Archive for the ‘Gene Therapy Research’ Category

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.

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The new pharma collaborations driving transformative research in oncology - - pharmaphorum

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

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

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.

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

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.

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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.

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

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

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?

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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

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

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.

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

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).

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

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

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

Culture-adapted AAV2 is a viral vector which is used to deliver gene therapy to the liver. However, clinical trials targeting diseases of the liver have had an 'unexpectedly low success rate' using the vector, according to researchers: who have now found that naturally occurring AAVs may be more effective.

The prototypical AAV2 discovered more than 50 years ago provides the serotype on which the field of AAV vectorology and gene therapy is based. The researchers from Australias Childrens Medical Research Institute (CMRI) say the discovery 'will shake the foundations of the field of AAV-based gene therapeutics and will mark the beginning of a new era not only for biomedical research, but most importantly, for millions of patients affected by genetic disorders'.

One area of interest in gene therapy is using AAVs to target the liver, which is involved in genetic disorders such as haemophilia and various enzyme deficiencies.

AAV2 is a viral vector used to deliver gene therapy to the liver, carrying therapeutic DNA to target cells in the body. It binds to a receptor on the target cell. However, the researchers found that while AAV2 binds to the attachment receptors - heparan sulfate proteoglycans (HSPCs) - it does so too tightly.

This means that the vector can get trapped on other cells in the body and not the target liver cells. This reduces the number of vectors that deliver their therapeutic cargo to the liver, diminishing therapeutic efficacy.

The teams of Dr Leszek Lisowski, Head of the Translational Vectorology Research Unit, and Prof Ian Alexander, Head of the Gene Therapy Research Unit, then turned to naturally occurring vectors isolated from liver samples. They found that these which use an as of yet unknown receptor are much more successful at delivering therapies to the liver.

CMRI researchers are now able to make vectors in the lab that use this better receptor, instead of HSPGs, potentially making the next generation of gene therapy targeting the liver 'vastly more successful'.

Theorizing that manufacturing methods could be playing a role, the researcherscompared traditional AAV vectors grown in culture with naturally occurring vectors that they isolated from liver samples. They observed that the cultured vectors rapidly mutated as they replicated in the lab: with these changes making the vectors bind more tightly to molecules called HSPGs on the surface of liver cells, but also impeding their ability to infect humanized liver cells in mice.

In contrast, the naturally occurring vectors infected liver cells more efficiently and bound less tightly to HSPGs, although these effects disappeared when the scientists grew the natural vectors in culture over time.

This really challenges a basic concept in our field that binding strongly to HSPG was essential for AAVs' entry into human cells and suggests that vectors targeting the other receptor used by natural AAVs, of human liver origin, are likely to be more effective for clinical gene therapy applications, said Dr Lisowski. The prototypical AAV2, discovered over 50yrs ago, is the serotype on which the entire field of AAV vectorology and gene therapy is based.

Our study sheds new light and challenges our previous understanding and corrects misconceptions about how the vector binds to the cells.

Researchers at the CMRI can now start to improve on the use of vectors to help children with liver conditions. A better vector can increase safety and improve efficiency, while the increased therapeutic efficacy will mean lower doses are needed and thus reduce the cost of treatment.

The insights on adeno-associated virus receptor binding can potentially be extended to other tissues beyond the liver, add researchers. This makes this a very impactful study which will change the trajectory of AAV-based gene therapies.

Adeno-associated viruses (AAVs) were discovered in the 1960s. The vectorization of AAV2, a human isolate, in 1984 set in motion the development the use of the viral vector in gene therapy.

The liver is a key target for developing more efficient AAV vector delivery, given its direct involvement in a number of genetic and acquired diseases.

Source: Science Translational Medicine, September 9, 2020.DOI: 10.1126/scitranslmed.aba3312

Title: Restoring the natural tropism of AAV2 vectors for human liver

Authors: M. Cabanes-Creus; C.V. Hallwirth; A. Westhaus; B.H. Ng; S.H.Y. Liao; E. Zhu; R.G. Navarro; G. Baltazar; M. Drouyer; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander; L. Lisowski at University of Sydney in Westmead, NSW, Australia; C.V. Hallwirth; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander at Sydney Children's Hospitals Network in Westmead, NSW, Australia; A. Westhaus; G. Santilli; A.J. Thrasher at University College London in London, UK.

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Researchers explore naturally occurring viral vectors: 'Our study will change the trajectory of AAV-based gene therapies' - BioPharma-Reporter.com

-- Continued functional improvements were observed at 18 months in the low-dose cohort --

-- First look at functional outcomes in high-dose cohort found improvements 6 months after administration --

-- Results in both cohorts continue to reinforce safety and tolerability profile of SRP-9003 --

CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Companys investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included 18-month functional data from three clinical trial participants in the low-dose cohort and 6-month functional data from three participants in the high-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

There are currently no approved treatments for people with LGMD2E a disease that causes significant disability in children and often leads to early mortality. Its very encouraging that we continue to see consistent, positive data from our investigational gene therapy SRP-9003 across several measures, as we know the community needs more options, said Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy, Sarepta Therapeutics. The improvements in functional measures at 18- and 6- months in participants from both cohorts who received SRP-9003 are distinctly different from what an age-matched, natural history group would predict with LGMD2E. This sustained durability of the response in functional outcomes reinforces that SRP-9003 is getting to the muscle and suggestive of improvement against disease-mediated muscle damage. When coupled with the strong expression results and encouraging safety profile seen to date, todays results increase our confidence in the construct and provide additional evidence as we advance the higher dose of SRP-9003 into the next stage of clinical testing.

Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed at 90 days. Cohort-specific results as follows:

Cohort 1 (low dose), at 18 months:

Cohort 2 (high dose), at 6 months:

About SRP-9003 and the studySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 5x1013vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 2x1014vg/kg. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days.

Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-Girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding future clinical testing for SRP-9003, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases, the potential benefits of SRP-9003 and potential market opportunities.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

InternetPosting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com.Weencourageinvestorsandpotentialinvestorsto consult our website regularly for important information about us.

Source:Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional...

