Market, By Therapy:

Oncolytic Virotherapyo Adenoo Lentiviruso Retro Viruso Adeno Associated Viruso Herpes Simplex Viruso Alpha Viruso Vaccinia Viruso Simian Viruso Others Gene Transfero Naked Plasmid Vectoro Electroporationo Sonoportiono Magnetofectiono Gene Gun Gene-Induced Immunotherapyo Delivery of Cytokines Geneo Delivery of Tumor Antigen GeneCancer Gene Therapy Market, By Geography:

North America Europe Asia Pacific Middle East & Africa Latin America

MAJOR TOC OF THE REPORT

Chapter One: Cancer Gene Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cancer Gene Therapy Market Competition, by Players

Chapter Four: Global Cancer Gene Therapy Market Size by Regions

Chapter Five: North America Cancer Gene Therapy Revenue by Countries

Chapter Six: Europe Cancer Gene Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Cancer Gene Therapy Revenue by Countries

Chapter Eight: South America Cancer Gene Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cancer Gene Therapy by Countries

Chapter Ten: Global Cancer Gene Therapy Market Segment by Type

Chapter Eleven: Global Cancer Gene Therapy Market Segment by Application

Chapter Twelve: Global Cancer Gene Therapy Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cancer Gene Therapy Market Report at:https://www.maximizemarketresearch.com/market-report/cancer-gene-therapy-market/520/

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Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.

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Cancer Gene Therapy Market : Research Report - MR Invasion

The report aims to provide an overview of Global Assistive Technologies for Visual Impairment Market along with detailed segmentation of market by applications, end-users and five major geographical regions. Global Assistive Technologies for Visual Impairment market is expected to witness an aggressive growth during the forecast period.

Leading players of Assistive Technologies for Visual Impairment Market:VFO Group, TQM, Humanware, Handy Tech Elektronik GmbH, Perkins Solutions, Papenmeier, Amedia, Eurobraille, Nippon Telesoft, Brailletec, VisionCue

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The Global Assistive Technologies for Visual Impairment Market Analysis to 2025 is a specialized and in-depth study of the Assistive Technologies for Visual Impairment market with a focus on the global market trend. The report aims to provide an overview of the global Assistive Technologies for Visual Impairment market with detailed market segmentation by service, technology, industry vertical, and geography. The global Assistive Technologies for Visual Impairment market is expected to witness high growth during the forecast period.

Segmentation by Type:Braille Displays

Note Takers

Magnifiers

Braille Printers and Embossers

Braille Writers

Segmentation by Application:Blind School

Disabled Persons Federation and Hospital

Enterprises and Social Organizations

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Exclusive Research Report on Assistive Technologies for Visual Impairment Market, Size, Analytical Overview, Growth Factors, Demand and Trends...

The Enzyme Replacement Therapy Market report market intelligence study intended to offer complete understanding of global market scenario with the Impact of COVID-19 (Corona Virus). It attempts to analyze the major components of the Market which have greater influence on it. This includes various elements of significant nature including market overview, segmentation, competition landscape, Market chain analysis, key players stratergyand more. Also, the report provides a 360-degree overview of global market on the basis of various analysis techniques including SWOT and Porters Five Forces. Approximations associated with the market values over the forecast period are based on empirical research and data collected through both primary and secondary sources. This might help readers to understand the strengths, opportunities, challenges and perceived threats of the market.

Based on Classification, each type is studied as Sales, Market Share (%), Revenue (Million USD), Price, Gross Margin and more similar information. The report can help to realize the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Enzyme Replacement Therapy industry.

The Enzyme Replacement Therapy Market report wraps:

There are 13 Chapters to thoroughly display the Enzyme Replacement Therapy market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

In the end, The objective of the market research report is the current status of the market and in accordance classifies it into a few objects. The report takes into consideration the first market players in every area from over the globe.

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

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Enzyme Replacement Therapy Market Forecast: By Regions, Type and Application with Sales and Revenue Analysis 2020-2026 - Latest Herald

Facts & Factors Market Research, a leading market research and consulting firm added the latest industry outlook report on Cancer Gene Therapy Market By Type (Ex-vivo and In-vivo) and By Product (Viral Vectors, Non-viral Vectors, and Others): Global Industry Outlook, Market Size, Business Intelligence, Consumer Preferences, Statistical Surveys, Comprehensive Analysis, Historical Developments, Current Trends, and Forecasts, 20202026 consisting of 190+ pages during the forecast period 2019 to 2026 and the Cancer Gene Therapy Market report offers comprehensive research updates and information related to market growth, demand, and opportunities in the Cancer Gene Therapy Market.

The Cancer Gene Therapy Market report provides an enormous info as well as sensible details based on the certainties Cancer Gene Therapy Market. The market report on Cancer Gene Therapy provides a profound sector forecast in perspective of the provincial evaluation via detailed assessment within the time span. It, in addition, passes on expansive information of the marketplace key players, sub traders, merchants, along with other supplementary sources.

It gives a forward-looking factor of view on diverse elements using or restricting enterprise quarter development. It offers a forecast based on how the marketplace is predicted to increase. Their key financials, widespread business enterprise assessment, weighted SWOT examination, key improvements, developments, land unfold, and methods are contemplated and were skillfully made on this a long way reaching Cancer Gene Therapy market file.

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The Major Market Players Dominating this Cancer Gene Therapy Market for its Products, Services, and Continuous Product Developments are:

Vigene Biosciences, Sirion Biotech, Bluebird bio, Cellectis, Ziopharm, Cobra, Uniqure, Finvector, Sarepta Therapeutics

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The Cancer Gene Therapy Market can be customized to the country level or any other market segment. Besides this, Report understands that you may have your own business need, hence we also provide fully customized solutions to clients.

The Cancer Gene Therapy market record covers a top to the bottom focused scene in which key giants are profiled. Exclusive manufacturers engaged with the Cancer Gene Therapy market are surveyed. The research report for the Cancer Gene Therapy market incorporates a global point of view. This report investigates Cancer Gene Therapy market size and trends primarily based on its marketplace fragments, chief geologies, and modern marketplace patterns. This may bolster the consumer in settling on knowledgeable choices and primary key systems.

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The report hands in-depth segmentation of the worldwide market based on supported technology, product type, application, and numerous processes and systems. The report attains economical competitive analysis, business trends within the market, and an alternative key characteristic of the worldwide Cancer Gene Therapy market. Our experts have genuinely concatenated the Cancer Gene Therapy market share report by alluding the lists and figures, primary sources, to boost the understanding of the associated procedural terms and conditions.

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The Cancer Gene Therapy market size report includes the leading advancements and technological up-gradation that engages the user to inhabit with fine business selections, define their future-based priority growth plans, and to implement the necessary actions. The global Cancer Gene Therapy market report also offers a detailed summary of key players and their manufacturing procedure with statistical data and profound analysis of the products, contribution, and revenue.

Exclusive manufacturers engaged with the Cancer Gene Therapy market share are surveyed. Their key financials, preferred enterprise assessment, weighted SWOT exam, key advancements, traits, land unfold, and approaches are pondered and had been skillfully made on this far-reaching Cancer Gene Therapy market document. The Cancer Gene Therapy market record covers a top to the bottom focused scene in which key giants are profiled. Exclusive manufacturers engaged with the Cancer Gene Therapy are surveyed. The research report for the Cancer Gene Therapy market incorporates a global point of view. This report investigates Cancer Gene Therapy market trends primarily based on its marketplace fragments, chief geologies, and modern marketplace patterns. This may bolster the consumer in settling on knowledgeable choices and primary key systems.

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Regions and Countries Level Analysis

Regional analysis is another highly comprehensive part of the research and analysis study of the Cancer Gene Therapy market presented in the report. This section sheds light on the sales growth of different regional and country-level Cancer Gene Therapy markets. For the historical and forecast period 2015 to 2025, it provides detailed and accurate country-wise volume analysis and region-wise market size analysis of the Cancer Gene Therapy market.

