The federalgovernment is in the market for another used icebreaker that could be converted for use by the Canadian Coast Guard on the Great Lakes much to the dismay of shipbuilders across the country.

A request for proposals to acquire an existing light icebreaker was posted on the government's procurement website in mid-September.

The timing is interesting.Federal decision-makers have known for five years that the coast guard needssuch a vessel for the region.

The request for proposals which closes at the end of October was posted as U.S. lawmakers began to push bipartisan legislation through Congress to strengthen the U.S. Coast Guard's capacity to break ice and keep commerce flowing on the Great Lakes.

The plan for Canada to buy a used icebreaker follows a separate decision by Transport Canada to purchase a used ferry from Spain on an emergency basis.

The Canadian Marine Industry and Shipbuilding Association (CMISA), which represents most of the marine suppliers and shipyards across the country, said both decisions represent a loss of domestic jobs and at least $250 million in federal spending that could have gone into a Canadian economy hard hit by the coronavirus.

"We're of the strong belief that vessels such as light icebreakers can and should be built in Canada," said Colin Cooke, president and chief executive officer of the shipbuilding association.

"We have the capacity. We have the skilled trades. We have the expertise, the technical expertise. We have the shipyards. And that was what the point of the National Shipbuilding Strategy was all about."

That shipbuilding strategy is supposed to direct governmentwork to Canadian shipyards.Cooke said the plan to purchasean existing icebreaker and the deal to acquire a former Spanish ferry would both be unacceptable in normal times but they're even lessacceptablenow.

"We are in a COVID time when we're looking for all sorts of ways to make sure that people are employed, that businesses are able to survive I won't say thrive, I will say survive through the lockdowns caused by this pandemic," he said.

Public Services and Procurement Canada was asked for comment last Thursdaybut did notrespond.

The tender for the light icebreaker, posted online Sept. 18, describes the purchase as a necessaryinterim step for the coast guard to "bridge the gap while awaiting the delivery of dedicated new vessels."

Significantly, the request for proposals noted that the need for such a ship was identifiedfive years ago around the same time a comprehensive analysis warned that the coast guard icebreakingfleet was in dire straits and in need of immediate replacement.

"In 2015-16 the CCG identified a requirement for interim icebreaking capabilities to fill gaps in capacity resulting from ships being temporarily withdrawn from service" for refit and life extension, said the tender.

Two years ago, the Liberal government concluded a deal worth $827 millionwith Chantier Davie of Levis, Que., which operates the Davie shipyard, to refit three medium-sized commercial icebreakers for the coast guard.

Tim Choi, a University of Calgary shipbuilding expert, saidthis recent tendersuggeststhe federal government isoperating on the flawed assumption that there is an abundance of used icebreakers on the market.

The deal with the Davie shipyard was an anomaly and federal officials "got lucky" last time because there happened to be three vessels available, he said.

Choisaid he believes the federal government isn'tlikely tobe sofortunate this time:his research suggests there may beonly one light icebreaker out therethat would fit in the bill in Finland and it's notclear the Finns are ready to part with it.

"There are very few requirements for a vessel like that outside of Canada and the United States in the Great Lakes, St. Lawrence region," said Choi. "It's not like there's a used car lot where you can just go out and buy these things."

The shipbuilding association said it can make a strong case for a fast-track build in Canada.Choi said he believes procurement services may be forced in that direction anyway because of market conditions.

In mid-September, three U.S. senators Tammy Baldwin (D-WI), Todd Young (R-IN) and Gary Peters (D-MI) introduced the Great Lakes Winter Commerce Act.

The bipartisan legislation is expected to codify the U.S. Coast Guard's icebreaking operations on the Great Lakes and, more importantly, increase the size of its fleet.

"Inadequate icebreaking capacity in the Great Lakes is costing us thousands of American jobs and millions in business revenue," said Baldwin in a statement. "We must boost our icebreaking capacity in the Great Lakes to keep our maritime commerce moving."

Continued here:
Shipbuilding industry pushes back as federal government shops for used icebreaker - CBC.ca

Recommendation and review posted by Bethany Smith

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Daxor Corporation (NYSE MKT: DXR), an investment company with innovative medical instrumentation and biotechnology operations focused on blood volume measurement, today announces compelling new data demonstrating further beneficial use of Daxors blood volume analysis technology presented during the Heart Failure Society of Americas Virtual Annual Scientific Meeting 2020.

New data titled, Cost-effectiveness Analysis of Early Blood Volume-Guided Management in Hospitalized Heart Failure Patients studied the economic benefits of the use of blood volume analysis in the population of heart failure patients, one of the largest cost-drivers of the national healthcare system. The data showed an extremely cost-effective incremental cost-effectiveness ratio (ICER) of $10,200 which was 80% less costly than other therapies that are considered good value by common quality metrics. The data also revealed an average life-extension of 2.32 quality-adjusted life years (QALYs) in addition to the cost savings.

The use of Daxors BVA-100 blood test in heart failure outcomes in this compelling study showed exceptional value by not only improving patient life span, but also at a cost which is significantly less than other commonly used therapies. Daxors blood test for use in treating heart failure is demonstrably better for patient life extension outcomes compared to many other treatment regimes, while also driving excellent economic value for hospital systems, said Jonathan Feldschuh, Daxors Chief Scientific Officer.

Jean Oertel, VP of Commercialization and Customer Experience at Daxor stated, This important data shows the full power of our diagnostic technology with the ability to achieve better outcomes at reduced costs. The value of Daxors BVA-100 blood test for hospital systems and payers managing heart failure is covered by both public and private insurance with CPT/APC coding, something that many other approved tests never achieve even years after market clearance by the FDA.

About Daxor Corporation

Daxor Corporation (NYSE: DXR) is an innovative medical instrumentation and biotechnology company focused on blood volume measurement. We developed and market the BVA-100 (Blood Volume Analyzer), the first diagnostic blood test cleared by the FDA to provide safe, accurate, objective quantification of blood volume status and composition compared to patient-specific norms. The BVA technology has the potential to improve hospital performance metrics in a broad range of surgical and medical conditions, including heart failure and critical care, by informing treatment strategies, resulting in significantly improved patient outcomes. Our mission is to partner with clinicians to incorporate BVA technology into standard clinical practice and improve the quality of life for patients. For more information, please visit our website at Daxor.com.

Forward-Looking Statements

Certain statements in this release may include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation, statements regarding the impact of hiring sales staff and expansion of our distribution channels. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this release, including, without limitation, those risks associated with our post-market clinical data collection activities, benefits of our products to patients, our expectations with respect to product development and commercialization efforts, our ability to increase market and physician acceptance of our products, potentially competitive product offerings, intellectual property protection, FDA regulatory actions, our ability to integrate acquired businesses, our expectations regarding anticipated synergies with and benefits from acquired businesses, and additional other risks and uncertainties described in our filings with the SEC. Forward-looking statements speak only as of the date when made. Daxor does not assume any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Relations Contact:Bret ShapiroSr. Managing Partner, CORE IR516-222-2560brets@coreir.com

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New Research Demonstrates Daxor BVA-100 Blood Test Provides Extremely Cost-Effective Improvement in Patient Life Span at the Heart Failure Society of...

Recommendation and review posted by Bethany Smith

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CRISPR Industry Size 2019, Market Opportunities, Share Analysis up to 2026 - The Research Process

Recommendation and review posted by Bethany Smith

Several years ago, a brand new method of genetic engineering called CRISPR was invented, and it was based on discoveries made about the rudimentary "immune system" possessed by bacteria. Essentially, bacteria have a way of "remembering" which viruses had infected them previously, and they possess a molecular system that destroys viral DNA that matches that of a prior infection.

The molecular system consists of a DNA-cutting protein called Cas9. (See infographic from Business Insider below.) When equipped with a special guide RNA, Cas9 can be used to cut specific DNA sequences, for instance, a mutated gene that is causing a health problem. Because a broken DNA molecule is dangerous, the cell will attempt to repair it. If a DNA segment is snuck into the cell before the repair occurs, the cell can insert the new (and usually improved) DNA piece, providing a method to "edit" DNA.

The implications for such a technology are obvious. Such a method could be used, for example, to cure a person of a genetic disease or more easily produce genetically enhanced crops for farmers. But there are even cleverer uses. Because the CRISPR-Cas9 system can be designed to be self-propagating, it can be used to force a gene into a population of animals, such as mosquitoes. If this system targets genes that are important for survival or reproduction, then once released, this "gene drive" would rapidly spread through the population, killing off mosquitoes. (See infographic from The Economist.)

Now, a team of researchers writing in the journal Nature Communications has shown that a gene drive can be used to suppress infection with cytomegalovirus, a type of herpesvirus. The underlying molecular mechanism of the gene drive is similar to others before it: A self-propagating chunk of DNA inserts itself into a gene that is important to the virus. In this case, the gene is UL23, which is needed for cytomegalovirus to avoid the human immune response.

The researchers showed that when a cell is infected by both the normal virus (called "wildtype" or "WT") and the modified virus carrying a gene drive ("GD"), the gene drive was able to quickly and efficiently spread through the entire population, representing up to 95% of the final proportion of viruses. The end result is the suppression of viral infection (in cell culture, not in an animal model) because the gene drive virus lacks the important UL23 gene, which is needed for the virus to avoid a potent immune molecule known as interferon gamma(IFN-), which the authors added to the cell culture.

Could such a system work to treat viral infections in humans? Possibly. The authors note that a different gene (other than UL23) might need to be targeted, since lack of this gene is only fatal to the virus if IFN- is added to the cell culture. There are also concerns that a gene drive system could cause the viruses to mutate in various ways and may have unforeseen consequences.

Still, the technology is powerful and should be researched further. The coronavirus pandemic reminds us that we want to have multiple weapons in the public health arsenal should we be confronted with another life-threatening microbe.