BOSTON and LONDON, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

We are encouraged by EMAs decision to grant PRIME designation to OTL-203, which was based on an initial clinical assessment of data supporting the potential benefit of our HSC gene therapy for patients with MPS-IH beyond the current standard of care, said Anne Dupraz-Poiseau, PhD., chief regulatory officer of Orchard. In 2021, we look forward to building upon the promising early data in the ongoing proof-of-concept study and plan to initiate a registrational trial to advance a potential new treatment for patients.

The PRIME program is designed to enhance regulatory support in the EU for the development of promising investigational medicines that, based on early clinical data, may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier: enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the marketing authorization application. For more information please visit the EMA website at http://www.ema.europa.eu.

Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of MPS-I. Eight patients have been treated in the study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Orchard expects to release full proof-of-concept results at one year and initiate a registrational study for OTL-203 in 2021.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidate referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidate referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended June 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I - BioSpace

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press release: The UW has a long history of pioneering medical advancements that have transformed the world. From performing the first bone marrow transplant in the United States to cultivating the first laboratory-derived human embryonic stem cells. Now, where will UW medical research go next?

On the next Wisconsin Medicine Livestream, meet trailblazing doctors, researchers, and medical leaders who are charting a bold course to completely alter the health care landscape. During this insightful panel discussion, well explore how gene therapy and cell replacements could hold the keys to treating inherited and acquired blindness. Youll also discover the remarkable potential in xenotransplantation where nonhuman animal source organs are transplanted into human recipients. In addition, you will learn about UW Healths journey to build a multidisciplinary program to serve the community. These, and other, fascinating developments in treatment and care are happening right now at the UW and are the future of medicine. The presentation will be moderated by Robert Golden, the dean of the University of WisconsinMadisons School of Medicine and Public Health.

Our Guests:

David Gamm, professor, Department of Ophthalmology and Visual Sciences; Emmett A. Humble Distinguished Director, McPherson Eye Research Institute; Sandra Lemke Trout Chair in Eye Research

Dr. Gamms lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases (RDDs). As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. The Gamm Lab uses induced pluripotent stem cells to create retinal tissues composed of authentic human photoreceptor cells rods and cones that can detect light and initiate visual signals in a dish. The aims of his laboratory are to investigate the cellular and molecular events that occur during human retinal development and to generate cells for use in retinal disease modeling and cell replacement therapies. In collaboration with other researchers at UWMadison and around the world, the lab is developing methods to produce and transplant photoreceptors and/or retinal pigment epithelium (RPE) in preparation for future clinical trials. At the same time, the Gamm Lab uses lab-grown photoreceptor and RPE cells to test and advance a host of other experimental treatments, including gene therapies. In so doing, the lab seeks to delay or reverse the effects of blinding disorders, such as retinitis pigmentosa and age-related macular degeneration, and to develop or codevelop effective interventions for these RDDs at all stages of disease.

Dhanansayan Shanmuganayagam, assistant professor, Department of Surgery, School of Medicine and Public Health; Department of Animal and Dairy Sciences, UWMadison; director, Biomedical, and Genomic Research Group

Dr. Shanmuganayagams research focuses on the development and utilization of pigs as homologous models to close the translational gap in human disease research, taking advantage of the overwhelming similarities between pigs and humans in terms of genetics, anatomy, physiology, and immunology. He and his colleagues created the human-sized Wisconsin Miniature Swine breed that is unique to the university. The breed exhibits greater physiological similarity to humans, particularly in vascular biology and in modeling metabolic disorders and obesity. He currently leads genetic engineering of swine at the UW. His team has created more than 15 genetic porcine models including several of pediatric genetic cancer-predisposition disorders such as neurofibromatosis type 1 (NF1). In the context of NF1, his lab is studying the role of alternative splicing of the nf1 gene on the tissue-specific function of neurofibromin and whether gene therapy to modulate the regulation of this splicing can be used as a viable treatment strategy for children with the disorder.

Dr. Shanmuganayagam is also currently leading the efforts to establish the University of Wisconsin Center for Biomedical Swine Research and Innovation (CBSRI) that will leverage the translatability of research in pig models and UWMadisons unique swine and biomedical research infrastructure, resources, and expertise to conduct innovative basic and translational research on human diseases. The central mission of CBSRI is to innovate and accelerate the discovery and development of clinically relevant therapies and technologies. The center will also serve to innovate graduate and medical training. As the only center of its kind in the United States, CBSRI will make UWMadison a hub of translational research and industry-partnered biomedical innovation.

Petros Anagnostopoulos, surgeon in chief, American Family Childrens Hospital; chief, Section of Pediatric Cardiothoracic Surgery; professor, Department of Surgery, Division of Cardiothoracic Surgery

Dr. Anagnostopoulos is certified by the American Board of Thoracic Surgery and the American Board of Surgery. He completed two fellowships, one in cardiothoracic surgery at the University of Pittsburgh School of Medicine and a second in pediatric cardiac surgery at the University of California, San Francisco School of Medicine. He completed his general surgery residency at Henry Ford Hospital in Detroit. Dr. Anagnostopoulos received his MD from the University of Athens Medical School, Greece. His clinical interests include pediatric congenital heart surgery and minimally invasive heart surgery.

Dr. Anagnostopoulos specializes in complex neonatal and infant cardiac reconstructive surgery, pediatric heart surgery, adult congenital cardiac surgery, single ventricle palliation, extracorporeal life support, extracorporeal membrane oxygenation, ventricular assist devices, minimally invasive cardiac surgery, hybrid surgical-catheterization cardiac surgery, off-pump cardiac surgery, complex mitral and tricuspid valve repair, aortic root surgery, tetralogy of Fallot, coronary artery anomalies, Ross operations, obstructive cardiomyopathy, and heart transplantation.

When: Tuesday, Sept. 29, at 7 p.m. CDT

Where: Wisconsin Medicine Livestream: wiscmedicine.org/programs/ending-alzheimers

Read more here:
ONLINE: The Future of Medicine - Isthmus

NEW YORK, Sept. 23, 2020 /PRNewswire/ --Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today the signing of a Sponsored Research Agreement (SRA) with Duke University to use the MPTP-induced Parkinson's Disease (PD) mouse model to establish in vivo proof-of-concept study to demonstrate that administration of LV-dCas9-DNMT3A virus can prevent and/or delay PD and test the efficacy and safety of SLS-004. Subsequently, other pre-clinical models would be utilized to further validate the investigational product.