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Cancer Gene Therapy Market Size, Share, Growth Drivers Analysis in a new research report Forecast 2020-2026 - Northwest Trail

Shandong Huge

Global Dental Consumables And Dental Instruments Market: Competitive Landscape

This section of the report lists various major manufacturers in the market. The competitive analysis helps the reader understand the strategies and collaborations that players focus on in order to survive in the market. The reader can identify the players fingerprints by knowing the companys total sales, the companys total price, and its production by company over the 2020-2026 forecast period.

Global Dental Consumables And Dental Instruments Market: Regional Analysis

The report provides a thorough assessment of the growth and other aspects of the Dental Consumables And Dental Instruments market in key regions, including the United States, Canada, Italy, Russia, China, Japan, Germany, and the United Kingdom United Kingdom, South Korea, France, Taiwan, Southeast Asia, Mexico, India and Brazil, etc. The main regions covered by the report are North America, Europe, the Asia-Pacific region and Latin America.

The Dental Consumables And Dental Instruments market report was prepared after various factors determining regional growth, such as the economic, environmental, technological, social and political status of the region concerned, were observed and examined. The analysts examined sales, production, and manufacturer data for each region. This section analyzes sales and volume by region for the forecast period from 2020 to 2026. These analyzes help the reader understand the potential value of investments in a particular country / region.

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The report provides an in-depth analysis of the size of the Dental Consumables And Dental Instruments world market, as well as recent trends and future estimates, in order to clarify the upcoming investment pockets.

The report provides data on key growth drivers, constraints and opportunities, as well as their impact assessment on the size of the Dental Consumables And Dental Instruments market.

Porters 5 Strength Rating shows how effective buyers and suppliers are in the industry.

The quantitative analysis of the Dental Consumables And Dental Instruments world industry from 2020 to 2026 is provided to determine the potential of the Dental Consumables And Dental Instruments market.

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Who are the main global players in this Dental Consumables And Dental Instruments market? What is the profile of your company, its product information, its contact details?

What was the status of the global market? What was the capacity, the production value, the cost and the profit of the market?

What are the forecasts of the global industry taking into account the capacity, the production and the value of production? How high is the cost and profit estimate? What will be the market share, supply, and consumption? What about imports and export?

What is market chain analysis by upstream raw materials and downstream industry?

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Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage and more. These reports deliver an in-depth study of the market with industry analysis, market value for regions and countries and trends that are pertinent to the industry.

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Tags: Dental Consumables And Dental Instruments Market Size, Dental Consumables And Dental Instruments Market Trends, Dental Consumables And Dental Instruments Market Growth, Dental Consumables And Dental Instruments Market Forecast, Dental Consumables And Dental Instruments Market Analysis

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Dental Consumables And Dental Instruments Market Size by Top Key Players, Growth Opportunities, Incremental Revenue , Outlook and Forecasts to 2026 -...

Global CNS Gene Therapy Market Analysis

Persistence Market Research, in a recently published market study, offers valuable insights related to the overall dynamics of the CNS Gene Therapy market in the current scenario. Further, the report assesses the future prospects of the CNS Gene Therapy by analyzing the various market elements including the current trends, opportunities, restraints, and market drivers. The COVID-19 analysis section within the report offers timely insights regarding the impact of the global pandemic on the market. The presented study also offers data regarding the business and supply chain continuity strategies that are likely to assist stakeholders in the long-run.

As per the report, the CNS Gene Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and exceed a value of ~US$ XX by the end of 2029. Some of the leading factors that are expected to drive the growth of the market include, focus towards research and development, innovations, and evolving consumer preferences among others.

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Regional Outlook

The report scrutinizes the prospects of the CNS Gene Therapy market in different geographical regions. The scope of innovation, consumer behavior, and regulatory framework of each region is thoroughly analyzed in the presented study.

Distribution-Supply Channel Assessment

The report provides a thorough analysis of the different distribution channels adopted by market players in the global CNS Gene Therapy market along with the market attractiveness analysis of each distribution channel. The impact of the COVID-19 pandemic on the different distribution channels is enclosed in the report.

Product Adoption Analysis

key players and product offerings

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The report aims to address the following pressing questions related to the CNS Gene Therapy market:

Key Takeaways from the CNS Gene Therapy Market Report

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CNS Gene Therapy Market to Slip Due to Delays in Production Amidst Coronavirus Outbreak Cole Reports - Cole of Duty

Analysis of the Global Hemophilia Gene Therapy Market

The report on the global Hemophilia Gene Therapy market reveals that the market is expected to grow at a CAGR of ~XX% during the considered forecast period (2019-2029) and estimated to reach a value of ~US$XX by the end of 2029. The latest report is a valuable tool for stakeholders, established market players, emerging players, and other entities to devise effective strategies to combat the impact of COVID-19

Further, by leveraging the insights enclosed in the report, market players can devise concise, impactful, and highly effective growth strategies to solidify their position in the Hemophilia Gene Therapy market.

Research on the Hemophilia Gene Therapy Market Addresses the Following Queries

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Competitive Landscape

The competitive landscape section offers valuable insights related to the business prospects of leading market players operating in the Hemophilia Gene Therapy market. The market share, product portfolio, pricing strategy, and growth strategies adopted by each market player is included in the report. The major steps taken by key players to address the business challenges put forward by the novel COVID-19 pandemic is discussed in the report.

Regional Landscape

The regional landscape section provides a deep understanding of the regulatory framework, current market trends, opportunities, and challenges faced by market players in each regional market. The various regions covered in the report include:

End-User Assessment

The report bifurcates the Hemophilia Gene Therapy market based on different end users. The supply-demand ratio and consumption volume of each end-user is accurately depicted in the report.

Competition AnalysisIn the competitive analysis section of the report, leading as well as prominent players of the global Hemophilia Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.On the whole, the report proves to be an effective tool that players can use to gain a competitive edge over their competitors and ensure lasting success in the global Hemophilia Gene Therapy market. All of the findings, data, and information provided in the report are validated and revalidated with the help of trustworthy sources. The analysts who have authored the report took a unique and industry-best research and analysis approach for an in-depth study of the global Hemophilia Gene Therapy market.The following players are covered in this report:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therapy Breakdown Data by TypeHemophilia AHemophilia BHemophilia Gene Therapy Breakdown Data by ApplicationHemophilia A Gene TherapyHemophilia B Gene Therapy

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Essential Findings of the Hemophilia Gene Therapy Market Report:

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Assessing the Fallout From the Coronavirus Pandemic Hemophilia Gene Therapy to Discern Steadfast Expansion During 2019-2053 - amitnetserver

CAMBRIDGE, Mass., May 1, 2020 /PRNewswire/ --Obsidian Therapeutics, a biotechnology company pioneering controllable cell and gene therapies, today announced it will share the architecture and components of its Safe Workplace Function Tool (SWFT) Productivity Solution. The SWFT Solution was designed in response to the COVID19 pandemic to support a safe work environment while maintaining productivity in the lab.

The SWFT Solution is a web-based application built and integrated into Microsoft 365 that allows scientists to view and schedule lab-based activities including by date, time and lab location. SWFT promotes collaboration and coordination between teams by predicting scheduling and occupancy conflicts, which allows team members to adjust their schedules to promote social distancing in the lab and office.

"The SWFT Solution has enabled Obsidian to continue to generate critical data across our cytoDRiVE development programs, while maximizing the safety of our staff," stated Catherine Stehman-Breen, M.D., Chief Research and Development Officer at Obsidian. "We have already shared this technology with large pharmaceutical and small biotechnology companies who are interested in our SWFT Solution to get their labs back up and running. We believe that it is more important than ever before to leverage one another's expertise in order to overcome challenges as we work tirelessly to deliver meaningful outcomes to patients in need."