Source: Walter, M., Verdin, E. Viral gene drive in herpesviruses. Nat Commun 11, 4884 (2020). Published: 28-Sept-2020. DOI: 10.1038/s41467-020-18678-0

More:
Gene Drives Could Kill Mosquitoes and Suppress Herpesvirus Infections - American Council on Science and Health

Recommendation and review posted by Bethany Smith

One summer day almost 20 years ago, a group of protestors arrived at a plot of genetically modified corn growing near the town of Montelimar in southern France.

They were led by Jos Bov, a left-wing activist famous for his skirmishes with the law and his tremendous moustache. Using machetes and shears, the protestors uprooted the crops and dumped the debris outside the offices of the regional government.

I thought about Bov this week as I read a new report on the next generation of genetic food technology. The techniques in the report make the processes that Bov opposed look clunky.

The GMOs he destroyed were created by inserting genes from other organisms say a stretch of DNA that confers resistance to a particular herbicide into a plants genome. This brute force approach is time-consuming and hard to control. Now scientists are using a new suite of gene-editing techniques, including a process known as CRISPR, to rapidly and precisely control the behavior of specific plant genes.

Gene-edited crops already exist. Scientists at the biotech firm Corteva, for example, have developed a high-yield strain of a variety of corn used in food additives and adhesives. Yet these initial advances belie the technologys potential.

Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones?

The power of gene editing can be wielded to modify plants and, among other things, achieve significant sustainability wins.

Here are a few potential outcomes explored in the new report, published by the Information Technology & Innovation Foundation, a pro-technology think tank:

This potential is thrilling, and there are signs that it will arrive soon. In China, where the government has made a big bet on gene-editing technology, numerous labs are working on crop strains that require less pesticides, herbicides and water. In the United States, a small but growing group of gene-editing startups is bringing new varieties to market, including an oilseed plant that can be used as a carbon-sequestering cover crop during the winter.

Yet when I read the ITIF report, I thought of Bov. Not because I agree with everything he said. Twenty years and many studies later, we know that the anti-GMO activists were wrong to say that modified crops posed a threat to human health. (The demonization of GMOs had profound consequences nonetheless: Fears about the risks posed by the crops are one reason why the crops are highly restricted in Europe and viewed warily by some consumers on both sides of the Atlantic.)

The reason I thought of Bov is that, at one level, he and other activists were pushing society to take a broader view of GMOs. They wanted people to ask who and what the crops were for, because they believed, rightly, that the crops were produced mainly with the profits of ag companies in mind.

Thats not to say its a bad thing for ag companies to be profitable. But our food systems affect so many aspects of our lives from the composition of the atmosphere to the prevalence of disease. When GMOs first began to be planted, there hadnt been enough debate about how the technology might affect these things. No wonder people were angry.

Thats a lesson I hope we can remember as gene editing shapes agriculture. Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones? If they can, we might end up with crops that everyone wants.

This article was adapted from the GreenBiz Food Weekly newsletter. Sign up here to receive your own free subscription.

See the original post:
Get ready for the next wave of GMOs | Greenbiz - GreenBiz

Recommendation and review posted by Bethany Smith

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CRISPR in Agriculture Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

Recommendation and review posted by Bethany Smith

The mistake Nobel Prize prognosticators yours truly included make is to look through the greatest hits of biochemistry, biology, and medicine (the areas STAT covers) nuclear hormone receptors! microRNAs! and figure (as last years prediction story did) one of those is due and deserving. The trouble is, as MITs Phillip Sharp, who shared the 1993 medicine Nobel, told me, There is just a lot of good science that will never get recognized.

So focusing on the greatest hits to forecast the science winners who will be announced next week is too simplistic. Theyre all contenders, but the smart money looks for other criteria. Like toggling between discoveries of what cells and molecules do and inventions of techniques that reveal what they do, or between disciplines, or (for medicine) between something that directly cures patients and something about the wonders of living cells.

By that criteria, it might be a techniques turn, since the last such winner in medicine was for turning adult cells into stem cells, in 2012. Could this be the year for optogenetics, which allows brain scientists to control genetically modified neurons with light? I dont think optogenetics has made a big enough impact outside of neuroscience yet, said cancer biologist Jason Sheltzer of Cold Spring Harbor Laboratory, who dabbles in Nobel predictions, but who knows.

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The last Nobel for DNA sequencing was way back in 1980, he pointed out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements. (Sheltzer correctly predicted 2018s medicine Nobel for immuno-oncology pioneer James Allison. The Human Genome Project could win it for the officials who led it, like Francis Collins of the National Institutes of Health and Eric Lander of the Broad Institute. Would Craig Venter, who led a competing private effort, make it to Stockholm, too? Let the betting commence!

Just to be clear, science Nobels arent chosen all that, well, scientifically. For medicine, a five-member Nobel Committee for Physiology or Medicine at Swedens Karolinska Institute sifts nominations and selects candidates. The 50-member Nobel Assembly votes, this year on Oct. 5. So you can get head-scratchers from, say, 20-18-12 or similarly split votes if, say, genetics fanciers split their votes among two contenders. (If you want to know if that happened, hang on until 2070: Nobel records are secret and sealed for 50 years.) For chemistry, chosen on Oct. 7 this year, the five-member Nobel Committee of the Royal Swedish Academy of Sciences likewise sifts nominations and recommends finalists to the academy for a vote.

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Besides invention and discovery switching off in the medicine Nobel, there certainly seems to be periodicity in terms of disciplines taking turns, said David Pendlebury of data company Clarivate Analytics. He has made 54 correct Nobel predictions (usually in the wrong year, but in 29 cases within just two) since 2002 by analyzing how often a scientists key papers are cited by peers and awarded predictive prizes like the Lasker or Gairdner awards.

Neuroscience won the medicine Nobel in 2000, 2004, 2014, and 2017, immunology in 2008, 2011, and 2018, for instance. Infectious disease and cancer win every decade or two, and so are probably also-rans for 2020. Thats why STAT said last year that the 2018 medicine award for immuno-oncology made cancer an unlikely 2019 winner. Yet William Kaelin, Peter Ratcliffe, and Gregg Semenza won for discovering how cells sense and adapt to oxygen availability, through gene regulation, which is tangentially related to cancer. Go figure.

For the medicine prize, periodicity also applies to toggling between super-basic molecular biology and stuff that actually cures people (not year by year, but generally). Last years award for how cells sense changing oxygen levels was pretty abstruse and might shape this years choice.

Prizes with a more clinical focus have been 2003 (MRI), 2005 (H. pylori and ulcers), 2008 (HIV), 2015 (roundworm and malaria therapy), and 2018 (immuno-oncology), [so] maybe a clinical type of prize this year, [such as] hepatitis C treatment, brain stimulation for Parkinsons, cochlear implant, statins Pendlebury said. We wouldnt be surprised at a hep C win for Charles Rice of Rockefeller University and Ralf Bartenschlager of Heidelberg University (2016 Lasker winners) for the super-basic discoveries that led to drugs that cure the viral disease.

Like Pendlebury, Sheltzer believes in predictive prizes. I looked back at the last 20 years of Nobel Prizes in medicine/physiology, he said. Eighty-three percent of them had won at least one of three prizes before the Nobel: the Lasker, the Gairdner, or the Horwitz Prize. Of the five people who have recently won all three, only one works in a field so far ignored by the Nobel committees, he said: Yale School of Medicines Arthur Horwich, a pioneer of protein folding and chaperone proteins. In addition to the Gairdner in 2004, Horwitz in 2008, and Lasker in 2011, he received the $3 million Breakthrough Prize in 2019. So thats guess #1, Sheltzer said.

Unless Weve had a few [medicine] awards that you could classify as cell biology recently oxygen sensing in 2019, autophagy in 2016, even immune regulation is kinda cell biological, Sheltzer acknowledged. So I think a genetics award is more likely than one to Horwich, whose discoveries about how cells fold the proteins they synthesize are central to the understanding of life. STATs nickel says look no further than the 2015 Lasker Basic Medical Research Award: It honored Evelyn Witkin of Rutgers and Stephen Elledge of Harvard for discovering how DNA repairs itself after being damaged.

Might David Allis of Rockefeller and Michael Grunstein of UCLA finally get the call to Stockholm? They discovered one way genes are activated (through proteins called histones). Theyve shared a 2018 Lasker and a 2016 Gruber Prize in Genetics, and basically launched the hot field of epigenetics. I think a prize related to epigenetic control of transcription by DNA and histone modifications could be in order, Kaelin told STAT.

For physiology or medicine, Pendlebury likes Pamela Bjorkman of Caltech and Jack Strominger of Harvard for determining the structure and function of major histocompatibility complex (MHC) proteins, a landmark discovery that has contributed to drug and vaccine development, as well as Yusuke Nakamura of the University of Tokyo for genome-wide association studies that led to personalized approaches to cancer treatment (personally, we doubt this is cancers year again), and Huda Zoghbi of Baylor College of Medicine for work on the origin of neurological disorders.

In chemistry, Pendlebury likes Moungi Bawendi of MIT, Christopher Murray of the University of Pennsylvania, and Taeghwan Hyeon of Seoul National University for synthesizing nanocrystals, a cool new way to deliver drugs, and Makoto Fujita of the University of Tokyo for discovering supramolecular chemistry, in which lab-made molecules self-assemble by emulating how nature makes them. That has some overlap with Frances Arnolds 2018 Nobel for chemistry, so were skeptical, but who knows?

Lets address the elephant in the Nobel anteroom, and the chatter that the revolutionary genome editing technique CRISPR will win for chemistry. (Its value in medicine is still TBD, but its stellar biochemistry.)

The discovery of the CRISPR-Cas9 system is certainly worthy of a Nobel Prize, Kaelin said. I suspect the challenge here will be to get the attribution right. Perhaps there could be a chemistry prize for the basic mechanism and a medicine prize for application to somatic gene editing in human cells.

By attribution, he means, who gets CRISPR credit? Only three people can share a Nobel. But CRISPR has more mothers and fathers than that. Jennifer Doudna of the University of California, Berkeley, and her collaborator Emmanuelle Charpentier have won a slew of predictive prizes for their work turning a bacterial immune system into a DNA editor, but dark horse Virginijus iknys of Vilnius University shared the 2018 $1 million Kavli Prize in nanoscience for his CRISPR work. And Feng Zhang of the Broad Institute is more widely cited than the above three, Pendlebury said, a marker of what colleagues think.