"We are extremely pleased to begin the in vivo target engagement study with SLS-004 at Duke as it builds onto the work we began in the spring focused on designing a vector capable of inducing and suppressing Parkinson's related phenotypes," said Raj Mehra Ph.D., Chairman and CEO of Seelos. "Initiating this next portion of the studies at Duke should help us further validate this approach in Parkinson's."

Seelos has also begun work on designing a vector capable of inducing and suppressing PD-related phenotypes by carrying a unit to overexpress the alpha-synuclein (-synuclein) protein and a unit to mediate inducible suppression of -synuclein. The process aims to create a next-generation suppressive unit, carrying a more effective effector molecule than previously used, in the form of DNA methyltransferase 3A and 3L (DNMT3A & L). This research may help to advance a potential novel, effective and precise tool for reversing SNCA pathologies that can provide a valuable new therapeutic strategy for treating PD.

About SLS-004

SLS-004 is a novel epigenome-editing approach to modulate expression of SNCA gene mediated by modification of DNA-methylation. SLS-004 utilizes an all-in-one lentiviral vector harboring dCas9-DNA methyltransferase 3A (DNMT3A) to enrich DNA-methylation within CpGs island at the SNCA intron 1 region. The system resulted in a precise and fine-tuned downregulation (30%) of SNCA overexpression in hiPSC-derived dopaminergic neurons from a PD patient with the triplication of the SNCA locus (SNCA-Tri). Most importantly, the reduction of SNCA expression mediated by the developed system was sufficient to ameliorate disease related cellular phenotypes. The in vitro studies achieved several key millstones including the establishment that DNA hypermethylation at SNCA intron 1 allows an effective and sufficient tight downregulation of SNCA expression levels and suggests the potential of this target sequence combined with the CRISPR-dCas9 technology as a novel epigenetic-based therapeutic approach for PD.

Forward Looking Statements

Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, among others, those regarding the initiation of a proof-of-concept study to demonstrate whether administration of the LV-dCas9-DNMT3A virus can prevent and/or delay PD, the potential for the LV-dCas9-DNMT3A virus to prevent and/or delay PD, the efficacy and safety of SLS-004, the potential use of other pre-clinical models to validate SLS-004, the potential for the in vivo proof-of-concept study to validate the design of a vector capable of inducing and suppressing PD-related phenotypes, and the potential for Seelos' research to advance a novel, effective and precise tool for reversing SNCA pathologies. These statements are based on Seelos' current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated with Seelos' business include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies and not gaining marketing approvals for its product candidates, the risk that prior test results may not be replicated in future studies and trials, the risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval, the risks associated with the implementation of a new business strategy, the risks related to raising capital to fund its development plans and ongoing operations, risks related to Seelos' current stock price, risks related to the global impact of COVID-19, as well as other factors expressed in Seelos' periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we do not know whether our expectations will prove correct. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, even if subsequently made available by us on our website or otherwise. We do not undertake any obligation to update, amend or clarify these forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.

Contact Information:Anthony MarcianoHead of Corporate CommunicationsSeelos Therapeutics, Inc. (Nasdaq: SEEL)300 Park Ave., 12th FlNew York, NY 10022(646) 293-2136anthony.marciano@seelostx.comwww.seelostherapeutics.comhttps://twitter.com/seelostxhttps://www.linkedin.com/company/seelos

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Seelos Therapeutics Announces Sponsored Research Agreement with Duke University for Gene Therapy Studies of SLS-004 in Parkinson's Disease - WFMZ...

Sept. 28, 2020 11:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the companys investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease, said Geoff MacKay, AVROBIOs president and CEO. Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. Were pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care and, we hope, the quality of life for people living with this rare genetic disorder.

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

About Gaucher Disease

Gaucher disease is a rare, inherited lysosomal storage disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages. Macrophages bloated with these fatty substances are called Gaucher cells which amass primarily in the spleen, liver and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts and an adjusted lifetime relative risk of developing Parkinson's disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy (ERT) the current standard of care people with Gaucher disease type 1 typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life. An estimated 1 in 44,000 people are diagnosed with Gaucher disease.

About AVR-RD-02

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patients own hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase 1/2 clinical trial.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated into the patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the vector.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, including AVR-RD-02 for the treatment of Gaucher disease; the anticipated benefits of the European Commissions grant of orphan drug designation for AVR-RD-02; the design, commencement, enrollment and timing of ongoing or planned clinical trials and regulatory pathways; the timing of patient recruitment and enrollment activities, clinical trial results, and product approvals; the anticipated benefits of our gene therapy platform including the potential impact on our commercialization activities, timing and likelihood of success; the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs; and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in pre-clinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized; the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators; the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials; the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform; the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate; the risk that prior results, such as signals of safety, activity or durability of effect, observed from pre-clinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates; the risk that we will be unable to obtain and maintain regulatory approval for our product candidates; the risk that the size and growth potential of the market for our product candidates will not materialize as expected; risks associated with our dependence on third-party suppliers and manufacturers; risks regarding the accuracy of our estimates of expenses and future revenue; risks relating to our capital requirements and needs for additional financing; risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises; and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200928005176/en/

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AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease -...

NEWARK, Del., Sept. 24, 2020 /PRNewswire/ --Neurophth Therapeutics, Inc., (hereinafter referred to as "Neurophth") today announced that its leading candidate, NR082 (rAAV2-ND4, NFS-01 project), was granted an orphan drug designation (ODD) by the U.S. FDA for the treatment of Leber's Hereditary Optic Neuropathy associated with ND4 mutation.

Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited mitochondrial disease, characterized by acute or subacute, painless vision loss or even loss simultaneously or sequentially, accompanied by central visual field defect and color vision impairment with poor prognosis. It was first reported by German scholar Leber in 1871. It affects about 1-9:100,000 people worldwide. LHON is one of the blinding diseases. The disease mainly occurs in young- and middle-aged men. Currently, there is no effective treatment for LHON. About 70% - 90% of LHON is caused by ND4 mutation of harboring a point mutation at nucleotide 11778 associated with a G-to-A transition. With the development of NR082, AAV-based gene therapy of LHON becomes possible.