To create Obsidian's bespoke application, (1) a capacity analysis was conducted, taking into consideration lab space and occupancy levels, and (2) in collaboration with scientists, workflow recommendations were implemented to determine an appropriate shift schedule. These steps facilitated the development of a solution that enabled scientists to plan their experiments with specific lab locations and shifts, as well as be alerted to and prevent any over-capacity issues. Obsidian implemented four three-hour lab blocks, with thirty-minutes of cleaning time between blocks, over a seven-day work week, to ensure that coronavirus-related safety recommendations were met.

Celeste Richardson, Ph.D., Vice President of Cell Therapy of Obsidian, stated, "We have a commitment to our employees to keep the health and safety of our employees top of mind while they work to bring innovative therapies to patients. The development of the SWFT Solution perfectly demonstrates Obsidian's culture of teamwork, determination and innovation."

Obsidian's IT Partner, TRNDigital, is continuing to iterate the tool to ensure it continues to meet scientists' needs and can be made available to others. The solution has been expanded to include density planning in the Obsidian offices. In addition, the SWFT Solution is scalable and flexible to other laboratory setups.

The SWFT solution was developed in-house by Henry Rogalin, Data Scientist, under the leadership of Nic Betts, Head of IT and Facilities, and in collaboration with a safety and facilities capacity team led by Jillian Giguere, Senior Manager of Laboratory Operations, Facilities, and EHS. For more information on SWFT, submit this inquiry form. Informational sessions and training will be hosted as requested.

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:Maggie BellerRusso Partners, LLCMaggie.beller@russopartnersllc.com646-942-5631

View original content to download multimedia:http://www.prnewswire.com/news-releases/obsidian-therapeutics-develops-and-shares-safe-workplace-productivity-solution-in-response-to-covid19-301050820.html

SOURCE Obsidian Therapeutics

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Obsidian Therapeutics Develops and Shares Safe Workplace Productivity Solution in Response to COVID19 - P&T Community

Sarepta Therapeutics, Inc.s (NASDAQ:SRPT): Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities approaches for the treatment of rare diseases. The US$9.2b market-cap company announced a latest loss of -US$715.1m on 31 December 2019 for its most recent financial year result. As path to profitability is the topic on SRPTs investors mind, Ive decided to gauge market sentiment. In this article, I will touch on the expectations for SRPTs growth and when analysts expect the company to become profitable.

Check out our latest analysis for Sarepta Therapeutics

According to the 22 industry analysts covering SRPT, the consensus is breakeven is near. They expect the company to post a final loss in 2021, before turning a profit of US$716m in 2022. Therefore, SRPT is expected to breakeven roughly 2 years from today. How fast will SRPT have to grow each year in order to reach the breakeven point by 2022? Working backwards from analyst estimates, it turns out that they expect the company to grow 59% year-on-year, on average, which is rather optimistic! If this rate turns out to be too aggressive, SRPT may become profitable much later than analysts predict.

Im not going to go through company-specific developments for SRPT given that this is a high-level summary, but, take into account that by and large biotechs, depending on the stage of product development, have irregular periods of cash flow. So, a high growth rate is not out of the ordinary, particularly when a company is in a period of investment.

One thing I would like to bring into light with SRPT is its relatively high level of debt. Generally, the rule of thumb is debt shouldnt exceed 40% of your equity, which in SRPTs case is 83%. Note that a higher debt obligation increases the risk in investing in the loss-making company.

There are key fundamentals of SRPT which are not covered in this article, but I must stress again that this is merely a basic overview. For a more comprehensive look at SRPT, take a look at SRPTs company page on Simply Wall St. Ive also compiled a list of key factors you should look at:

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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When Will Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Become Profitable? - Simply Wall St

DetailsCategory: AntibodiesPublished on Friday, 01 May 2020 15:04Hits: 468

Recommendation for approval based on results from pivotal Phase 3 MEDALIST and BELIEVE studies

PRINCETON, NJ & CAMBRIDGE, MA, USA I April 30, 2020 IBristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion, recommending the approval of Reblozyl (luspatercept) for the treatment of:

This CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Reblozyl would be the first erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients. The safety and efficacy results provided in the application are from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

"Patients with myelodysplastic syndromes who experience anemia have limited treatment options, and some have been shown to not respond to available erythropoietin-based therapies," said Uwe Platzbecker, M.D., Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital and lead investigator of the MEDALIST study. If approved, the introduction of a new class of therapy in Reblozyl could provide a promising option to help relieve patients from the burden of regular transfusions to manage their disease.

Todays positive CHMP opinion of Reblozyl is an important milestone for adult beta thalassemia patients in the EU who have limited treatment options to address anemia, a serious consequence of the disease, said Maria Domenica Cappellini, M.D., Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda and lead investigator of the BELIEVE study. Reblozyl has the potential to significantly decrease the number of red blood cell transfusions patients need.

This decision by the CHMP is an important step towards making this first-in-class therapy an option for eligible patients with anemia due to beta thalassemia or myelodysplastic syndromes, said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. We, and our partners at Acceleron, look forward to the opportunity to make this treatment option available in the EU and are extremely appreciative of the patients, families and individuals who continue to help us progress important research in a range of serious diseases.

About MEDALIST

MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) myelodysplastic syndromes (MDS). All patients were red blood cell (RBC) transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA nave and unlikely to respond due to endogenous serum erythropoietin levels of 200 U/L, and had no prior treatment with disease modifying agents. Results of the MEDALIST trial were first presented during the Plenary Session of the 2018 American Society of Hematology (ASH) Annual Meeting and were selected for the Best of ASH. The New England Journal of Medicine published the MEDALIST trial results in January 2020.

About MDS

MDS are a group of closely related blood cancers characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections. People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of iron overload, transfusion reactions and infections. There are approximately 50,000 patients with MDS in the EU5 countries.

About BELIEVE

BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing luspatercept plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia. Results of the BELIEVE trial were first presented at the 2018 ASH Annual Meeting and selected for the Best of ASH. The New England Journal of Medicine published the BELIEVE trial results in March 2020.

About Beta Thalassemia

Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemia a condition that can be debilitating and can lead to more severe complications for patients as well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

About Reblozyl

Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

Please see full Prescribing Information for REBLOZYL

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol-Myers Squibb Company and Juno Therapeutics, a Bristol-Myers Squibb Company.

About Acceleron

Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States and are developing luspatercept for the treatment of chronic anemia in myelofibrosis. Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial and actively enrolling patients in the Phase 2 SPECTRA trial.

For more information, please visit http://www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

SOURCE: Bristol-Myers Squibb

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Reblozyl (luspatercept) Receives Positive CHMP Opinion for the Treatment of Adults with Anemia in Beta Thalassemia and Myelodysplastic Syndromes |...

In the first article we looked at face masks (when do you need to where one and why?); in the second we looked at disinfection (which disinfectants work best?); and the third topic was social distancing (why is 2 metres considered to be a safe distance?) The subject matter for the fourth article was the lockdown and whether the idea of an 'immunity passport' is a good idea (which is probably isn't). For the firth dive into COVID-19 subjects, we look at where we are with vaccine development, what the most promising antivirals are, and what do the different tests for coronavirus do and how do they vary?In this article, the topic is testing, vaccines and other efforts to help to combat the disease.

A laboratory technician works on coronavirus samples at "Fire Eye" laboratory in Wuhan

STR, STR, AFP

A fluorescent antibody (DFA) stain reveals the presence of rabies viral antigen in a tissue sample harvested from a mandibular salivary gland, suspected of being infected with rabies.

CDC/PHIL

A nurse wearing blue gloves administers a vaccine into a male patient's arm.