CRISPR citations built up more to Feng Zheng et al. than to Doudna and Charpentier, but I dont think that matters as much as judgments about priority claim, Pendlebury said. There are more than three to credit and I do think that is problematic. Bad feelings are not something the Nobel Assembly wants to generate, I am sure.

CRISPR will win, said CSHLs Sheltzer. Its a question of when, not if. Zhang/Doudna/Charpentier/Horvath/Barrangou shared the Gairdner. Pick 2 or 3 of them?

Go here to read the rest:
Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT

Recommendation and review posted by Bethany Smith

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

From Concept to Market: Overcoming the Challenges of Manufacturing and Clinical Trials

Learn how CRO/CDMOs successfully address operational and regulatory challenges for pharmaceutical and biotechnology clients; and how this can make the difference between study success or failure.

Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

The rest is here:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

Recommendation and review posted by Bethany Smith

SAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation toBMN 307, an investigational gene therapy for the treatment of individuals with PKU.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.

"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.BioMarin is conducting this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

BioMarin's clinical program is composed of two key studies. Phearless, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, Phenom, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

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

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company's BMN 307 program being eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions, BioMarin's gene therapy manufacturing capabilities and the anticipation that the current manufacturing capabilities will meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future and the impact of using material manufactured at commercial scale in a clinical trial on reducing risk and speeding up overall development timelines. 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:the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 -...

Recommendation and review posted by Bethany Smith

With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.

This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.

The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners nownot 18 months from now.

"Full-service CDMOs that can assist with both development and manufacturing are in highest demand,"said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."

"The supply chain is much more complex,"added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."

Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments two therapeutic areas where much of the research lies.

Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases,"said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."

Pharma and biopharma companies of all sizes can learn from this "single-source"approach. Partnering with a CDMO earlier in the processfrom preclinical development through packagingfrees up resources to focus on innovation and communication.

"It is neither simple nor cheap to develop and manufacture gene therapies,"said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."

Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.

Before you start your CDMO search, consider the following two factors:

When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.

Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead."McKinsey, October 2, 2019

Link:
Gene therapy solution: The value of a CDMO as your end-to-end partner - BioPharma Dive

Recommendation and review posted by Bethany Smith

Cell and Gene Therapy Market research report is the new statistical data source added by A2Z Market Research.

Cell and Gene Therapy Market is growing at a High CAGR during the forecast period 2020-2026. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

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Top Key Players Profiled in this report are:

JW CreaGene, Vericel, Tego Sciences, CHIESI Farmaceutici, Spark Therapeutics, GC Pharma, MolMed, AnGes, Takeda Pharmaceutical Company, APAC Biotech, Gilead Sciences, Corestem, AVITA Medical, Novartis AG, JCR Pharmaceuticals, Dendreon, CO.DON, Medipost, Osiris Therapeutics, Amgen, Biosolution, CollPlant, Japan Tissue Engineering, Organogenesis, Orchard Therapeutics, Stempeutics Research

The key questions answered in this report:

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Cell and Gene Therapy market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Cell and Gene Therapy markets trajectory between forecast periods.

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The cost analysis of the Global Cell and Gene Therapy Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

Global Cell and Gene Therapy Market Segmentation:

Market Segmentation by Type:

Cell TherapyGene Therapy

Market Segmentation by Application:

HospitalsWound Care CentersCancer Care CentersAmbulatory Surgical CentersOthers

Regions Covered in the Global Cell and Gene Therapy Market Report 2020:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The report provides insights on the following pointers:

Table of Contents

Global Cell and Gene Therapy Market Research Report 2020 2026

Chapter 1 Cell and Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Cell and Gene Therapy Market Forecast

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Comprehensive Report on Cell and Gene Therapy Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | JW CreaGene, Vericel, Tego...

Recommendation and review posted by Bethany Smith

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in boys with DMD.

Fast Track is a process designed to facilitate the development, and expedite the review, of new drugs that are intended to treat or prevent serious conditions that have the potential to address an unmet medical need. This designation was granted based on data from the Phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. DMD is a devasting condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.

DMD is a devastating and life-threatening X-linked disease that is caused by mutations in the gene encoding dystrophin, which is needed for proper muscle membrane stability and function. Patients present with muscle degeneration that progressively worsens with age to the extent that they require wheelchair assistance when they are in their early teens, and unfortunately, usually succumb to their disease by the time they are in their late twenties. It is estimated that there are ~10-12,000 individuals affected with DMD in the US.

About PF-06939926

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue. Pfizer initiated the Phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018. The goal of the study is to assess the safety and tolerability of this investigational gene therapy. Other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release is as of October 1, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy, including its potential benefits and a planned Phase 3 study for PF-06939926, that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risks associated with initial and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when regulatory authorities will approve the commencement of our planned Phase 3 study; whether and when drug applications may be filed in any jurisdictions for any potential indication for PF-06939926; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether PF-06939926 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of PF-06939926; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

Read more:
Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy - Business Wire

Recommendation and review posted by Bethany Smith

Latest Research Report: Gene Therapy industry

Gene Therapy Market report is to provide accurate and strategic analysis of the Profile Projectors industry. The report closely examines each segment and its sub-segment futures before looking at the 360-degree view of the market mentioned above. Market forecasts will provide deep insight into industry parameters by accessing growth, consumption, upcoming market trends and various price fluctuations.

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

Gene Therapy Market competition by top manufacturers as follow:SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVicalAdvantagene

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Global Gene Therapy Market research reports growth rates and market value based on market dynamics, growth factors. Complete knowledge is based on the latest innovations in the industry, opportunities and trends. In addition to SWOT analysis by key suppliers, the report contains a comprehensive market analysis and major players landscape.The Type Coverage in the Market are: Ex vivoIn vivo

Market Segment by Applications, covers:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

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Gene Therapy Market Incredible Possibilities, Growth Analysis and Forecast To 2025 - The Daily Chronicle

Recommendation and review posted by Bethany Smith

The pharmaceutical industry is one of the most scientifically innovative and competitive industries, particularly in oncology. As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. As a result, there is a need for continued innovation and increased efficiency in terms of drug development to manage cost, complexity and speed to provide potentially transformative therapies for cancer patients.

Within the last two decades, large pharmaceutical corporations have established themselves firmly in oncology by prioritising internal R&D efforts, as well as developing and accessing novel science and technology through collaborations and alliances with biotech companies and academic institutions.

Dramatic advances in the understanding of basic molecular mechanisms of underlying disease has continued to shift R&D focus toward precision medicine choosing the right therapy for a patient based on molecular understanding of their disease and less on traditional cancer therapies such as cytotoxic chemotherapies and broad-cell cycle inhibitors.

As a result of this shift in drug development, a highly concentrated overlay in product modalities and mechanisms of action has crowded the oncology pipeline across a very broad range of hematological and solid tumour indications.

The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients and how to do this faster and more efficiently.

A diversity of collaboration types

There is broad recognition that given the breadth and complexity of emerging science driving innovation in oncology, collaborations are essential in order that relevant expertise, know-how and capabilities can be combined in the right way to address patient needs.

Such collaborations take on many forms, ranging from early, multi-party alliances and consortia which are often pre-competitive in nature driving the development and shared learnings from technologies that may be enabling the field as a whole, through to more bespoke collaborations between entities.

Cell therapy research has been built on collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit

These may be more focused on collaborative research and development of novel products, to secure the necessary data for regulatory approvals to make such products available widely to the patients who can benefit from them.

Pre-competitive collaborations, often in basic and preclinical research, can reduce the barrier of competition and drive benefits for all stakeholders, most notably, the patient. As summarised by The National Institutes of Health, this includes reducing the number of redundant clinical trials, enhancing the statistical strength of studies, reducing overall costs and risks, and improving study participant recruitment, all while triggering creativity and innovation between collaborators.

These benefits strengthen capabilities and accelerate product development, ultimately producing higher quality and more effective therapies.

One powerful example is The National Institutes of Healths Partnership for Accelerating Cancer Therapies (PACT), which brought together 11 pharmaceutical companies to accelerate the development of new cancer immunotherapies.

Aligning with the focus of the Cancer Moonshot Research Initiative, PACT aimed to retrospectively analyse patient data from past clinical trials with the goal of predicting future patient outcomes.

This type of approach supports the ability to compare data across all trials and facilitates information sharing between partners, undoubtedly accelerating the pathway to effective therapies.

A second example is the establishment of The Parker Institute for Cancer Immunotherapy, to enable leading academic researchers and companies to come together in a pre-competitive setting, to enable rapid shared understanding and development of immunotherapeutic approaches, including the study of combination regimens.

Such combination trials, particularly those encompassing investigational products, have historically been challenging to undertake given the need for bespoke company-to-company and other 1:1 collaborative agreements. Bringing together multiple academic and industry participants under an open innovation model provides a basis to significantly accelerate the generation of scientific and clinical data that may substantially inform the field of cancer immunotherapy as a whole.

Oncology cell therapy research has been built on foundational academic collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit.

Examples include Kite Pharma, formed from the foundational work at the National Cancer Institute, Juno from the collaboration between the Fred Hutchinson Cancer Center and Memorial Sloan Kettering Cancer Center (all working on the first CAR T-cell candidates), or Adaptimmune working with University of Penn to first show efficacy of optimised TCR T-cells.

For collaborations that are more geared to development of novel therapies, aiming for regulatory approval and commercial availability, bespoke collaborations between biotech and pharma companies are commonplace, whereby the respective expertise and capabilities of each partner are combined in order to optimise and accelerate development, and to enable subsequent, larger scale manufacture and distribution. There are many examples of such collaborations, for which the structure can vary widely depending on the expertise of each partner, and the collaborative ways of working.

For example, under a traditional pharma/biotech collaboration and licensing model, a biotech partner may have primary responsibility for significant elements of research and early product development, and the pharma partner may lead the majority of later stage development, as well as post-approval commercial manufacture and supply. This logically aligns with organisational expertise and scale, and this type of collaboration structure has historically proven to work well. Many novel therapies have been successfully developed through such partnerships.