"Due to the lack of effective treatment, the quality of life of LHON patients associated with ND4 mutation is very poor, and a huge unmet medical needs have not been fulfilled," said Dr. Alvin Luk, Chief Executive Officer at Neurophth. "NR082 is the first candidate drug developed by Neurophth. It uses recombinant adeno-associated virus serotype 2 to deliver the genetically modified ND4 gene (rAAV2-ND4). After a single intravitreal injection, the gene is translated and expressed in cells, which effectively supplements the function loss caused by endogenous mutation. Through this gene therapy, the electron transport function of mitochondrial respiratory chain was maintained, and the increase of ATP synthesis restored the normal function of mitochondria, which in turn improved the sensory function of the retinal ganglion cells and improved the visual acuity of LHON patients."

Luk added, "the significance of orphan drug designation is that regulators recognize the unmet medical needs of rare diseases like LHON. The recognition of NR082 will reduce the R&D investment to a certain extent and accelerate the progress of clinical trials and marketing registration. Furthermore, Neurophth is committed to fundamentally solve the causes and change the quality of life of patients through a single treatment of gene therapy."

Professor Bin Li, Founder, Chairman and Chief Scientific Officer at Neurophth, said: "NR082 has been granted as orphan drug by U.S. FDA, which further strengthens our focus on gene therapy for rare ophthalmic diseases, and develops more drugs for treatment of ocular genetic diseases, bringing hope to patients with ocular genetic diseases in the world".

*FDA grants orphan drug designation to drugs and biological products designed to safely and effectively treat, diagnose, or prevent rare diseases or conditions affecting less than 200,000 people in the US. According to the Orphan Drug Act of FDA, Orphan Drug Designation (ODD) provides opportunities for grant funding, fast approval channel, and some incentives, such as waiver of New Drug Application (NDA) fees, tax credits for clinical trial expenses and exemption for prescription drug users' fees as well as the products are entitled to a seven-year of market exclusivity and will not be affected by patents.

About NR082 (rAAV2-ND4; NFS-01 Project)

LHON disease is caused by mutations in mitochondrial DNA 11778, 14484 or 3460. ND4 gene of 11778 G>A mutation is the main pathological factor, which exists in 55-70% of European and American patients and 90% of Chinese patients. NR082 (NFS-01 project) is an innovative candidate drug for ophthalmic AAV-based gene therapy. It uses AAV2 vector to express human ND4 gene in the retinal ganglion cells to repair optic neuropathy caused by 11778 G>A mutation.

As early as 2011, Professor Bin Li's team started the world's first LHON gene therapy investigator-initiated trial (IIT) with this candidate drug. Nine subjects who participated in the clinical trial have been followed up for up to 8 years with no serious adverse reactions, and 5 of them have significant improvement in their vision. This result is the longest follow-up record of gene therapy in the world, which has already been published in the Scientific Report, EBioMedicine and Ophthalmology journals, and has fully proven the long-term safety, effectiveness, and durability of AAV gene therapy in clinical settings.

After the gratifying results of the first study, Professor Li's team conducted a more comprehensive IIT clinical study from 2017 to 2018, with 159 subjects (including 10 subjects from Argentina), which is the largest clinical trial in the entire gene therapy in the world. Among those, 143 of the patients has completed the 12-month follow-up and 56.6% showed a significant BCVA (best-corrected visual acuity improved by at least 0.3 LogMAR) improvement. No serious adverse reaction was found. In May 2020, at the 23rd online annual meeting of the American Society for Gene and Cell Therapy (ASGCT) and the online annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), Neurophth presented these two clinical research data on the treatment of LHON with NR082 (NFS-01 project of rAAV2-ND4), demonstrating the international advanced level of this research in the field of gene therapy.

Following the positive results of these two IIT trials, Neurophth is actively preparing the China/U.S. IND (Investigational New Drug) applications, and plans to carry out the registration clinical Phase 1/2/3 registration trial to evaluate the safety, efficacy and durability of NR082.

About Neurophth

As a clinical-stage R & D company, Neurophth is committed to exploring and developing new therapies for global patients with ophthalmic diseases. With the help of the mature AAV ophthalmic gene therapy technology platform and the deep understanding of the ophthalmology field by the founding team for decades, Neurophth has established a rich, robust product pipeline, including more than 10 research projects for various ophthalmic diseases, such as dominant hereditary optic atrophy, optic nerve injury diseases, vascular retinopathy, etc., and gradually expanded from rare to common ophthalmic diseases. Additionally, the company is preparing to build a GMP commercial production platform for gene therapy drugs accordance with the international quality standards, and plans to build an ophthalmic gene therapy transformational excellence center, aiming to become a global leader in gene therapy in ophthalmology to benefit patients all over the world.

Prospect of Gene Therapy in Ophthalmology

Inherited retinal diseases (IRDs) have long been regarded as an ideal disease area for gene therapy, because most of the gene mutations leading to the disease have been identified (more than 200 gene defects are associated with the most common IRDS). The eye is, to some extent, an immune privilege. Clinical trials have shown that gene therapy using adeno-associated virus (AAV) or lentivirus (LV) vectors in the eye does not cause systemic side effects and does not cause significant immune responses. The most common IRDs were Retinitis Pigmentosa (RP), Achromatopsia color blindness (ACHM), Leber Hereditary Optic Neuropathy (LHON), Leber Congenital Amaurosis (LCA), Stargardt disease and X-linked Retinoschisis (XLRS).

To date, only one ophthalmic AAV gene therapy product has been approved in the world, namely the first AAV2 gene therapy voretigene neparvovec-rzyl (LUXTURNA; Spark Therapeutics) approved by FDA in December 2017 to treat IRD caused by RPE65 double allele mutation in adult or pediatric patients. The approval of LUXTURNAhas brought confidence and hope to the global ophthalmic gene therapy field. Public information disclosed that at least 20-30 kinds of gene therapy for ophthalmic diseases are in the research and development stage, and the international representative companies include Applied Genetic Technologies Corporation and Meira GTX, and new companies represented by Neurophth have also begun to enter the international stage of ophthalmic gene therapy.