Rhoda Baer (National Institutes of Health)

Images of COVID-19 released by US science agencies

John SAEKI, AFP

France has now detected a total of 11 cases of the novel coronavirus

JEFF PACHOUD, AFP/File

This opinion article was written by an independent writer. The opinions and views expressed herein are those of the author and are not necessarily intended to reflect those of DigitalJournal.com

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Op-Ed: Where are we with the coronavirus vaccine? - Digital Journal

Complete study of the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

Key companies operating in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market include , BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

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Segmental Analysis

The report has classified the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry.

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segment By Type:

, BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segment By Application:

, BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key companies operating in the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market include , BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Brammer Bio, VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences

Key questions answered in the report:

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TOC

1 Market Overview of Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview1.1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Product Scope1.1.2 Market Status and Outlook1.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size Overview by Region 2015 VS 2020 VS 20261.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Region (2015-2026)1.4 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Region (2015-2020)1.5 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size Forecast by Region (2021-2026)1.6 Key Regions Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.1 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.2 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.3 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.4 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.5 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.6.6 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size YoY Growth (2015-2026)1.7 Coronavirus Disease 2019 (Covid-19): Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Industry Impact1.7.1 How the Covid-19 is Affecting the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Industry1.7.1.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Business Impact Assessment Covid-191.7.1.2 Supply Chain Challenges1.7.1.3 COVID-19s Impact On Crude Oil and Refined Products1.7.2 Market Trends and Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Potential Opportunities in the COVID-19 Landscape1.7.3 Measures / Proposal against Covid-191.7.3.1 Government Measures to Combat Covid-19 Impact1.7.3.2 Proposal for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Players to Combat Covid-19 Impact 2 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview by Type2.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Type: 2015 VS 2020 VS 20262.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Type (2015-2020)2.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Type (2021-2026)2.4 AAV2.5 Adenoviral2.6 Lentiviral2.7 Retroviral2.8 Plasmid DNA2.9 Other Vectors 3 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview by Type3.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application: 2015 VS 2020 VS 20263.2 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Historic Market Size by Application (2015-2020)3.3 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Forecasted Market Size by Application (2021-2026)3.4 Cancers3.5 Inherited Disorders3.6 Viral Infections3.7 Others 4 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Competition Analysis by Players4.1 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size (Million US$) by Players (2015-2020)4.2 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing as of 2019)4.3 Date of Key Manufacturers Enter into Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market4.4 Global Top Players Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Headquarters and Area Served4.5 Key Players Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Product Solution and Service4.6 Competitive Status4.6.1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Concentration Rate4.6.2 Mergers & Acquisitions, Expansion Plans 5 Company (Top Players) Profiles and Key Data5.1 BioReliance5.1.1 BioReliance Profile5.1.2 BioReliance Main Business and Companys Total Revenue5.1.3 BioReliance Products, Services and Solutions5.1.4 BioReliance Revenue (US$ Million) (2015-2020)5.1.5 BioReliance Recent Developments5.2 Cobra Biologics5.2.1 Cobra Biologics Profile5.2.2 Cobra Biologics Main Business and Companys Total Revenue5.2.3 Cobra Biologics Products, Services and Solutions5.2.4 Cobra Biologics Revenue (US$ Million) (2015-2020)5.2.5 Cobra Biologics Recent Developments5.3 Oxford BioMedica5.5.1 Oxford BioMedica Profile5.3.2 Oxford BioMedica Main Business and Companys Total Revenue5.3.3 Oxford BioMedica Products, Services and Solutions5.3.4 Oxford BioMedica Revenue (US$ Million) (2015-2020)5.3.5 UniQure Recent Developments5.4 UniQure5.4.1 UniQure Profile5.4.2 UniQure Main Business and Companys Total Revenue5.4.3 UniQure Products, Services and Solutions5.4.4 UniQure Revenue (US$ Million) (2015-2020)5.4.5 UniQure Recent Developments5.5 FinVector5.5.1 FinVector Profile5.5.2 FinVector Main Business and Companys Total Revenue5.5.3 FinVector Products, Services and Solutions5.5.4 FinVector Revenue (US$ Million) (2015-2020)5.5.5 FinVector Recent Developments5.6 MolMed5.6.1 MolMed Profile5.6.2 MolMed Main Business and Companys Total Revenue5.6.3 MolMed Products, Services and Solutions5.6.4 MolMed Revenue (US$ Million) (2015-2020)5.6.5 MolMed Recent Developments5.7 MassBiologics5.7.1 MassBiologics Profile5.7.2 MassBiologics Main Business and Companys Total Revenue5.7.3 MassBiologics Products, Services and Solutions5.7.4 MassBiologics Revenue (US$ Million) (2015-2020)5.7.5 MassBiologics Recent Developments5.8 Richter-Helm5.8.1 Richter-Helm Profile5.8.2 Richter-Helm Main Business and Companys Total Revenue5.8.3 Richter-Helm Products, Services and Solutions5.8.4 Richter-Helm Revenue (US$ Million) (2015-2020)5.8.5 Richter-Helm Recent Developments5.9 FUJIFILM Diosynth Biotechnologies5.9.1 FUJIFILM Diosynth Biotechnologies Profile5.9.2 FUJIFILM Diosynth Biotechnologies Main Business and Companys Total Revenue5.9.3 FUJIFILM Diosynth Biotechnologies Products, Services and Solutions5.9.4 FUJIFILM Diosynth Biotechnologies Revenue (US$ Million) (2015-2020)5.9.5 FUJIFILM Diosynth Biotechnologies Recent Developments5.10 Lonza5.10.1 Lonza Profile5.10.2 Lonza Main Business and Companys Total Revenue5.10.3 Lonza Products, Services and Solutions5.10.4 Lonza Revenue (US$ Million) (2015-2020)5.10.5 Lonza Recent Developments5.11 Aldevron5.11.1 Aldevron Profile5.11.2 Aldevron Main Business and Companys Total Revenue5.11.3 Aldevron Products, Services and Solutions5.11.4 Aldevron Revenue (US$ Million) (2015-2020)5.11.5 Aldevron Recent Developments5.12 Eurogentec5.12.1 Eurogentec Profile5.12.2 Eurogentec Main Business and Companys Total Revenue5.12.3 Eurogentec Products, Services and Solutions5.12.4 Eurogentec Revenue (US$ Million) (2015-2020)5.12.5 Eurogentec Recent Developments5.13 Cell and Gene Therapy Catapult5.13.1 Cell and Gene Therapy Catapult Profile5.13.2 Cell and Gene Therapy Catapult Main Business and Companys Total Revenue5.13.3 Cell and Gene Therapy Catapult Products, Services and Solutions5.13.4 Cell and Gene Therapy Catapult Revenue (US$ Million) (2015-2020)5.13.5 Cell and Gene Therapy Catapult Recent Developments5.14 Biovian5.14.1 Biovian Profile5.14.2 Biovian Main Business and Companys Total Revenue5.14.3 Biovian Products, Services and Solutions5.14.4 Biovian Revenue (US$ Million) (2015-2020)5.14.5 Biovian Recent Developments5.15 Brammer Bio5.15.1 Brammer Bio Profile5.15.2 Brammer Bio Main Business and Companys Total Revenue5.15.3 Brammer Bio Products, Services and Solutions5.15.4 Brammer Bio Revenue (US$ Million) (2015-2020)5.15.5 Brammer Bio Recent Developments5.16 VGXI5.16.1 VGXI Profile5.16.2 VGXI Main Business and Companys Total Revenue5.16.3 VGXI Products, Services and Solutions5.16.4 VGXI Revenue (US$ Million) (2015-2020)5.16.5 VGXI Recent Developments5.17 PlasmidFactory5.17.1 PlasmidFactory Profile5.17.2 PlasmidFactory Main Business and Companys Total Revenue5.17.3 PlasmidFactory Products, Services and Solutions5.17.4 PlasmidFactory Revenue (US$ Million) (2015-2020)5.17.5 PlasmidFactory Recent Developments5.18 bluebird bio5.18.1 bluebird bio Profile5.18.2 bluebird bio Main Business and Companys Total Revenue5.18.3 bluebird bio Products, Services and Solutions5.18.4 bluebird bio Revenue (US$ Million) (2015-2020)5.18.5 bluebird bio Recent Developments5.19 Novasep5.19.1 Novasep Profile5.19.2 Novasep Main Business and Companys Total Revenue5.19.3 Novasep Products, Services and Solutions5.19.4 Novasep Revenue (US$ Million) (2015-2020)5.19.5 Novasep Recent Developments5.20 Spark Therapeutics5.20.1 Spark Therapeutics Profile5.20.2 Spark Therapeutics Main Business and Companys Total Revenue5.20.3 Spark Therapeutics Products, Services and Solutions5.20.4 Spark Therapeutics Revenue (US$ Million) (2015-2020)5.20.5 Spark Therapeutics Recent Developments5.21 Vigene Biosciences5.21.1 Vigene Biosciences Profile5.21.2 Vigene Biosciences Main Business and Companys Total Revenue5.21.3 Vigene Biosciences Products, Services and Solutions5.21.4 Vigene Biosciences Revenue (US$ Million) (2015-2020)5.21.5 Vigene Biosciences Recent Developments 6 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application6.1 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)6.2 North America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 7 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application7.1 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)7.2 Europe Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 8 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application8.1 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)8.2 China Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 9 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application9.1 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)9.2 Rest of Asia Pacific Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 10 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application10.1 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)10.2 Latin America Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 11 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing by Players and by Application11.1 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size and Market Share by Players (2015-2020)11.2 Middle East & Africa Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Size by Application (2015-2020) 12 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Dynamics12.1 Industry Trends12.2 Market Drivers12.3 Market Challenges12.4 Porters Five Forces Analysis 13 Research Finding /Conclusion 14 Methodology and Data Source 14.1 Methodology/Research Approach14.1.1 Research Programs/Design14.1.2 Market Size Estimation14.1.3 Market Breakdown and Data Triangulation14.2 Data Source14.2.1 Secondary Sources14.2.2 Primary Sources14.3 Disclaimer14.4 Author List