The rapid emergence of cell and gene therapy has required the industry to establish new and distinct capabilities, such as optimal process development and manufacture of autologous, patient specific cell therapies, whilst minimising the vein-to-vein time (the elapsed time between apheresis treatment for a patient, and reinfusing the final autologous manufactured product).

There are a growing number of biotech and pharma companies that have established or are establishing such end-to-end cell therapy capabilities, which can also play into how collaborations are structured in the field.

Case Study: From Technology Agreement to co-development and co-commercialisation partnership

In 2015, Adaptimmune and Universal Cells signed an agreement to drive the development of technologies leveraging gene-edited Induced Pluripotent Stem Cell (iPSC) lines, towards the development of allogeneic, or off-the-shelf, T-cell therapies. Universal Cells brought leading gene editing capability to make targeted gene edits to modify the characteristics of selected iPSC cell lines, and Adaptimmune the technology to differentiate iPSCs into T-cells.

Back then the science for this collaboration was early and under-developed with both parties embarking on a long-term effort and making significant at-risk investments to determine if edited, functional T-cells could be produced.

Today, Universal Cells (now an Astellas company) and Adaptimmune have established capabilities and expertise to progress novel cell therapies into clinical development, as well as with manufacturing and supply chain.

Based on this progress, in January 2020, Adaptimmune and Astellas signed a product-focused agreement to co-develop and co-commercialise up to three new stem-cell derived allogeneic T-cell therapies for people with cancer.

Given the scientific synergy between Universal Cells and Adaptimmune, and that each company is developing capabilities that may effectively address later stage product development and post-approval commercial supply, the 2020 partnership was structured as a co-development and co-commercialisation agreement. It enables the companies to work closely together, throughout the continuum of research, development and commercialisation.

Astellas and Adaptimmune will collaborate through to the end of phase 1, with Universal Cells leading gene editing activities and Adaptimmune leading iPSC to T-cell differentiation, early product characterisation and development. Beyond that, Astellas and Adaptimmune will decide whether to develop and commercialise a product candidate together under a co-development and co-commercialisation cost and profit-sharing arrangement, or for one company to take it forward alone.

This partnership is an example of how companies can harness their individual science and bring together highly complementary skills and expertise. It will enable the development of new, off-the-shelf T-cell therapies for people with cancer, which could potentially offer significant advantages such as broader access, reduced vein-to-vein time, and lower cost. The co-development and co-commercialisation nature of the agreement allows both companies to collaborate closely and on a long term basis, whilst leveraging end-to-end capabilities established by each company, maximising the velocity of product development, and ultimately delivering novel therapies to patients.

This type of agreement exemplifies how early speculative scientific collaboration can benefit all parties, most importantly the patient. It is one example from many in oncology, that underlines the value of long-term partnership within a field that is evolving rapidly across many scientific, operational and commercial frontiers.

Bringing together both teams of passionate and forward-thinking scientists may contribute to unlocking the current opportunities and challenges of off-the-shelf T-cell therapy development more effectively and efficiently for patients.

Similarly to what we are seeing as the world comes together to fight COVID-19, we as leaders in oncology owe it to patients to constantly look for ways to bring our innovative ideas as quickly as possible to the market. Working together might make that happen faster.

About the author

Helen Tayton-Martin is chief business officer at Adaptimmune.

The rest is here:
The new pharma collaborations driving transformative research in oncology - - pharmaphorum

Recommendation and review posted by Bethany Smith

As the COVID-19 pandemic has progressed, we have seen an alarming amount of disinformation spread online, including by our elected officials. In Colorado we have seen elected officials visiting restaurants in the middle of stay-at-home orders without wearing a mask.

Other elected officials have flouted local health department advice by gathering with thousands at Bandimere Speedway, where few wore masks.

We have heard them say that statewide mask mandates somehow infringe on their rights, ignoring that placing the health of others infringes on the rights of so many innocent Coloradans.They have even sued the state government for its attempts to protect public health.

Despite advice from our public health leaders to practice social distancing and wear masks to prevent the spread of the virus, President Donald Trump and Vice President Mike Pence continue to refuse to wear masks in public.

Pence leads the White Houses coronavirus task force, yet he toured the Mayo Clinic without a mask on April 28 (that same day, the U.S. reached one million COVID-19 cases).

The latest from the coronavirus outbreak in Colorado:

>> FULL COVERAGE

Recently, we have learned the alarming fact that President Trump knew back in February that this virus is spread through the air and five times deadlier than the flu.

Amid lies and conspiracy theories, our light at the end of the tunnel is scientific innovation and trust in experts. Thanks to round-the-clock collaboration between the public and private sectors, there are several COVID-19 vaccines and treatments in development.

One, for example, blocks the novel coronavirus from binding to human cells and reproducing; by stopping the virus from connecting with human cells, the drug prevents it from multiplying and attacking the body.

READ:Colorado Sun opinion columnists.

There are more than 100 different vaccines at various stages of development, and researchers are using different avenues such as gene therapy, DNA and antibodies from survivors to develop an effective vaccine.

U.S. health care innovation has saved millions of lives.HIV is now a manageable disease, no longer a death sentence.Thanks to developments in early-detection mammogram technology, female breast cancer cases dropped by 40% in 2016.

We now have a drug that can treat over 90% of Hepatitis C patients, whereas older drugs took nearly a year to become effective and even then only worked on 50% of patients.

And vaccines have saved the health of millions by preventing once-fatal illnesses. History teaches us that our best bet is to support the researchers working to develop treatments and vaccines for COVID-19.

We owe it to the frontline essential workers our grocery store workers, health care workers, sanitation services, public transit operators and so many more risking their lives every day to do better in this pandemic.

We need to ignore disinformation, whether it comes from the internet or the White House and follow the advice of our public health professionals.

We need more medical professionals and scientists to run for office to be that expert voice to help stop the spread of misinformation before it starts. Supporting and investing in their research and innovation will get us through this crisis.

Dr. Yadira Caraveo, D-Thornton, is a pediatrician and represents House District 31 in the Colorado House of Representatives.

The Colorado Sun is a nonpartisan news organization, and the opinions of columnists and editorial writers do not reflect the opinions of the newsroom. Read our ethics policy for more on The Suns opinion policy and submit columns, suggested writers and more to opinion@coloradosun.com.

Support local journalism around the state.Become a member of The Colorado Sun today!

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Opinion: Scientific innovation and trust in experts is our light at the end of the tunnel - The Colorado Sun

Recommendation and review posted by Bethany Smith

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).

Additional presentation details can be found below:

Oral Presentation

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Session Title: TreatmentPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Friday, October 16, 2020Session Time: 10:45 a.m. 12:01 p.m. CESTLecture Time: 11:45 a.m. CESTLocation: Hall D

This session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST

E-Poster

Title: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial InitiationPresenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other Agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting - Business Wire

Recommendation and review posted by Bethany Smith

LONDONandNEW YORK, Oct. 03, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced nine-month data from the ongoing Phase 1/2 clinical trial (NCT03252847) of AAV-RPGR, an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Data presented today at the EURETINA 2020 Virtual Congress demonstrated significant improvement in vision-guided mobility and retinal sensitivity in treated eyes compared to untreated eyes nine months after treatment.

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July 2020, MeiraGTx and Janssen announced six-month data from the ongoing MGT009 clinical trial showing significant improvement in retinal sensitivity in the low (n=3) and intermediate (n=4) dose cohorts in the dose escalation phase of the trial.

Data at the nine-month time point continued to demonstrate significant improvement in retinal sensitivity in treated eyes in both the low and intermediate dose cohorts. In addition, data from the assessment of vision-guided mobility carried out at the nine-month timepoint demonstrated a significant improvement in walk time compared to baseline in treated eyes compared to untreated eyes in the low and intermediate dose cohorts (n=6).

There are currently no treatment options for XLRP, and vision in patients suffering from this disease inevitably declines over time, said Michel Michaelides1, BSc MB BS MD(Res) FRCOphth FACS, MGT009 trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London. Data from this clinical trial demonstrate that patients treated with AAV-RPGR had significant and sustained improvement in retinal sensitivity, as well as improved ability to navigate in low light conditions. These exciting results continue to suggest that AAV-RPGR has the potential to be a much-needed and important treatment option for those living with XLRP.

EURETINA Data Summary:

Retinal sensitivity XLRP is characterized by progressive deterioration of the visual field. Octopus 900 full-field static perimetry and MAIA microperimetry were employed to determine change in retinal sensitivity following intervention.

Perimetry is a sensitive standard-of-care measure of retinal function that reproducibly determines retinal sensitivity both cross-sectionally and longitudinally, thereby accurately defining disease progression over time.

At the nine-month analysis (Octopus 900 static perimetry), compared to baseline:

Vision-guided mobility Markedly impaired mobility in low illumination is a hallmark symptom of XLRP. As part of the study, patients completed a vision-guided mobility maze to assess their ability to navigate across a broad range of controlled light levels (1 lux = deep twilight, 4 lux = residential street lighting, 16 lux = twilight conditions, 64 lux = car park and 256 lux = office work).

At nine-month analysis, compared to baseline:

Safety and tolerabilitySafety data obtained to date continue to suggest AAV-RPGR is well-tolerated. No dose-limiting events occurred. As previously presented, signs of inflammation were observed in two out of three patients in the high dose cohort, which may have been associated with decreased activity of the AAV-RPGR treatment in these patients. Inflammation was effectively managed with an extended steroid protocol.

Based on the safety and efficacy profile demonstrated to date, the low and intermediate doses are being evaluated in the ongoing randomized, controlled expansion portion of the Phase 1/2 study, which completed enrollment in the first half of 2020. As previously disclosed, MeiraGTx and development partner Janssen plan to advance AAV-RPGR into a Phase 3 pivotal study, called the Lumeos clinical trial.