References

Contact:Dr. Alvin LukAlvin.Luk@neurophth.com

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SOURCE Neurophth Therapeutics, Inc.

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Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by US FDA - BioSpace

SHANGHAI, Sept. 28, 2020 /PRNewswire/ -- On September 25, 2020, BIORAY LABORATORIES Inc. ("Bioraylab"), a company specialized in gene therapy and cell drug R&D, announced for the first time that clinical trials of non-viral PD1 specific targeted CAR T therapy in relapsed/refractory B-cell non-Hodgkin lymphoma, undertaken by the company in cooperation with East China Normal University and the First Affiliated Hospital, School of Medicine, Zhejiang University, has achieved significant breakthroughs. This is the world's first application of gene editing technology to realize PD1-knockin CAR T treatment, and is also the world's first clinical trial treating lymphoma with non-viral PD1 specific targeted CAR T cells.

The latest research results, finished by East China Normal University, the First Affiliated Hospital, School of Medicine, Zhejiang University and Bioraylab, were announced on the preprint platform medRxiv on September 23, 2020.

PD1 specific targeted CD19-CARTis Bioraylab's Quikin CART platform technology using its proprietary IP, precisely inserting CAR cassette into PD1 locus without using virus, thus generating the CAR T product in just one step. The product combines PD1 immune checkpoint inhibition with CART anti-tumor activity, generating effects of both anti-PD1 immunotherapy and CAR T therapy. Several ongoing clinical trials demonstrate the outstanding safety and effectiveness of this CAR T product.

Two patients showed complete remission following a three-month treatment

The clinical trial program enrolled 15 patients, among which, four patients who were able to be evaluated showed partial remission (PR) after receiving the one-month treatment, while two patients showed complete remission (CR) after three months of treatment.

There were no CAR T cell related high-grade (3) adverse eventsduring the entire treatment, including cytokine release syndrome (CRS)and neurologic toxicity. After infusion, the CAR T cells were well sustained in vivo.

The Quikin CART technology produces CAR T cells without using virus, greatly reducing high costs of producing CAR T products, while avoiding the cancer risk from random insertion. The regulation of T cell endogenous genes and the constant expression of CAR are realized in just one step. Compared with other CAR T technologies, Quikin CART has a variety of advantages, including a simpler process, fewer production links, shorter time preparation and higher product uniformity.

Liu Mingyao, chief scientist at Bioraylab and professor at East China Normal University, said, "Without using virus, the Quikin CART technology can realize the precise integration of CAR cassette into genome as well as regulation of T cell endogenous genes in just one step, delivering many unparalleled advantages compared with existing CAR T technologies. The technology offers a strong platform for the development of more diverse CAR T products in the future."

Huang He, the project investigator of this clinical trial and chairman of the First Affiliated Hospital, School of Medicine, Zhejiang University, commented, "The current results showed that the non-viral PD1-knockin CAR T cellsproduced by using the Quikin CART technology have great potential to treat patients. We are very delighted to see patients rapidly show CR after treatment, and are looking forward to safer and more durable long-term effects on relapsed/refractory patients through this novel CAR T technology."

In addition to the ongoingclinical trials of non-viral PD1 specific targeted CD19-CART, Bioraylab is conducting research on other non-viral specific targeted CART products for treating solid tumors, with the aim of achieving more breakthroughs in CAR T therapy.

About Bioraylab

Founded in Shanghai in 2013, BiorayLaboratories Inc.("Bioraylab")is already well positioned to become the world's leading gene cell pharmaceutical manufacturer, by way of innovation with gene editing and the development of breakthrough therapies that benefit all mankind. Bioraylab owns 108 patents, conductedIIT trials for fiveprojects in eightof the world's leadinghospitals, three of which having entered the IND application stage. Bioraylab has, over the past five years, published 12 high-level academic papers in internationally renownedjournals, including Nature Biotechnology and Nature Medicine. The firmhas built three technology platforms for gene editing, cell therapy and gene therapy, and has a 6,000-square-meter GMP pilot plant and an operating team comprising nearly 100 people, placing the firm in the position of being able to guarantee the rapid transformation and application of innovative research results.

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World's first clinical trial of non-viral PD1 specific targeted CAR T therapy achieves great breakthroughs - WFMZ Allentown

Timothy Ray Brown, the first person known to have been cured of HIV infection, says he is now terminally ill from a recurrence of the cancer that prompted his historic treatment 12 years ago.

Brown, dubbed "the Berlin patient" because of where he lived at the time, had a transplant from a donor with a rare, natural resistance to the AIDS virus. For years, that was thought to have cured his leukemia and his HIV infection, and he still shows no signs of HIV.

But in an interview with the Associated Press, Brown said his cancer returned last year and has spread widely. He's receiving hospice care where he now lives in Palm Springs, Calif.

"I'm still glad that I had it," Brown said of his transplant. "It opened up doors that weren't there before" and inspired scientists to work harder to find a cure, which many had begun to think was not possible, the 54-year-old said Sept. 24.

"Timothy proved that HIV can be cured, but that's not what inspires me about him," said Dr. Steven Deeks, an AIDS specialist at the University of California at San Francisco, who has worked with Brown to further research toward a cure.

"We took pieces of his gut, we took pieces of his lymph nodes. Every time he was asked to do something, he showed up with amazing grace," Deeks said.

Brown was an American working as a translator in Berlin in the 1990s when he learned he had HIV. In 2006, he was diagnosed with leukemia.

Dr. Gero Huetter, a blood cancer expert at the University of Berlin, believed that a marrow transplant was Brown's best chance of beating the leukemia. He wondered, could he also cure Brown's other life-threatening disease by using a donor with a gene mutation that provides natural resistance to the AIDS virus?

Donors like these are very rare and transplants are risky. Doctors have to destroy the patient's diseased immune system with chemotherapy and radiation, then transplant the donor's cells and hope they develop into a new immune system for the recipient.

Brown's first transplant in 2007 was only partly successful: His HIV seemed to be gone but his leukemia was not. He had a second transplant from the same donor in March 2008 and that one seemed to work.