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Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market 2020: Analysis By Regional Outlook, Competitive Landscape, Strategies And...

CHELMSFORD May 1, 2020 (Thomson StreetEvents) -- Edited Transcript of Brooks Automation Inc earnings conference call or presentation Thursday, April 30, 2020 at 8:30:00pm GMT

* Lindon G. Robertson

Brooks Automation, Inc. - Executive VP & CFO

Brooks Automation, Inc. - Director of IR

* Stephen S. Schwartz

* J. Ho

Stifel, Nicolaus & Company, Incorporated, Research Division - MD of Technology Sector

* Jacob K. Johnson

Crdit Suisse AG, Research Division - MD, Global Technology Strategist and Global Technology Sector Head

Janney Montgomery Scott LLC, Research Division - MD, Head of Healthcare Research & Senior Equity Research Analyst

Greetings, and welcome to the Brooks Automation Q2 2020 Financial Results. (Operator Instructions) As a reminder, this conference is being recorded Thursday, April 30, 2020. I would now like to turn the conference over to Mark Namaroff. Please go ahead.

Mark Namaroff, Brooks Automation, Inc. - Director of IR [2]

Thank you, Malaika. Good afternoon everyone on the line today. We hope everyone is staying healthy in this environment. We'd like to welcome you to our earnings conference call for the second quarter of fiscal 2020. Our Q2 earnings press release was issued after the close of the market today and it is available on our Investor Relations website located at brooks.investorroom.com, as are the supplementary PowerPoint slides that we'll be using during the prepared remarks.

I would like to remind everyone that during the course of the call today, we will be making a number of forward-looking statements within the meaning of the Private Litigation and Securities Act of 1995. There are many factors that may cause actual financial results or other events to differ from those identified in such forward-looking statements. I would refer you to the section of our earnings release titled Safe Harbor Statement, the safe harbor slide on our aforementioned PowerPoint presentation on our website, and our various filings with the SEC, including our annual reports on Form 10-K and our quarterly reports on Form 10-Q. We make no obligation to update these statements should future financial data or events occur that differ from the forward-looking statements presented today.

We would also refer to a number of non-GAAP financial measures, which are used in addition to and in conjunction with results presented in accordance with GAAP. We believe non-GAAP measures provide an additional way of viewing aspects of our operations and performance. But when considered with GAAP financial results, the reconciliation of GAAP measures, and a reconciliation of GAAP measures, they provide an even more complete understanding of the Brooks business. Non-GAAP measures should not be relied upon to the exclusion of GAAP measures themselves.

On the call with me today is our President and Chief Executive Officer, Steve Schwartz, and Executive Vice President and Chief Financial Officer, Lindon Robertson. We will open the call up with remarks from Steve on the highlights of the second quarter, and then Lindon will provide a more detailed look into our financial results for the quarter and our outlook for the third fiscal quarter of 2020. We will then have time to take your questions at the end of our prepared remarks.

And with that, now I'd like to turn the call over to our CEO, Steve Schwartz.

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Stephen S. Schwartz, Brooks Automation, Inc. - CEO, President, Interim GM of Life Sciences & Director [3]

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Thank you, Mark, and good afternoon, everyone. We're glad to have you with us today. As we've already published preliminary results in a press release we issued on April 13th, I'll recap those results briefly and focus my remarks on some extra color at the segment level to give you some indication about how we see the current and near term business environment.

I'd like to start by discussing our priorities during this pandemic. First is the safety and wellbeing of our employees and their families as well as our partners including our suppliers and customers. Second, our ability to serve our customers that are deemed essential businesses with specific focus on any and all COVID-19 requests. And there have been many. And finally, the ongoing health of the company for the long term and actions we're taking to ensure that we emerge from this crisis stronger than ever.

With the exceptions of only a few brief work interruptions, all of our 20 plus factories, laboratories and repair centers were up and running throughout Q2 and all are currently operating. Of our approximately 3,300 global employees, about 1,800 have jobs that require them to be onsite. The rest of our employees are working from home. We are particularly proud of the way that each and every employee has embraced the new work practices that include social distancing, the diligent use of PPE, temperature screenings, staggered shifts and other practices which have allowed us to continue serving customers. All have adapted to this new work environment and we could not be more pleased with the resiliency of our operations during these turbulent times.

We consistently met our delivery commitments to customers as they struggled with their supply chains. And in many instances on the Life Sciences side, we stepped up to provide GENEWIZ, Sample Management and Informatic Services to customers who had either lost their regular services providers or who needed new services from us that came about because of the COVID-19 crisis. It's been energizing to bring even more capability to our customers and their need for our services underscores our purpose and mission.

Finally, we're building this company for the long term and our quarter to quarter and year to year revenue and earnings growth speaks to the momentum that we've generated. We've amassed an incredible technology and scientific portfolio, a global presence that allows us to deliver our world class capability to customers around the world, and we possess market positions in trusted brands that are the envy of our competitors. We've accomplished this through the energy and commitment of our global workforce. They know just how to ensure that we deliver on our long-term goals.

There is so much opportunity in front of us and it would be shortsighted of us to slow down now, especially since the capability we're building will be even more valuable in the future. For that reason, we intend to retain our team and fund their activities for technology development, new discovery, market capture, and customer support.

With each day that goes by, we're taking actions and making investments to emerge from the pandemic even stronger and more necessary to our customers. Our decision to continue these investments will have some impact on our earnings performance through the rest of this year, but we believe that it's absolutely the right action to take.