About AAV-RPGRAAV-RPGR is an investigational gene therapy for the treatment of patients with XLRP caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. AAV-RPGR has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIME, ATMP and Orphan designations by the European Medicines Agency (EMA).

About the Phase 1/2 MGT009 Clinical TrialMGT009 is a multi-center, open-label Phase 1/2 trial (NCT03252847) of AAV-RPGR gene therapy for the treatment of patients with XLRP associated with disease-causing variants in the RPGR gene. MGT009 consists of three phases: dose-escalation, dose-confirmation, and dose-expansion. Each patient was treated with subretinal delivery of AAV-RPGR in the eye that was more affected at baseline. The patients other eye served as an untreated control. In dose-escalation (n=10), adults were administered low, intermediate, or high dose AAV-RPGR. The primary endpoint was safety. Visual function was assessed at baseline, three, six, nine and 12 months with Octopus 900 full-field static perimetry and mesopic fundus-guided microperimetry (MP); mean retinal sensitivity, visual field modeling and analysis (VFMA; Hill-of-vision volumetric measure), and pointwise comparisons were examined.

About X-Linked Retinitis Pigmentosa (XLRP)XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.

AboutMeiraGTxMeiraGTx(Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs.MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team,MeiraGTxhas taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTxs initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland,MeiraGTxintends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visit http://www.meiragtx.com.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of AAV-RPGR, plans to advance AAV-RPGR into Phase 3 clinical trial and anticipated milestones regarding our clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words expect, intend, plan, believe, project, forecast, estimate, may, should, anticipate and similar statements of a future or forward-looking nature. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, acquire additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:MeiraGTxElizabeth (Broder) Anderson(646) 860-7983elizabeth@meiragtx.com

or

Media:W2O pureChristiana Pascale (212) 257-6722cpascale@purecommunications.com

_____________________________1 Professor Michaelides is a scientific founder of and consultant to MeiraGTx.

Originally posted here:
MeiraGTx Announces Nine-Month Data from Phase 1/2 Trial of AAV-RPGR Demonstrating Significant and Sustained Vision Improvement in X-Linked Retinitis...

Recommendation and review posted by Bethany Smith

Gene Therapy Market analysis report encompasses infinite knowledge and information on what the markets definition, classifications, applications, and engagements are and also explains the drivers & restraints of the market which is obtained from SWOT analysis. Gathered market data and information is denoted very neatly with the help of most appropriate graphs, charts or tables in the entire report. Utilization of well established tools and techniques in this Gene Therapy Market document helps to turn complex market insights into simpler version. Competitive analysis studies of this market report provides with the ideas about the strategies of key players in the market.

A large scale Gene Therapy Market report endows with the data and statistics on the current state of the industry which directs companies and investors interested in this market. By applying market intelligence for this market research report, industry expert measure strategic options, summarize successful action plans and support companies with critical bottom-line decisions. The most appropriate, unique, and creditable global market report has been brought to important customers and clients depending upon their specific business needs. Businesses can accomplish great benefits with the different & all-inclusive segments covered in the Gene Therapy Market research report hence every bit of market is tackled carefully.

Get Sample PDF (including COVID19 Impact Analysis) of Market Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-gene-therapy-market&rp

Market Analysis: Global Gene Therapy Market

Global gene therapy market is rising gradually with a healthy CAGR of 36.1% in the forecast period of 2019-2026. Increasing incidence of cancer and rare life threatening diseases and strong clinical pipeline drugs for gene therapy are major drivers for market growth.

Key Market Players:

Few of the major competitors currently working in the globalgene therapy marketarePfizer Inc., Thermo Fisher Scientific Inc., F. Hoffmann-La Roche Ltd, Spark Therapeutics, Inc., bluebird bio, Inc., ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc., Sarepta Therapeutics, Novartis AG, MeiraGTx Limited, Rocket Pharmaceuticals, Lonza, Biogen, Gilead Sciences, Inc., REGENXBIO Inc., uniQure N.V., Solid Biosciences Inc., Audentes Therapeutics among others.

Get Full TOC, Tables and Figures of Market Report @https://www.databridgemarketresearch.com/toc/?dbmr=global-gene-therapy-market&rp

Market Definition: Global Gene Therapy Market

Gene therapy is a technique of insertion of genes into cells and tissues for treatment of any disease. In this technique the defective gene is replaced with a functional gene. It is the strategy of manipulation of expression of specific genes responsible for the disease. This therapy is a promising treatment option for a number of diseases. The application of gene therapy is wide and it is mostly used for treatment of cancer, cystic fibrosis, heart disease, diabetes, AIDS among others.

Gene Therapy Market Drivers

Gene Therapy Market Restraints

Segmentation:Global Gene Therapy Market

Gene Therapy Market : By Type

Gene Therapy Market : By Gene Type

Gene Therapy Market : By Viral Vector

Gene Therapy Market : By Non-Viral Vector

Gene Therapy Market : By Application

Gene Therapy Market : By End Users

Gene Therapy Market : By Distribution Channels

Gene Therapy Market : ByGeography

Key Developments in the Market:

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Originally posted here:
Gene Therapy Market Sees a Faster Rebound in the Era of COVID-19 || Leading Players ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc.,...

Recommendation and review posted by Bethany Smith

Denise Paglinawan , The Canadian Press Published Wednesday, September 30, 2020 10:22PM EDT

The father of a young Ontario boy with a rare genetic disease is asking the federal government to help fund research that could treat his child's condition, saying the support is particularly needed since the COVID-19 pandemic has made it tough to raise donations.

Terry Pirovolakis said his two-year-old son Michael was diagnosed last year with SPG50, an extremely rare disorder that causes a loss of mobility and a decline in brain functions over time.

His family has been raising money for research that could help develop treatment for Michael but the pandemic has meant many planned fundraising events had to be cancelled, he said.

It's been very difficult because we had to stop all of our fundraising campaigns, Pirovolakis said. We're all in the same situation where our lives are turned upside down but ours is a bit more complex, where we're trying to build a cure.

The family noticed Michael was not meeting his milestones as a baby, Pirovolakis said. After many tests and doctors appointments, the young boy was diagnosed with the disease that could lead to developmental delays and confine him to a wheelchair by the age of 10, the family said.

Determined to help their son, the Pirovolakis family has been trying to raise money to get the child into experimental treatment.

The family had put on events that included a golf tournament, a gala and a Christmas market to fundraise last year, but can now only rely largely on online campaigns such as their GoFundMe page while COVID-19 restrictions are still in place, Pirovolakis said.

In an effort to keep raising funds and awareness for their son's condition, Pirovolakis said he planned to bike from Pickering, Ont., to Ottawa - starting on Saturday - and has asked to meet with the Prime Minister Justin Trudeau next week.

We're seeing if there's any way that we can get some sort of funding from the federal government, he said.

Hopefully, we'll meet Mr. Trudeau and bring awareness and bring funding and support not just for my son, but for the other children affected by this disease.

The family has raised almost $1.6 million so far but is hoping to raise $3 million. Pirovolakis said research on SPG50 is being conducted in several hospitals outside Canada and involves gene therapy.

Alex Wellstead, a spokesman for the prime minister's office, said the government is in contact with the family.

It is truly inspiring to see the love and dedication that the Pirovolaskis' have for their son Michael and the hard work they've put towards this campaign, he said in an email. We will let you know when we have more to say.

This report by The Canadian Press was first published on Sept. 30, 2020.

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Family of boy with rare disease asking Ottawa to fund research into condition - CP24 Toronto's Breaking News

Recommendation and review posted by Bethany Smith

Basel, October 1, 2020 Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma (onasemnogene abeparvovec) that demonstrated patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials. SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement.1,2,3 These data as of December 31, 2019, and presented today during a virtual Clinical Trial Poster Session as part of the World Muscle Society (WMS) 2020 Virtual Congress, support the robust clinical evidence that has demonstrated a consistent, transformative benefit across Zolgensma clinical trials for the treatment of patients with SMA.

We are seeing further evidence of the potential of Zolgensma to effectively halt motor neuron loss following a one-time, intravenous infusion. In STR1VE-EU, patients achieved rapid improvements in motor function following treatment with Zolgensma, and most have already achieved motor milestones not observed in the natural history of SMA Type 1, said Professor Eugenio Mercuri, M.D., PhD., Department of Pediatric Neurology, Catholic University, Rome, Italy. These interim results are especially encouraging considering STR1VE-EU includes some patients with a more severe phenotype than in the START and STR1VE-US studies, further supporting the gene therapys positive benefit/risk profile, even in this more fragile population.

"These strong interim results from the STR1VE-EU clinical trial continue to demonstrate consistent and significant therapeutic benefit in patients with SMA Type 1, the most common form of the disease, adding to the robust body of clinical evidence for Zolgensma, said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies. With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients.

Phase 3 STR1VE-EU Data as of December 31, 2019STR1VE-EU is designed to evaluate the efficacy and safety of a single, one-time IV infusion of Zolgensma in patients with SMA Type 1 who are less than six months of age at the time of gene therapy, with one or two copies of theSMN2backup gene and who have bi-allelicSMN1gene deletion or point mutations. The mean age of dosing was 4.1 months and the mean age at the onset of symptoms was 1.6 months. The mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score at baseline was 28. Thirty-one of 33 patients (93.9%) were able to swallow thin liquids, and 10 patients (30.3%) required feeding support at baseline. Nine of thirty patients (27.3%) required ventilatory support at baseline. STR1VE-EU is distinct in its inclusion and exclusion criteria and baseline clinical characteristics of enrolled patients compared with START or STR1VE-US. Specifically, some patients in STR1VE-EU had a more severe disease phenotype at baseline, including lower CHOP-INTEND scores and the need for nutritional and ventilatory support.