Since then, Brown has repeatedly tested negative for HIV and has frequently appeared at AIDS conferences where cure research is discussed.

"He's been like an ambassador of hope," said Brown's partner, Tim Hoeffgen.

A second man, Adam Castillejocalled "the London patient" until he revealed his identity earlier this yearalso is believed to have been cured by a transplant similar to Brown's in 2016.

But donors like these are scarce and the procedure is too risky to be widely used.

Scientists have been testing gene therapy and other ways to try to get the effect of the favorable gene mutation without having to do a transplant. At an AIDS conference in July, researchers said they may have achieved a long-term remission in a Brazil man by using a powerful combination of drugs meant to flush dormant HIV from his body.

Mark King, a Baltimore man who writes a blog for people with HIV, said he spoke with Brown recently and is grateful for what Brown has contributed to AIDS research.

"It is unfathomable what value he has been to the world as a subject of science. And yet this is also a human being who is a kind, humble guy who certainly never asked for the spotlight," King said. "I think the world of him."

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First man cured of HIV infection now has terminal cancer - Modern Healthcare

The continuing spread of Coronavirus (COVID-19) amongst major global economies has become an important factor of concern for import and export activities. Learn how companies in the Gene Therapy Industry are responding to the Coronavirus crisis by gaining efficacy in alternative strategies that are stabilizing various business activities. Browse through our latest research analysis on COVID-19 and its impact on the global market landscape.

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The report on the global Gene Therapy market published by Fact.MR provides a clear understanding of the flight of the Gene Therapy market over the forecast period (2020 to 2026). The study introspects the various factors that are tipped to influence the growth of the Gene Therapy market in the upcoming years. The current trends, growth opportunities, restraints, and major challenges faced by market players in the Gene Therapy market are analyzed in the report.

The study reveals that the global Gene Therapy market is projected to reach a market value of ~US$XX by the end of 2026 and grow at a CAGR of ~XX% during the assessment period. Further, a qualitative and quantitative analysis of the Gene Therapy market based on data collected from various credible sources in the market value chain is included in the report along with relevant tables, graphs, and figures.

Relevant Takeaways from Report:

Gene Therapy Segmentation

By Product Type

The report highlights the product adoption pattern of various products in the Gene Therapy market and provides intricate insights such as the consumption volume,

By End-User,

Key Players

The global Gene Therapy market expected to be fragmented due to the low to medium presence of international and local market players. Some of the key players are identified across the value chain of the global Gene Therapy market which is as-

The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to market segments such as geographies, applications, and industry.

The report covers exhaustive analysis on:

Regional analysis includes:

The report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts, and industry participants across the value chain. The report provides an in-depth analysis of parent market trends, macro-economic indicators, and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

Report Highlights:

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The report addresses the following doubts related to the Gene Therapy Market:

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COVID19: Impact on Gene Therapy Industry, Finds Fact.MR - The Cloud Tribune

Monday, September 28, 2020

The prospect of genetic engineering using CRISPR (clustered regularly interspaced short palindromic repeats) and CRISPR-associated nucleases (Cas) has long been hailed as a revolutionary development in medicine.

This technology is rapidly advancing, and several CRISPR/Cas-based drugs have entered clinical trials over the past several years. One kind of product in clinical trials is CRISPR-modified cells, such as CTX001 (CRISPR-Cas9-modified autologous hematopoietic stem cells), currently under study for the treatment ofb-thalassemiaand severe sickle cell anemia. Another CRISPR-based product,AGN-151587, is injected into the eye with the goal of eliminating a genetic mutation in patients with Leber congenital amaurosis 10, a leading cause of childhood blindness. In parallel, others are working to harness theCRISPR/Cas system to develop drugs for rare diseases, including bespoketherapiestailored to an individual patients needs.

Given CRISPR/Cas-based drugs potential to treat rare diseases, issues relating to orphan drug exclusivity will arise as these products are developed. In May 2020, for example, CTX001 received anorphan drug designationfor transfusion-dependent b-thalassemia.

In January 2020, the FDA provideddraft guidanceregarding orphan drug exclusivity for gene therapy products, whichincludesCRISPR/Cas gene editing (Draft Guidance). This guidance focuses on the analysis of whether two gene therapy products are the same under the Orphan Drug Act. Although informative, the limited scope of the Draft Guidance invites more questions than it answers.

Obtaining orphan drug exclusivity involves a two-step process. First, a sponsor requests designation of a drug for a particular rare disease or condition.See21 C.F.R. 316.20. If this drug is the same drug as a drug already approved to treat the same rare disease or condition, the sponsor must provide a plausible hypothesis that the new drug is clinically superior to the previously-approved drug.Id.Whether two drugs are the same depends on consideration of structural features relevant to that type of drug.See id. 316.3(b)(14).

If the new drug later obtains marketing approval for a use or indication within the rare disease or condition for which it received orphan drug designation, the FDA will determine if the drug is eligible for orphan drug exclusivity.See21 C.F.R. 316.31(a). In this situation, to receive exclusivity, the sponsor of the new drug must show that its drug is clinically superior to the same previously-approved drug for the same rare disease or condition.See id. 316.34(c). A clinical superiority determination is based on the new drugs greater efficacy, greater safety, or a major contribution to patient care.See id. 316.3(b)(3).

To determine whether one gene therapy product is the same as another, per 316.3(b)(14)(ii), the FDA will evaluate the principal molecular structural features of the two products, particularly transgenes (e.g., transgenes that encode different enzymes for treatment of the same rare disease) and vectors. For example:

If two gene therapy products express different transgenes, the FDA generally intends to consider them to be different drugs even if they have or use the same vector.

Conversely, if two gene therapy products have or use vectors from a different viral class (e.g., gammaretrovirus or adeno-associated virus), the FDA generally intends to consider them to be different drugs even if they express the same transgene.

In the case of two vectors from the same class (e.g., AAV2 or AAV5), the FDA intends to determine their same-ness on a case-by-case basis.

However, the FDA generally does not intend to consider these principal molecular structural features to be different based solely on minor differences between the transgenes and/or the vectors.