As companies and countries are contemplating how to reemerge from the pandemic, we have a definitive plan in place and a dedicated team that will continue to drive the momentum that we've established.

Now I'll report on Q2. Second quarter performance was strong with revenue of $220 million, up 11% year-over-year with the same 11% growth for each of our Semiconductor and Life Sciences segments. As I mentioned, for the most part our factories and laboratories were running throughout the quarter as we worked to meet customer demand, albeit it with many adjustments to mix because of both supply and demand changes throughout the quarter.

I'll begin with Life Sciences which delivered strong results with $95 million in revenue, up 4% sequentially with growth coming from both GENEWIZ and Sample Management. As each of these subsegments witnessed its own dynamics, I'll cover them separately starting with GENEWIZ.

Once again, GENEWIZ outperformed even our expectations, delivering a 25% year-over-year increase in revenue to $41 million. We were advantaged by our unique capability as both readers and writers of genes which allowed us to overcome some of the curveballs thrown our way. First, in early February when we announced our Q1 results, we told you that except for a team of scientists who remained onsite in support of urgent COVID-19 research, our Suzhou, China operations were basically shut down. It's in our Suzhou site where we perform the vast majority of our gene synthesis. Although we were bracing for what could have been a prolonged slowdown, China operations came back much faster than we'd imagined. By the end of February, more than 90% of our employees were back at work. And even without recovery in academia in China, demand from global customers was strong. In fact, despite the speedbump in February, the March quarter was a record for synthesis at just over $10 million.

Similarly, in next generation sequencing, we also had a record quarter with year-over-year growth of 46% and consistent 7% sequential growth, but with more volatility as the quarter progressed. We saw a normal pattern for NGS demand in the first two months of the quarter and then something of a spike in orders in the first half of March followed by two weeks of lower order volume. As we assess the NGS and synthesis businesses, we believe that much of the Q2 increase was the result of our normal customer capture. At the same time, it appears that some of the March burst came from researchers who no longer had capacity from their core laboratories or were facing reduced productivity from their existing suppliers while GENEWIZ remained at full capability and was eager to serve.

That said, order patterns for April have been steady but slightly lower in both synthesis and NGS but we have mixed signals as customer engagement for both synthesis and NGS projects remains quite active.

In Sanger sequencing, which is a high volume, overnight turns business where we perform millions of measurements for thousands of customers each quarter, revenue was steady for the first 10 weeks of the quarter. But when shelter in place orders closed many academic labs and caused a shutdown of nonessential research, we saw an abrupt reduction of more than 50% of our average daily volumes for the last two weeks of the quarter. And that level has persisted throughout most of April.

Though total Sanger revenue actually decreased slightly quarter-over-quarter, it was still up low single digits from Q2, one year ago. In Q3, we anticipate lower Sanger revenue until academic labs come back online and industry reaccelerates. And although we've seen average daily volumes increase during each of the past 4 weeks, we still remain below 50% of average. So at this point in the quarter, it would be too early to say that we have any concrete signs of a meaningful return to work.

In terms of overall GENEWIZ outlook, we expect business to be slightly down until academic researchers return to their benches and the activities that support clinical trials are back on the upswing. We don't have a prediction for when that will be, but we modeled a gradual return toward more normal volumes as we make our way through the rest of this quarter.

A difficult environment like the one we're in provides opportunities for companies that are prepared. In Q2, customers challenged us with hundreds of COVID-19 projects that gave us opportunities to demonstrate our incredible scientific acumen, round-the-clock collaboration, and fast turn, high quality results in support of their essential research. In addition, we took on many new urgent projects for first time customers. We believe that over the long run, it's this attention to solving customer issues that cement them to us and enabled GENEWIZ to add more than 200 new customers in the quarter.

Now I'll turn to Sample Management which even with all the disruption from coronavirus came in right on forecast I remind you that this year in Sample Management we have two areas of focus. A return to double digit revenue growth and sustainable gross margin improvement. And we're pleased with our progress against these goals. Even in this environment, Q2 was a very solid quarter. Revenue came in at $54 million, up 5% from Q1 and 3% year-over-year. Moreover, gross margin was up another 290 basis points from Q1. We're very encouraged by these results and it gives us confidence that this business is solidly on a path to be able to deliver on our expectations.

At the time we announced Q1 results, we knew two things. That the changes we had made to the organization structure and our focus on performance were set to deliver more growth and keep us on our trajectory for reacceleration of revenue in 2020, and that we were on track to deliver more profitability improvements.

That said, we also mentioned to you that we could foresee approximately $2 million of potential headwind from the coronavirus because of the inability of our field teams to complete system startups and perform revenue generating service on our equipment. Unfortunately, our customers obviously did not open for our employees and we did incur the headwinds that we had built into our forecast.

That said, there were many accomplishments in the quarter and I do want to highlight a few as they relate to trends in the business. Our cryogenics cold-chain products continue to make strong progress. Revenue topped $3 million in the quarter and we shipped B3C Cryo systems to 9 different customers. Six of those were repeat buyers. To date, 3/4 of our systems have been sold into cell and gene therapy applications and the momentum for these automated solutions continues to build.

In spite of the significant slowdown in clinical trial activity in March, Q2 Bio Storage activity generated our largest sample intake quarter since 2017, and we're encouraged by the reinvigoration of our customer capture activities. And in our consumables and instruments business lines, we made fast turn capital addition to respond to increased volume of consumables that will be used for COVID-19 tests as part of packages that we sell to diagnostics companies. This added capacity should allow us to increase revenue in Q3.

All-in, it was a very good quarter for Sample Management. Even with some COVID-19 related delays, revenue growth was right on track, profitability was ahead of schedule, and the result of our deliberate action is something that we'll build upon. The team is energized and active and we plan to come out of the COVID-19 days stronger than ever. What energizes us even more is the amount and level of large customer, large deal activity that's underway. During these days of no travel, our customer engagement activity has moved to the internet and deals that we've been working on for months are still being advanced. We are currently negotiating the final Ts and Cs on two large contracts and we have more multimillion dollar contracts in the pipeline. There opportunities speak to our capabilities and confidence to resume growth and profitability in this business.

On the semiconductor side, our performance in Q2 demonstrates the value of our product portfolio, and because of our close working relationship with our supply chain, we experienced only a small COVID-19 related revenue impact in the quarter which caused no impact to our customers. At $125 million in Q2, we established a new record high for quarterly semiconductor revenue and that's all the more remarkable because as an industry, we are still not back to our highs for semiconductor capital equipment spending. We attribute this outperformance to two factors. The strong high market share position of our Contamination Control Solutions business which satisfies a rapidly growing technology need, and continued design win and market share capture in our equipment automation products for OEM process equipment along with the steadily evolving advanced packaging market.

I'll give some specific color from our major Semiconductor business drivers, tool automation, advanced packaging and Contamination Control Solutions. Our automation products remained strong in the quarter with systems up approximately 10% and robots similar to last quarter's results. We had record bookings for our vacuum robots and vacuum systems, indicating demand remains very strong.

Sales to Chinese equipment manufacturers is picking up and this is a good indicator for both China fab activity and advanced packaging capability. We saw an uptick in advanced packaging in the quarter to just over $13 million or up 30% from Q1. This is still lower than one year ago, but it's the first sequential uptick in the last three quarters and in general a positive indicator. Based on order activity, we do have some indication that advanced packaging opportunities may be staring to percolate, especially in China.

Finally, I'll give a brief update on Contamination Control business which has been extremely strong as we delivered a record $45 million in revenue. The $45 million number is extremely significant for several reasons. First, we truly tested our supply chain and determined it to be very capable. In the first 2 quarters of 2020, we shipped as much revenue as in the last 3 quarters of 2019. And those were already healthy business levels.