At last visit before the data cutoff, patients in STR1VE-EU were between 6.9 and 18.6 months of age, and mean duration in the study was 10.6 (1.815.4) months. Thirty-one out of 32 (97%) patients in the intent-to-treat (ITT) population survived event-free, including 30 (93.8%) who could have reached 10.5 months of age and 18 (56.3%) who could have reached 13.6 months of age. An event is defined as the need for tracheostomy or the requirement of 16 hours of respiratory assistance per day (via non-invasive ventilatory support) for 14 consecutive days in the absence of an acute reversible illness, excluding peri-operative ventilation. Untreated natural history indicates that only 50% and 25% of babies with SMA Type 1 will survive event-free by the time they reach 10.5 months of age and 13.6 months of age, respectively.3

Twenty-one patients (65.6%) achieved motor milestones not observed in the natural history of SMA Type 1. This includes six patients (18.8%) who could sit independently for 10 seconds (the primary efficacy endpoint), 20 patients (66.7%) who gained head control, eight patients (25%) who were able to roll from back to sides and one patient who could stand with assistance, crawl and walk with assistance. The mean increase in CHOP INTEND from baseline was 5.9 points (n=31) which was observed as early as at one month post-dosing, 10.1 points at 3 months (n=29) post-dosing, and 13.3 points at six months (n=27) post-dosing. Twenty-one children (65.6%) enrolled in STR1VE-EU achieved and maintained a CHOP INTEND score of 40 points and 12 children (37.5%) were able to achieve a score of 50. According to natural history, untreated patients with SMA Type 1 almost never achieve a CHOP INTEND score 40.3,4

The majority (91.7%) of patients who were free of ventilatory support at baseline remained either completely free of ventilatory support or received prophylactic BiPAP support during the study for acute reasons. Two-thirds (66.7%) of patients in the ITT population were able to feed orally without the need for feeding support, an important indicator of stabilization/halting of disease progression.

As previously reported, one patient discontinued the study because of a serious adverse event of hypoxic-ischemic brain damage and respiratory distress that resulted in death. Novartis and the investigator considered the events and death to be unrelated to treatment with Zolgensma based on autopsy findings. Thirty-two of 33 patients were reported to have at least one adverse event (AE), of which six patients experienced serious adverse events that were considered by the investigator to be related to Zolgensma. Liver transaminase elevations, some of which were reported as adverse events, were experienced by 29 of 33 patients (87.9%), but all resolved with the use of prednisolone. Four patients had reported decreases in platelet counts <75,000, three of which were isolated laboratory abnormalities without adverse events reported. Overall, no new safety signals have been identified and the reported adverse events are consistent with the cumulative safety profile with Zolgensma.

Novartis Gene Therapies is grateful to the courageous patients and families who participate in clinical trials, enabling the company to further its efforts to make a meaningful difference in the lives of patients with rare genetic diseases.

About Zolgensma (onasemnogene abeparvovec)Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma was approved by the U.S. Food and Drug Administration in May 2019 and represents the first approved therapeutic in Novartis Gene Therapies proprietary platform to treat rare, monogenic diseases using gene therapy.5 In addition to the United States, Zolgensma is approved in Japan, Europe and Brazil. More than 600 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program. Novartis Gene Therapies is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, and South Korea in late-2020 or early 2021.5

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

About Spinal Muscular AtrophySMA is the leading genetic cause of infant death.1,2 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.3,4SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.1 It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.5 This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.6 More than 30% of patients with SMA Type 2 will die by age 25.7

About Novartis Gene TherapiesNovartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are turning promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). This therapy is now approved in the U.S., Japan, Europe and Brazil, and additional registrations are being pursued in close to three dozen countries, with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, Argentina and South Korea in late 2020 or early 2021. Our robust AAV-based pipeline is advancing treatments for Rett syndrome; a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene; and Friedreichs ataxia. We are powered by the worlds largest gene therapy manufacturing footprint of more than one million square feet, enabling us to bring these therapies to patients around the world at quality and scale.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, can, will, plan, may, could, would, expect, anticipate, seek, look forward, believe, committed, investigational, pipeline, launch, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact media.relations@novartis.com

References1. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15:895908.2. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. Available at: http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed October 29, 2019.3. Finkel RS, McDermott MP, Kaufmann P. et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83:8107.4. Kolb SJ, et al. Ann Neurol. 2017;82:88391.5. SolerBotija C, et al. Brain. 2002;125:162434.6. Wang CH, et al. J Child Neurol. 2007;22:102749.7. Darras BT, Finkel RS. Natural history of spinal muscular atrophy. In: Sumner CJ, Paushkin S, Ko CP, eds. Spinal Muscular Atrophy: Disease Mechanisms and Therapy, 2nd ed. London, UK: Academic Press/Elsevier;2017:399421.

# # #

Novartis Media RelationsE-mail: media.relations@novartis.com

Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

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Zolgensma data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival,...

Recommendation and review posted by Bethany Smith

Global Gene Therapy Market Research Report 2020: COVID-19 Outbreak Impact Analysis

Brand Essence Market Research has developed a concise study on the Gene Therapy market to depict valuable insights related to significant market trends driving the industry. The report features analysis based on key opportunities and challenges confronted by market leaders while highlighting their competitive setting and corporate strategies for the estimated timeline.

Download Sample PDF (including full TOC, Tables, and Figures) of Gene Therapy Market Research 2020-2026:- https://brandessenceresearch.biz/Request/Sample?ResearchPostId=72699&RequestType=Sample

Global Gene therapy market is valued at USD 0.67 Billion in 2018 and expected to reach USD 5.18 Billion by 2025 with the CAGR of 33.9 % over the forecast period. Increasing prevalence of the cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure is further driving Gene therapy market.

Gene Therapy is associated with the experimental technique that uses genes to treat or prevent disease. In the future, this system could permit doctors to treat a condition by inserting a gene into patients cells rather than exploitation medication or surgery. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve bodys ability to fight disease. Gene therapy holds potential for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. For example, suppose a brain tumor is forming by rapidly isolating cancer cells.

Global gene therapy market report is segmented on the basis of Therapy type, Vector Type, application, and regional& country level. Based upon Therapy type, gene therapy market is classified as somatic and germline. Based upon Vector type Gene Therapy Market is classified into non-viral vectors and viral vectors. Based upon Application Gene therapy market is classified into Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others.

The regions covered in this Global Gene therapy market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of Gene therapy is sub divided into U.S., Mexico, Canada, UK, France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Gene therapy Market ReportsGlobal Gene therapy market Report covers prominent players are like Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene.

Increasing prevalence of cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure is expected to grow Gene therapy market. Growing prevalence of cardiovascular disease, cancer, genetic disorder, and painful existing treatment procedure coupled with the increasing application of public health and clinical facilities is expected to drive Gene therapy market. As this therapy is transforming the current approaches to promote the excellence in health and prevent from cardiovascular diseases such as cancer and diabetes. In 2017, it is projected that around 14% (more than 34 million adults) were current smokers. While People with diabetes comprise 8.8% of the worlds population and IDF predicted that the number of cases of diabetes will rise to 642 million by 2040. In low-income countries, mainly in Africa, the older population faces a considerable burden of both non-communicable and communicable diseases. High cost of gene therapy treatment and unwanted immune responses such factors hindering the growth of gene therapy market. Technological Advancements leading to provide better technological procedures is the lucrative opportunity for gene therapy market in forecast period.

Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global Gene Therapy Market.

Key Benefits for Gene Therapy Market Reports Global market report covers in-depth historical and forecast analysis. Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level. Global market report helps to identify opportunities in market place. Global market report covers extensive analysis of emerging trends and competitive landscape.

By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions Others

By Type of Vectors Viral vectors Non-viral vectors

By Type of Cells Somatic cells Germline cells

By Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

North America is dominating the Gene therapy Market North America is dominating the gene therapy market during the forecast period due to increasing healthcare expenditure coupled with advancement of technologies. The majority of gene therapies clinical trials recognized targeted cancer diseases. In the US, around 66.81% of gene therapy clinical trials are carried out. While all other countries participated in a small percentage of the trials such as 9.45% in the UK, 3.95% in Germany and around 2% each in Switzerland, France, China, and Japan. Globally, regeneration medicine companies providing Gene cell therapy and tissue engineering for therapeutic developers is expected to grow gene therapy market in this region.

Gene Therapy Market Key Players: Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

This comprehensive report will provide:

Enhance your strategic decision making Assist with your research, presentations and business plans Show which emerging market opportunities to focus on Increase your industry knowledge Keep you up-to-date with crucial market developments Allow you to develop informed growth strategies Build your technical insight Illustrate trends to exploit Strengthen your analysis of competitors Provide risk analysis, helping you avoid the pitfalls other companies could make Ultimately, help you to maximize profitability for your company.

Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market? Which are the roadblock factors of this market? What are the new opportunities, by which market will grow in coming years? What are the trends of this market? Which are main factors responsible for new product launch? How big is the global & regional market in terms of revenue, sales and production? How far will the market grow in forecast period in terms of revenue, sales and production? Which region is dominating the global market and what are the market shares of each region in the overall market in 2017? How will each segment grow over the forecast period and how much revenue will these segment account for in 2025? Which region has more opportunities?

>>>>Get Full Customize report@ https://brandessenceresearch.biz/Request/Sample?ResearchPostId=72699&RequestType=Customization

Our industry professionals are working reluctantly to understand, assemble and timely deliver assessment on impact of COVID-19 disaster on many corporations and their clients to help them in taking excellent business decisions. We acknowledge everyone who is doing their part in this financial and healthcare crisis.

The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global Gene Therapy market.

Chapter 2:Evaluating the leading manufacturers of the global Gene Therapy market which consists of its revenue, sales, and price of the products.

Chapter 3: the competitive nature among key manufacturers, with market share, revenue, and sales.

Chapter 4:Presenting global Gene Therapy market by regions, market share and revenue and sales for the projected period.

Chapters 5, 6, 7, 8 and 9:To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

Read More: https://brandessenceresearch.biz/Lifesciences-and-Healthcare/Gene-Therapy-Market-Share/Summary

About us: Brandessence Market Research and Consulting Pvt. ltd.

Brandessence market research publishes market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students. We have a delivery center in Pune, India and our sales office is in London.