Additionally, [w]hen applicable, the FDA generally intends to consider additional features of the final gene therapy product, such as regulatory elements or, in the case of genetically-modified cells, the type of cell that is transduced. It generally intends to consider requests for designation and exclusivity of gene therapy products to evaluate whether such additional features may also be considered to be principal molecular structural features.

The Draft Guidance helps answer certain high-level questions relating to whether two gene therapy products would be considered the same under the Orphan Drug Act. As various stakeholders haverecognized, however, it is short on the details that meaningfully aid the process of drug research and development.

It is clear from the Draft Guidance that a new product can be considered the same as a previously-approved product even if the two products are not perfectly identical, but the guidance does not explain what would constitute a minor difference between such products, or what the scope of additional features would be.

For example, the Draft Guidance does not clarify what makes two transgenes the same. Nor does it cite to prior guidance or regulations that may answer this question. The question is significant becauseCas nucleasesand otherpartsof the CRISPR/Cas system may be modified in various ways. To address whether these modifications bar a finding of same-ness, the FDA could potentially import the kinds of considerations that govern same-ness of other kinds of large-molecule products, such as polynucleotide drugs or closely related, complex partly definable drugs with similar therapeutic intent (e.g., viral vaccines).See21 C.F.R. 316.3(b)(14)(ii)(C), (D). However, this is not clear from the Draft Guidance.

The Draft Guidance also does not explain what will factor into the case-by-case basis assessment of whether viral vectors from the same viral class are the same. In the case of AAV2 and AAV5the two viruses identified in the guidanceresearchers have foundthat these viruses differ with respect to sequence analysis, tissue tropism, and heparin sensitivity. It is not clear from the guidance, however, whether a plausible hypothesis of clinical superiority will be required to seek orphan drug designation for a drug based on AAV2 if the previously-approved drug expresses the same transgene(s) but is based on AAV5.

It would be beneficial to sponsors and other stakeholders if these aspects of gene therapy drugs sameness are clarified further before they invest significant resources into the design and development of these therapeutics.

2020 Proskauer Rose LLP. National Law Review, Volume X, Number 272

More:
Orphan Drug Exclusivity for CRISPR/Cas-Based Therapeutics - The National Law Review

LEIDEN, The Netherlands and SEVILLE, Spain, Sept. 25, 2020 /PRNewswire/ -- Amarna Therapeutics, a Dutch privately held biotechnology company developing the next-generation SV40-based gene delivery vector platform transforming gene-replacement and immunotherapy across many disease areas. The company today announced it has entered into a collaboration with scientists from the Progreso y Salud Foundation (FPS) at Cabimer in Seville, to jointly examine the efficacy of Amarna's SVecTMgene delivery vector to develop effective immunotherapies for diabetes mellitus type 1 (DM1) and multiple sclerosis (MS).

The collaboration is a joint effort between the research group of the FPS at research institute Cabimer, led by Dr. Benoit Gauthier, and Amarna Therapeutics, represented by Dr. Peter de Haan (CSO) and Miguel Garca Toscano (Head of Laboratory in Spain).

To date, the symptoms of DM1 and MS can be managed, but patients cannot be cured from both autoimmune diseases. The aim of this joint effort is to study the efficacy of Amarna's SV40-based gene delivery vector platform, denoted SVecTM, for downregulation of pathological immune responses that underlie the destruction of own cells in DM1 and in MS patients.

The research will focus on the induction of SVecTM-mediated immune tolerance to the primary self-antigens of both diseases. The studies will use advanced animal models of both autoimmune diseases, that have been established by the collaboration partners. Amarna will invest some 0.6 million over the next two years in the Gauthier research group to conduct the animal proof-of-principle studies for these two indications, for which at present there are no cures available.

Benoit Gauthier, Staff Scientist at Junta de Andalucia-Consejeria de Salud y Familias, comments:

"We are thrilled to start this new venture with Amarna Therapeutics, a world leader in viral gene therapy and we anticipate the studies to generate exciting results."

Peter de Haan, Amarna Therapeutic's Chief Scientific Officer, adds:

"We are delighted entering this collaboration with such a renowned academic partner like FPS and we look very much forward to initiate the planned studies. Since the quality of life for patients with DM1 and MS is so severely impaired given the lack of cures for these invalidating diseases, the more efficiently we can develop our groundbreaking SV40-based gene delivery vector basedtherapies, the sooner patients will experience the positive impact of our solution on their lives."

Contact details:

For further inquiries please contact: Amarna Therapeutics Steen Klysner, CEO E-mail: [emailprotected]

LifeSpring Life Sciences Communication, Amsterdam Lon Melens Tel: +31 6 538 16 427 E-mail: [emailprotected]

About FPS

For more than ten years, Benoit Gauthier's research group has focused on the field of diabetes and recently other autoimmune diseases. Its basic quality research has generated important new knowledge which enable the development of new therapies for this disease cluster. An important finding of the group was the mandatory association of immune responses to pancreatic beta cells with their capacity to regenerate in patients with type 1 diabetes. In addition, the discovery of the PAX8 gene, and the relationship between type 2 diabetes (T2D) and increased risk of pancreatic cancer, led to international recognition and generated numerous publications in peer-reviewed scientific journals.

The group is funded by different national and international public and private institutions, as well as from diabetes patient associations and supported by the Andalusian Government.

Amarna Therapeutics

Amarna Therapeutics is a privately held Biotech company founded in 2008. Its head office is located in Leiden (The Netherlands), and it also holds a research facility in Seville (Spain). The company has developed a proprietary production and gene therapy delivery platform in its SuperVeroTM cell line and SVecTM vector for the development of safe and efficient therapies. The company's pipeline targets several major indications as well as orphan diseases within the field of degenerative, inflammatory and autoimmune diseases. The company plans to take the first candidate from its pipeline into clinical development in 2021.

In October 2019, Amarna secured 10 million in new equity, with the aim of bringing the first product into clinical studies. The financing round was led by the Swedish Flerie Invest AB, together with existing shareholders and an innovation credit from the "Netherlands Enterprise Agency" (RVO.nl).