Second, we met the demand for the expansion of the 5 Nanometer Foundry ramp on schedule and with very high quality. And finally, we continue to win additional business across a broad range of customers, device technologies that will serve us well as tier one foundry spending subsides.

Revenue in CCS will necessarily be lower in Q3 after two very strong back to back quarters, but still we expect healthy levels of more than $30 million in revenue which will be largely made up of a broader base of customers across different geographies and technology applications.

All-in, we're prepared for another strong quarter in our Semiconductor business. That said, we're also aware of some COVID-19 related supply chain issues that are slowing delivery of some parts. So far, we've been able to navigate through most of these issues, but it remains to be seen what the impact might be on actual demand from our customers. There's currently a lot of speculation as to the outlook for semiconductor processing equipment in 2020. Already our March quarter Semiconductor revenue was up 11% from one year ago and our Q2 Semiconductor book to bill was 1.2 for the quarter on a record revenue quarter. Depending upon June quarter capability of the global supply chain, our backlog and customer demand expectations would allow us to deliver yet another record quarter in June if we do not see any meaningful interruptions.

All-in, we had a very solid second quarter. We adjusted well as changes hit us and all companies. And we know that we'll need to rely on these same adaptive skills in the June quarter which may be even more uncertain. But already, we're well positioned and we're extremely confident in the long-term opportunities that we're diligently winning with all that we're investing today.

That concludes my formal remarks, and I'll turn the call now over to Lindon.

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Lindon G. Robertson, Brooks Automation, Inc. - Executive VP & CFO [4]

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Thank you, Steve. I'd like to refer your attention to the slides on our website, starting with Slide 3. Since Steve hit on these messages in his remarks, I will also be brief on what we see as the headlines of our performance. First, we had excellent momentum that has only modestly slowed by COVID-19 disruptions.

We have performed remarkably well with double digit year-over-year revenue growth in both segments and have established ourselves truly as a top reliable supplier in every market we serve.

Next, we expanded earnings substantially with continued performance enhancements in the Life Science segment. To put a finer point on it, gross margins in the Sample Management business are up nearly 600 basis points from a year ago. And third, we are well equipped for what lies ahead. Our liquidity is strong with $198 million of net cash on the balance sheet and healthy cash generation. To this point, our year-to-date cash flow from operations when you exclude the taxes paid on the sale of the semi cryo business, are $51 million. This a year-to-date improvement of $29 million over the prior year. The business is healthy and both sides are contributing growth, profit and cash flow.

Let's move onto Slide 4 to review the overall P&L. Let me first point you to the GAAP earnings and highlight what is different in that comparison. The growth of revenue and operating income is similar to that shown in the Non-GAAP profile on the right side. But what is different on the left side is in the tax line. If the first fiscal quarter, as guided under GAAP, we recognized a windfall tax benefit of approximately $6 million which is related to long term incentive stock units divested in the first quarter. That allowed deduction resulted in a net tax benefit of $3 million for the first fiscal quarter that in the second quarter is back to normal with the $3 million tax expense. The $6 million swing offsets the positive operating performance picture in the GAAP Profile.

In the Non-GAAP results, we apply this windfall to the projected tax rate and take it across the quarters of the fiscal year so that you can use the financials for a level performance indicator.

Continuing with the Non-GAAP performance on the right side, you can see the performance supported 48% year-to-year growth in earnings per share. Starting at the top, the revenue growth came similarly from each business with each growing 11% year to year. We saw a nice uptick in gross margins of 60 basis points sequentially, but Semiconductor gross margins remained approximately level from Q1 as expected. And the uptick was driven by Life Sciences improvement. On a year-over-year basis, the dynamics are also similar with improvement in Life Sciences and a bit softer margins in semiconductor. But all-in, gross margins continue to make progress and contribute to the bottom line.

Operating expenses during the quarter were up. On a sequential basis, the increase was in R&D for our Semiconductor business. Our engineering team has been kept busy throughout the COVID-19 environment, committed to advancing the projects with customers. On a year-over-year basis, operating expense was $7 million higher, driven by both R&D and SG&A. In the SG&A line, the growth was driven primarily around investments in Life Sciences including our IT transition to a single cloud based ERP platform, GENEWIZ growth, and some additional structure from our recent acquisition of RURO Software.

While we are spending some extra to keep our team in a safe environment and onboard with us through the COVID crisis, we anticipate reductions in travel expenses and G&A which will result in lower operating expense by approximately $1 million to $2 million in the third quarter. You can see operating margin expansion. Combining the growth with the margin expansion, you can also see Non-GAAP net income grew 51% year-over-year and expanded 11% sequentially.

Moving to below the operating income line, net interest expense was $600,000, lower by about $7 million compared with last year when we were carrying debt associated with the GENEWIZ acquisition. The non-GAAP tax rate for the quarter came in at about 23%, very consistent with our expectation of 21% to 25% for the year.

Let's turn now over to Slide 5 to discuss the segment results, starting with Life Sciences. In the second quarter, Life Sciences revenue grew 11% to $95 million compared with the second quarter last year. On an organic basis, Life Sciences grew 11% as well. This quarter was the first full year-over-year comparison which includes a full quarter of GENEWIZ revenue since the acquisition of the business in November of 2018. GENEWIZ had a strong second quarter with a revenue of $41 million, growing 25% from last year despite the impacts of COVID-19 as Steve addressed. As a reminder, our GENEWIZ China operations were largely closed down and observed strict quarantine restrictions from that Chinese New Year through February 10th. The exception to that were the labs from which GENEWIZ provided gene synthesis support for COVID-19 research customers. We estimated that the shutdown had an impact of approximately $2 million in the quarter. We are currently fully operational and demand from the China market has largely returned.

Sample Management provided 3% organic growth during the quarter in line with our expectation when we started the quarter. We had described and we did see about $2 million headwind from COVID-19 in the large store systems and post warranty services. This was due to some delays in an onsite installation and general lack of access to customer sites. But there were bright spots in Sample Management as well. Year-over-year growth was driven by cryo which more than doubled, and more substantively, Bio Storage services grew 7%. We have been very encouraged by the continued significant engagements and wins by the teams in the large storage business and in Bio Storage during this environment, fueling the path to return to double digit growth.

In addition, during the second quarter we acquired the Life Science Informatics Software firm RURO. RURO provides cloud-based software solutions to manage the laboratory workflow and bio sample data for a broad range of customers in the biotech, healthcare and pharmaceutical sectors. The business contributed approximately half a million of revenue in the time that we've owned them since mid-February.

As we announced in a March press release effective April 1st, we reorganized our Life Sciences business. We've combined the Bio Storage services with GENEWIZ, leveraging the common lab services and sample handling capabilities. This business combined is now $64 million of this quarter's $95 million business and showed year-to-year growth on that basis of 17%.

The remaining portion of the segment of Life Sciences is the Life Sciences products which was about $31 million and was up 1% year-over-year. If we had avoided the negative impact of the COVID-19 constraints, we estimate that the Life Science Products business would have grown about 4%.

Another bright spot in the quarter was on the gross margin line. The segment improved nicely in the second quarter up to 45.8%, up 330 basis points compared with last year. The year-over-year improvement was driven by the performance in Sample Management. In fact, we had improvement on every area of the business including our large TwinBank's store systems and services, the cryo systems, the consumables and instruments, and the Bio Storage services. This is driven by the performance improvements and cost reductions and the improved price management that's been implemented.

The growth of the segment and gross margin expansion covered the increased operating expense and drove 260 basis points of incremental operating margin year-over-year. The leverage of this business model and growth is fully evident on this page.