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Global Gene Therapy Market 2020: Growth, Latest Trend Analysis and Forecast 2025 - The Daily Chronicle

Recommendation and review posted by Bethany Smith

MIAMI--(BUSINESS WIRE)--OrganaBio, LLC (OrganaBio) announces the launch of the MesenPAC and ImmunoPAC product lines, designed to support translational researchers in their quest to rapidly develop next generation cell-based therapies. These products provide a unique and timely solution to critical supply chain issues that are hindering the regenerative medicine, cell therapy, gene therapy, and immunotherapy arenas.

MesenPAC mesenchymal stem/stromal cells (MSCs) and ImmunoPAC natural killer cells (NKs) are derived from full-term placenta, umbilical cord tissue, and umbilical cord blood obtained from consented, non-compensated donors under IRB approved protocols. OrganaBios focus on building its proprietary supply chain ensures donors are strictly qualified, screened, and enrolled, fresh tissues are used to manufacture the companys cells, and robust, well-characterized cell banks are created in formats that cell and gene therapy developers can readily plug into their process and product development workflows, said Mr. Justin Irizarry, CEO.

The MesenPAC-MSC product line consists of high-volume placenta and umbilical cord MSCs isolated and manufactured under xeno-free conditions. Paired with a high-efficiency bioprocess media system and supported by process recommendations for rapid expansion, the MesenPAC system generates lot sizes in the billions of cells in a matter of days, compared to the months of cell culture necessitated by traditional MSC culture regimens. MesenPAC-MSCs are also supported by a comprehensive quality data package elucidating the critical quality attributes of each lot manufactured, including expansion potential, population doubling level, differentiation potential, immunomodulatory potential, and cytokine secretion in vitro. The initial ImmunoPAC product offering consists of NK cells isolated via positive selection from fresh umbilical cord blood. Cells demonstrate high viability and purity post-thaw. In addition, all donors are HLA typed, and this information is provided with each cell lot manufactured.

OrganaBios supply chain ownership gives us the unique ability to donor match MSCs from placenta and umbilical cord, and to further pair them with donor matched immune cells. Now, researchers may test the effects of tissue of origin on cell function, without the confounding effect of donor variability. For immunotherapy developers, our 2x resolution HLA typing also allows exploration of how donor matching or mismatching, and to what degree, affects preclinical outcomes. This is especially intriguing since clinical outcomes suggest that less stringent resolution HLA matching is sufficient with the transplantation of nave immune cells compared to those from adult peripheral blood, said Dr. Priya Baraniak, Vice President of Corporate Development and Process and Product Development lead for OrganaBio. We look forward to rapidly bringing additional cell types (including hematopoietic stem cells (HSCs), T cells and B cells) that the industry urgently needs for development of allogeneic therapies to market in the coming months.

With the launch of the MesenPAC and ImmunoPAC product lines, OrganaBio is positioned to set new industry standards in cellular raw materials for therapeutics development. Our steadfastness in building a robust, secure supply chain, coupled with our dedication to providing translational researchers high quality products that fit their process and product development needs, and further supporting this with a cGMP manufacturing solution via our multi-tenant GMP facility, provides much needed solutions to the cell, gene, and immunotherapy arenas and will rapidly move allogeneic product development through the preclinical pipeline and into First-in-Man studies, said Justin Irizarry.

ABOUT ORGANABIO

OrganaBio, LLC is a privately held solutions provider for the cell, gene, and immunotherapy fields. OrganaBio has a proprietary supply chain of perinatal tissue (including cord blood and cord tissue) from non-compensated donors and offers a range of starting cellular materials from these tissues. OrganaBio manufactures off-the-shelf research grade products for allogeneic cell therapy development and is building cGMP manufacturing capabilities, with a forecasted launch in Q3 2021. OrganaBios state-of-the-art, multi-tenant cGMP facility will not only be used to manufacture the companys cellular raw materials for therapeutics development but will also offer the industry cleanrooms and support services to rapidly manufacture clinical materials. This new paradigm in cGMP manufacturing affords companies flexibility and agility, significantly reducing manufacturing costs and timelines. For more information, visit http://www.organabio.com and follow the company on LinkedIn and Twitter (@organabio).

Read this article:
OrganaBio Launches Portfolio of Novel Perinatal Tissue-Derived Products to Spur Rapid Development of Cell and Gene Therapies - Business Wire

Recommendation and review posted by Bethany Smith

Gene Therapy for Age-related Macular Degeneration Marketreport analyses the market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, and market demand and supply scenarios. The report covers the present scenario and the growth prospects of the global Gene Therapy for Age-related Macular Degenerationmarket for 2020-2025.

The Gene Therapy for Age-related Macular DegenerationMarket Report further describes detailed information about tactics and strategies used by leading key companies in the Gene Therapy for Age-related Macular Degenerationindustry. It also gives an extensive study of different market segments and regions.

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The Top players are RetroSense Therapeutics, REGENXBIO, AGTC, .

Market Segmentation:

By Product Type: Subretinal, Intravitreal, Unspecified

On the basis of the end users/applications, Monotherapy, Combination Therapy

Impact of COVID-19:

Gene Therapy for Age-related Macular Degeneration Market report analyses the impact of Coronavirus (COVID-19) on the Gene Therapy for Age-related Macular Degeneration industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Gene Therapy for Age-related Macular Degeneration market in 2020.

The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.

COVID-19 can affect the global economy in 3 main ways: by directly affecting production and demand, by creating supply chain and market disturbance, and by its financial impact on firms and financial markets.

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FOR ALL YOUR RESEARCH NEEDS, REACH OUT TO US AT:Address: 6400 Village Pkwy suite # 104, Dublin, CA 94568, USAContact Name: Rohan S.Email:[emailprotected]Phone: +1-909-329-2808UK: +44 (203) 743 1898Website:

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Top key Players Impacting the Growth of the Gene Therapy for Age-related Macular Degeneration Market 2020 | COVID19 Impact Analysis | Key Players:...

Recommendation and review posted by Bethany Smith

Acquisition to support accelerated commercialization of Koligos KYSLECEL,a personalized islet cell therapy available in the U.S. for chronic and recurrent acute pancreatitis

Goal to rapidly advance KT-PC-301, an autologous cell therapy under investigationfor the treatment of COVID-19-related Acute Respiratory Disease Syndrome (ARDS)

Orgenesis to leverage Koligos 3D-V bioprinting technology across its POCare Platform

GERMANTOWN, Md., Sept. 29, 2020 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS) (Orgenesis or the Company), a global biotech company working to unlock the full potential of cell and gene therapies, and Koligo Therapeutics, Inc. (Koligo), a regenerative medicine company, today announced that the two companies have entered into a definitive merger agreement, subject to final closing conditions, with expected completion before year-end (Transaction).

Koligo is a leader in developing personalized cell therapies utilizing the patients own (autologous) cells. Koligo has successfully launched its first commercial product, KYSLECEL, and plans to commence a phase 2 trial of KT-PC-301 for COVID-19-related ARDS. Koligos development stage technology utilizes 3D bioprinting and vascularization with autologous cells (3D-V technology) to create biodegradable and shelf-stable three-dimensional cell and tissue implants. The 3D-V technology is being developed for diabetes and pancreatitis, with longer term applications for neural, liver, and other cell/tissue transplants.

Following closing of the Transaction, Orgenesis plans to accelerate the commercial scaleup of KYSLECEL throughout the United States and, subject to regulatory and logistical considerations, in international markets as well. After closing of the Transaction, and subject to FDA review and clearance of the Companys Investigational New Drug application, Orgenesis expects to start patient recruitment for a phase 2 randomized clinical trial of KT-PC-301 in COVID-19 patients. Orgenesis also plans to leverage Koligos 3D-V bioprinting technology across its POCare platform.

Under the terms of the merger agreement, Orgenesis will acquire all of the outstanding stock of Koligo from its shareholders (the founders and staff of Koligo and a subsidiary of Bergen Special Opportunity Fund, LP, an institutional investor managed by Bergen Asset Management, LLC). The agreed consideration terms are an aggregate of $15 million in shares of Orgenesis common stock valued at $7.00 per share which shall be issued to Koligos accredited investors (with certain non-accredited investors to be paid solely in cash) and an assumption of $1.3 million in Koligos liabilities, estimated to be substantially all of Koligos liabilities. Additional details of the Transaction will be available in the Companys Form 8-K, which will be filed with the Securities and Exchange Commission, and will be available at http://www.sec.gov.

KYSLECELKoligos KYSLECEL is commercially available in the United States for chronic and recurrent acute pancreatitis in a surgical procedure commonly called Total Pancreatectomy with Islet Autologous-Transplant (TPIAT). TPIAT has been proven to provide significant pain relief, improved quality of life, and a reduction in the need for pain medication for patients suffering from chronic or recurrent acute pancreatitis. KYSLECEL infusion after a total pancreatectomy helps preserve insulin secretory capacity and reduce the risk of diabetic complications. KYSLECEL is made from a patients own pancreatic islets the cells that make insulin to regulate blood sugar.

Koligo has commenced its commercial pilot program for KYSLECEL at six U.S. hospitals, treating 40 patients to date. The KYSLECEL pilot program has generated approximately $2 million in sales revenue. KYSLECEL has also been shown to result in significant savings to payors over traditional chronic pancreatitis management. Following the closing of the Transaction, Orgenesis plans to make KYSLECEL available to an increasing number of hospitals throughout the United States through its POCare Network.

KT-PC-301Koligos lead clinical development program is for KT-PC-301, an autologous cell therapy under investigation for the treatment of COVID-19-related Acute Respiratory Disease Syndrome (ARDS). KT-PC-301 is comprised of autologous stromal and vascular fraction cells (SVF) derived from each patients adipose (fat) tissue. KT-PC-301 contains a population of mesenchymal stem cells, vascular endothelial cells, and immune cells which migrate to the patients lungs and other peripheral sites of inflammation. Nonclinical and clinical evidence demonstrate that KT-PC-301 may: (1) stabilize microcirculation to improve oxygenation; (2) maintain T and B lymphocytes to support antibody production; and (3) induce an anti-inflammatory effect.