As we look into the third quarter, we are more cautious due to the continued and varied impacts of COVID-19. As Steve described, we've seen inside GENEWIZ sustained demand in synthesis, some volatility in the next generation sequencing, and a lower run rate from Sanger sequencing. Most notable to use is that continued absence of the academic research institutions and we have seen only a portion of the commercial teams return to full active status. And in Sample Management, it is unclear when customer sites will fully open around the world, limiting our access for installations and services. So at this point, we're expecting Life Sciences to deliver revenue in the range of $85 million to $91 million which is about 5% to 10% lower quarter-to-quarter.

Let's turn over to Slide 6 to review our Semiconductor business. Semiconductor Solutions revenue was $125 million for the second quarter, an increase of 5% sequentially and 11% year-over-year. If you were to look into the details of the quarter-to-quarter, you would see an uptick in the systems business which we ship primarily to tier two OEMs. I would highlight that the strength in that line in is the atmospheric systems as we expected. We do expect to see more vacuum systems coming in the second half which should assist gross margin.

The real story in the revenue line however is the strength of the Contamination Control Solutions which indeed ships to end user fabs. As referenced, these are record quarters for us in CCS and we continue to see expansion of qualifications and orders across many fabs. The rest of the Semiconductor story is in robots. While it is stable quarter-to-quarter, it is significantly higher year-over-year and our tier one OEM customers continue to call for additional output as we head into our second half.

It may appear we face no challenges in the second quarter, but in fact, the accomplishment reflects much diligence to coordinate supply availability, factory production and of course customer needs. We estimate that the negative impact to COVID-19 on revenue during the quarter for Semiconductor was approximately $5 million due to supply chain and the services challenges. On the positive side, these are delayed, not lost opportunities and due to the coordination efforts, our customers were not surprised nor disappointed by us.

Gross margins in Semi were similar to the prior quarter at about 40% and operating margins improved by 70 basis points. As mix improves in the second half, we expect gross margins to strengthen and if the market holds, growth will bring operating margins back upward on the leverage of the model.

Similar to the Life Science business, we're cautious about the outlook for the third quarter. We expect our supply chain will continue to face some issues. We also are sensitive that the broader supply chain of our customers aside from us affects the end user's timing for when they need our products. Finally, we know the impact of COVID-19 on the end markets can change rapidly. We expect our semiconductor business to deliver revenue in the range of $115 million to $124 million. This is approximately flat to lower -- I'm sorry, flat to down 8% quarter-to-quarter.

Let's turn now over to Slide 7 for a summary of our cash flow over the quarter. We generated $26 million of adjusted operating cash flow during the quarter. This excludes $92 million of income tax payments associated with a gain of the 2019 sale of our semiconductor cryo business. We also used approximately $16 million of cash for the acquisition of RURO as mentioned previously.

Our CapEx amounted to $12 million for the quarter, driven primarily by investments in operations and it includes approximately $1 million for the new GENEWIZ operation facility in Suzhou, China. Total uses of cash during the quarter totaled $105 million, and the change in net cash and equivalents during the quarter brings our cash, restricted cash and cash equivalents balance down to $249 million. The highlight here is the cash capability of the business we have built with GENEWIZ and subsequent to the divestiture of the semi cryo business. Year-to-date, as I said before, our operating cash flow when you exclude the tax on the gain in the sale, was $51 million or an increase of $29 million year-over-year.

On Slide 8, you will see a summary of the balance sheet. You can see at the top of the March 31st column we now carry $249 million in cash and marketable securities. With $51 million of debt, we have $198 million of net cash for operations and investments. This strong position provides us the fuel to invest for growth and gives us the confidence we will weather these uncertain times, and customers and our employees the confidence that we will sustain our investments.

Let's turn now to the summary on Slide 9. Fundamentals of the business are intact and we've gained customers and market share during these times. We have extended our leadership and continue preparing ourselves to be ready to accelerate when the recovery of the market. The balance sheet is strong, provides us the fuel for the journey and the strategic flexibility. We have seen growth momentum in each business. In Semiconductor we saw strong orders and have healthy backlog. But we also know the WFE CapEx environment comes down to the path which the chip makers decide to take. And in this time in particular, the OEM customers depend on a broad supply chain that is vulnerable.

Our Life Sciences business continues to win new business. The active markets of pharma, biotech and clinical continue to engage on improving their infrastructure with our capabilities and to request our support for the COVID research that they have stepped up to do themselves. So meanwhile, the academic research institutions have not returned to work yet and the timing of that return is uncertain and varied across the regions. So we find ourselves with unusually mixed signals. Good customer demand momentum, but uncertain market continuity in the near term. We have solid backlog but uncertain access to some customer locations. But there is one aspect that is very clear to us. We have the momentum with customers and the opportunity to delight them. If we keep that momentum with us, we will maintain our resources and be ready for the ramp as markets do fully return. In the short term, we are carrying some costs. We have some elements of our business less active during this time, but we're going to keep the team employed. We are providing premium pay for onsite labor, ensuring the PPE and clean facilities make a safe environment and expediting shipments to satisfy stressed delivery schedules. We will carry some so this cost in the short term and we will win additional customers as we are there for them throughout and we will be prepared for the full recovery ramp as the market returns to working order.

Let's cover that guidance for the third fiscal quarter of 2020. Revenue is expected to be in the range of $200 million to $215 million. Adjusted EBITDA is anticipated to be $26 million to $34 million and non-GAAP diluted earnings per share to be $0.16 to $0.24 per share. GAAP will be in the range of $0.04 to $0.12. For Life Sciences we factored in a return of the markets in the June month. This now concludes our prepared remarks, and I'll turn the call back over to Malaika, the Operator, to take questions from the line.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first phone question is from the line of Patrick Ho with Stifel. Please go ahead, your line is open.

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J. Ho, Stifel, Nicolaus & Company, Incorporated, Research Division - MD of Technology Sector [2]

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Thank you very much and hope everyone is well. And congrats on a really nice quarter in these challenging circumstances. Steve, maybe first off, in terms of the disruptions and the volatilities you've been seeing, can you give a little bit of color on your end how you were able to manage a very fluid environment and ensure that your top tier customers got what they needed? What were some of the steps you took? How did the business continuity plan work? If you could just give a little bit of color on that first.

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Stephen S. Schwartz, Brooks Automation, Inc. - CEO, President, Interim GM of Life Sciences & Director [3]

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Sure, Patrick, and thanks for the question on this. Specifically, on the Semi side, we give a lot of credit to the large OEM customers. They give us a good look at the business. And they adjust it quite frequently, so we're able to get out in front of the supply chain. And then our team managed the supply base extremely well. I think this is something over the past years that's become really fluid. When we get a signal from our tier one OEMs, we're able to transmit that quickly to our suppliers to get them prepared and I think the team managed it really well. It wasn't without problems for sure, but everybody knew how to accelerate and to get the job done. And Patrick, inside the company, everybody knows that deliveries to the customers are top priority, so if we go overtime, we spend. If we do something to get shipments there, we do it. And so I think it's a successful model that played out through the COVID period. And I think the business continuity plans that we have in place, we kicked them into action and I think they played out extremely well. I don't know that anybody could have anticipated the magnitude of what everybody was going to be faced with. But I think the team responded well. We're functioning completely differently just even two weeks into the shutdown period. Two weeks into the shutdown period we were operating with the same fluidity, but in a really different mode. So in general, the things that should have happened did. And we continue to manage going forward the supply base. There are air pockets we're finding coming from India, from Malaysia that we've been trying to work through now for the past 5 weeks. I think we're doing very well, but when you hear uncertainty from everybody in and around the supply chain, I think we're all dealing with the same kinds of things. But I do believe the team is in front of it and we seem to be weathering it pretty well.

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J. Ho, Stifel, Nicolaus & Company, Incorporated, Research Division - MD of Technology Sector [4]

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Edited Transcript of BRKS earnings conference call or presentation 30-Apr-20 8:30pm GMT - Yahoo Finance