Koligo has completed a pre-IND (Investigational New Drug) consultation with the U.S. Food and Drug Administration to start clinical trials of KT-PC-301 in COVID-19-related ARDS. Following the closing of the Transaction, and subject to FDA review and clearance of the Companys Investigational New Drug application, Orgenesis expects to start patient recruitment for a phase 2 randomized clinical trial of KT-PC-301 in COVID-19 patients. As currently planned, the phase 2 trial is expected to enroll 75 patients and evaluate the safety and efficacy of KT-PC-301. Mohamed Saad, MD, Chief of Division of Pulmonary, Critical Care, and Sleep Disorders Medicine at the University of Louisville, will be the lead clinical investigator on the trial.

3D-V Technology Koligos 3D-V bioprinting technology is designed to support development of a number of product candidates for the treatment of diabetes, cancer, neurodegenerative disease, and other serious diseases. The 3D-V technology platform is able to print three-dimensional cell and tissue constructs with a vascular network. Key benefits of the 3D-V approach include: faster revascularization/inosculation of cell/tissue transplant to improve engraftment; host tolerance of the graft while minimizing need for immune suppressive drugs; better site of transplant administration of such products; and scaffolding to keep cell/tissue in place in vivo. These solutions are ideally suited for islet transplant and other cell/tissue transplant applications.

Koligo ManagementFollowing the closing of the Transaction, Koligos management team will be joining Orgenesis to continue commercial and development activities. Koligos CEO, Matthew Lehman, is an accomplished executive in the biotech and regenerative medicine fields. Prior to co-founding Koligo, he was CEO of Prima Biomed Ltd (now Immutep Ltd, a Nasdaq (IMMP) and ASX (IMM) listed biotech company). Stuart Williams, PhD, Chief Technology Officer, is a bioengineer and thought leader in regenerative medicine, with over 300 publications and 20 issued patents in the field. Dr. Williams has co-founded three other biotech companies and is an experienced academic-industry collaborator. Michael Hughes, MD, Chief Medical Officer, is a transplant surgeon who started the islet transplant program at University of Louisville which was the genesis of Koligos KYSLECEL program. He has successfully treated nearly 50 chronic pancreatitis patients with islet autologous transplant after pancreatectomy. Balamurugan Appakalai, PhD, has more than 20 years of islet isolation experience, having processed more than 800 human pancreases. He is a leader in the field of islet transplant with 100+ publications.

Vered Caplan, Chief Executive Officer of Orgenesis, stated, We are pleased to announce this transformative acquisition, which we expect will add broad capabilities to our therapeutic and technology platform, and will further our leadership in the cell and gene therapy field. Based on several phase 1 studies, Koligos KT-PC-301, using a patients own cells, has demonstrated safety and tolerability, and has shown signs of efficacy to support continued development in COVID-19-related ARDS. If successful for the treatment of COVID-19-related ARDS, KT-PC-301 is likely to have applications in other acute and chronic respiratory indications, areas that represent significant unmet medical need. In addition, we see significant potential in KYSLECEL, a commercial stage asset for the treatment of chronic and acute recurrent pancreatitis, which we plan to introduce through our global network of hospitals. Finally, Koligos 3D-V bioprinting technology is highly complementary to our POCare Platform, as we implement new technologies to improve efficacy and lower the costs of cell and gene therapies. I would like to personally welcome Matthew and the rest of the Koligo team to the Orgenesis organization when the Transaction closes. We believe that their skills and experience will be an important addition as we execute on our strategy to unlock the power of cell and gene therapies and make them accessible to all.

Matthew Lehman, Chief Executive Officer of Koligo Therapeutics, stated, The merger with Orgenesis marks a major milestone for our company and builds on our recent progress, including the Pre-IND package submitted to the U.S. FDA for KT-PC-301 and our pilot commercial program for KYSLECEL. The Orgenesis team brings extensive clinical, regulatory, and manufacturing expertise well suited to supporting Koligos goals. Orgenesis intellectual property is highly complementary to Koligos technology and the combined companies will work to advance a robust commercial and development product portfolio. Orgenesis POCare technologies are also ideally suited for low-cost and efficient production of autologous cell therapies at the point of care, which we believe will considerably enhance the delivery of these therapies to patients. Additionally, we believe Orgenesis global network of leading hospitals and healthcare institutions will enable us to accelerate the commercial rollout of KYSLECEL. We are quite encouraged by the outlook for the business and look forward to leveraging Orgenesis POCare Platform in order to accelerate the timeline to bringing our innovative cell therapies to market. Through this merger, we believe we can maximize value for all shareholders and we are grateful to Orgenesis for this opportunity.

Pearl Cohen Zedek Latzer Baratz LLP and KPMG advised Orgenesis on the Transaction. Maxim Group LLC acted as a finder and Nelson Mullins Riley & Scarborough, LLP advised Koligo on the Transaction.

About Koligo Therapeutics Koligo Therapeutics, Inc. is a US regenerative medicine company. Koligos first commercial product is KYSLECEL (autologous pancreatic islets) for chronic and acute recurrent pancreatitis. Koligos 3D-V technology platform incorporates the use of advanced 3D bioprinting techniques and vascular endothelial cells to support development of transformational cell and tissue products for serious diseases. More information is available at http://www.koligo.net.

About OrgenesisOrgenesis is a global biotech company working to unlock the full potential of celland gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis POCarePlatform is comprised of three enabling components: a pipeline of licensedPOCare Therapeuticsthat are processed and produced in closed, automatedPOCare Technologysystems across a collaborativePOCare Network. Orgenesisidentifies promising new therapies and leverages its POCare Platform to provide a rapid, globally harmonized pathway for these therapies to reach and treat large numbers of patients at lowered costs through efficient, scalable, and decentralized production. The Network brings together patients, doctors, industry partners, research institutes and hospitals worldwide to achieve harmonized, regulated clinical development and production of the therapies. Learn more about the work Orgenesis is doing atwww.orgenesis.com.

Notice Regarding Forward-Looking Statements The information in this release is as of September 29, 2020. Orgenesis assumes no obligation to update forward-looking statements contained in this release as a result of new information or future events or developments. This release contains forward looking statements about Orgenesis, Koligo, Koligos technology, and potential development and business opportunities of Koligo and Orgenesis following the closing of the Transaction, each of which involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of the Companys products; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any such regulatory authorities may approved the Companys development products, and, if approved, whether such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of the Companys products; uncertainties regarding the impact of COVID-19 on the Companys business, operations and financial results and competitive developments.

A further description of risks and uncertainties can be found in the Companys Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov.

Contact for Orgenesis:Crescendo Communications, LLCTel: 212-671-1021ORGS@crescendo-ir.com

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Orgenesis Announces Agreement to Acquire Koligo Therapeutics, a Leader in Personalized Cell Therapies - GlobeNewswire

Recommendation and review posted by Bethany Smith

Newswise For over 15 years, ETH Professor Andreas Hierlemann and his group have been developing microelectrode-array chips that can be used to precisely excite nerve cells in cell cultures and to measure electrical cell activity. These developments make it possible to grow nerve cells in cell-culture dishes and use chips located at the bottom of the dish to examine each individual cell in a connected nerve tissue in detail. Alternative methods for conducting such measurements have some clear limitations. They are either very time-consuming - because contact to each cell has to be individually established - or they require the use of fluorescent dyes, which influence the behaviour of the cells and hence the outcome of the experiments.

Now, researchers from Hierlemann's group at the Department of Biosystems Science and Engineering of ETH Zurich in Basel, together with Urs Frey and his colleagues from the ETH spin-off MaxWell Biosystems, developed a new generation of microelectrode-array chips. These chips enable detailed recordings of considerably more electrodes than previous systems, which opens up new applications.

Stronger signal required

As with previous chip generations, the new chips have around 20,000 microelectrodes in an area measuring 2 by 4 millimetres. To ensure that these electrodes pick up the relatively weak nerve impulses, the signals need to be amplified. Examples of weak signals that the scientists want to detect include those of nerve cells, derived from human pluripotent stem cells (iPS cells). These are currently used in many cell-culture disease models. Another reason to significantly amplify the signals is if the researchers want to track nerve impulses in axons (fine, very thin fibrous extensions of a nerve cell).

However, high-performance amplification electronics take up space, which is why the previous chip was able to simultaneously amplify and read out signals from only 1,000 of the 20,000 electrodes. Although the 1,000 electrodes could be arbitrarily selected, they had to be determined prior to every measurement. This meant that it was possible to make detailed recordings over only a fraction of the chip area during a measurement.

Background noise reduced

In the new chip, the amplifiers are smaller, permitting the signals of all 20,000 electrodes to be amplified and measured at the same time. However, the smaller amplifiers have higher noise levels. So, to make sure they capture even the weakest nerve impulses, the researchers included some of the larger and more powerful amplifiers into the new chips and employ a nifty trick: they use these powerful amplifiers to identify the time points, at which nerve impulses occur in the cell culture dish. At these time points, they then can search for signals on the other electrodes, and by taking the average of several successive signals, they can reduce the background noise. This procedure yields a clear image of the signal activity over the entire area being measured.

In first experiments, which the researchers published in the journalNature Communications, they demonstrated their method on human iPS-derived neuronal cells as well as on brain sections, retina pieces, cardiac cells and neuronal spheroids.

Application in drug development

With the new chip, the scientists can produce electrical images of not only the cells but also the extension of their axons, and they can determine how fast a nerve impulse is transmitted to the farthest reaches of the axons. "The previous generations of microelectrode array chips let us measure up to 50 nerve cells. With the new chip, we can perform detailed measurements of more than 1,000 cells in a culture all at once," Hierlemann says.

Such comprehensive measurements are suitable for testing the effects of drugs, meaning that scientists can now conduct research and experiments with human cell cultures instead of relying on lab animals. The technology thus also helps to reduce the number of animal experiments.

The ETH spin-off MaxWell Biosystems is already marketing the existing microelectrode technology, which is now in use around the world by over a hundred research groups at universities and in industry. At present, the company is looking into a potential commercialisation of the new chip.

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Recording thousands of nerve cell impulses at high resolution - Newswise

Recommendation and review posted by Bethany Smith