SOURCE Amarna Therapeutics

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Dutch Amarna Therapeutics enters research collaboration with Spanish FPS, examining the efficacy of its SV40-based SVec gene delivery vector platform...

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Key Market Insights

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Gene Therapy Market is projected to be worth USD 10 Billion by 2030 - The Daily Chronicle

Axovant Gene Therapies (AXGT) is a gene therapy company focused on treating neurodegenerative diseases, including Parkinson's Disease. Despite being in early clinical stages and being relatively underfunded, in my opinion, at today's valuation, it offers a very interesting risk/reward proposition. I explain why in this article.

(source)

AXGT's premier drug under development is AXO-Lenti-PD aimed at treating Parkinson's Disease. The drug is a re-engineered version of an earlier gene therapy developed by Oxford BioMedica (OTCPK:OXBDF) under the name "ProSavin". The re-engineered version is thought to improve gene payload and delivery, but because the same genes are being delivered, the data from ProSavin will also be included in any eventual FDA submission.

As Parkinson.org reminds us, "Parkinsons disease (PD) is an extremely diverse disorder. While no two people experience Parkinsons the same way, there are some commonalities. PD affects about one million people in the United States and ten million worldwide. The main finding in brains of people with PD is loss of dopaminergic neurons in the area of the brain known as the substantia nigra."

AXO-Lenti-PD's mechanism of action is to deliver three genes (TH, CH1 and AADC) involved in converting tyrosine and levodopa into dopamine. By increasing dopamine in the brain, AXGT hopes to reverse the symptoms of Parkinson's in its patients.

(source)

(As an aside, the only direct gene therapy competition in the space that I'm aware of is a collaboration between Neurocrine Biosciences (NBIX) and Voyager (VYGR) whose treatment delivers only one of the three genes (AADC) to its patients.)

AXGT is using the Unified Parkinson's disease rating scale (UPDRS) motor score (subscale III) to assess improvements in Parkinson's symptoms. This is the most commonly accepted measure for the disease and to gage the effectiveness of treatments.

One JAMA meta study attempted to equate changes in UPDRS scores into levels of clinically important differences (CIDs). The results were as follows:

Concordance among multiple approaches of analysis based on subjective and objective data show that reasonable estimates for the CID on the UPDRS motor score are 2.5 points for minimal, 5.2 for moderate, and 10.8 for large CIDs.

The JAMA study is helpful in assessing AXGT's early results.

AXGT treated 15 patients at three dose levels in the initial ProSavin trial. The data showed dose dependence and the overall improvements in UPDRS-III scores would have ranked the two higher dose groups as having "large" clinically important differences according to the JAMA researchers.

Six patients have been treated with AXO-Lenti-PD at two dose levels, with results having been attained for the first two treated at the lower dose and partial results for one patient in the higher dose group.

(source)

The initial results are an improvement over the original ProSavin data and overall the potential for this treatment is tantalizing, given the expected deterioration of patients on today's standard of care (levodopa). Compare the timeline of AXGT's treatment results to an older graph showing the 40 week data for levodopa (note that the second graph is total score, not subsection III as I wasn't able to find exactly comparable data). For a deeper dive, see this NCBI paper titled "Progression of motor and nonmotor features of Parkinson's disease and their response to treatment".

(source)

Changes in Total Scores on the Unified Parkinson's Disease Rating Scale (UPDRS) from Baseline through Evaluation at Week 42.

The changes in subjects treated with levodopa at different doses or with placebo were determined on the basis of the total scores on the UPDRS. (source)

The initial market for AXGT's gene therapy is on the order of 100K patients. Given that it's a one time treatment, charging $75K to $100K wouldn't be unreasonable. That's a market opportunity of $7.5B to $10.0B should everything work out.

(source)

However Oxford BioMedica didn't exactly give the technology away, such that the licensing agreement allows Oxford to materially participate in any future success AXGT may have with this gene therapy. From the most recent 10Q (with my emphasis):

In June 2018, we, through our wholly owned subsidiary, ASG, entered into the Oxford Agreement, pursuant to which we received a worldwide, exclusive, royalty-bearing, sub-licensable license under certain patents and other intellectual property controlled by Oxford to develop and commercialize AXO-Lenti-PD and related gene therapy products for all diseases and conditions. In June 2018, as partial consideration for the license, we made an upfront payment to Oxford of $30.0 million, $5.0 million of which was applied as a credit against the process development work and clinical supply that Oxford is obligated to provide to us over the term of the Oxford Agreement. Under the terms of the Oxford Agreement, we could be obligated to make payments to Oxford totaling up to $55.0 million upon the achievement of specified development milestones and $757.5 million upon the achievement of specified regulatory and sales milestones. In April 2019, certain development milestones were achieved resulting in a $13.0 million net payment due to Oxford. We will also be obligated to pay Oxford a tiered royalty from 7% to 10%, based on yearly aggregate net sales of the underlying gene therapy products, subject to specified reductions upon the occurrence of certain events as set forth in the Oxford Agreement. These royalties are required to be paid, on a product-by-product and country-by-country basis, until the latest to occur of the expiration of the last to expire valid claim of a licensed patent covering such product in such country, the expiration of regulatory exclusivity for such product in such country, or 10 years after the first commercial sale of such product in such country.

Personally I think that these high costs validate the potential of the treatment, but they of course also limit the benefit AXGT will see from it successful deployment.

Before looking at the company's valuation and the risk/reward it presents, let's quickly look at the company's other two product candidates which are aimed at treating rare neurological diseases.

This gene therapy is intended to treat Gangliosidosis, a very rare but fatal pediatric disease for which there is currently no treatment. Initial 6 month data from the Stage 1 low dose cohort is expected some time next quarter.

(source)

This gene therapy is a dual vector program aimed at delivering HEXA and HEXB genes into patients with GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease).

(source)

There is initial data from two patients, with the second one showing some promise. From the most recent 10Q:

Patient #2

In June 2019, a six month old child with early symptomatic infantile Tay-Sachs disease received AXO-AAV-GM2 prior to the onset of severe symptoms, delivered into the thalamus bilaterally as well as into the cisterna magna and lumbar intrathecal space, the planned routes of administration for patients in the registrational program. The surgical procedure was well tolerated with no neurological defects noted. There were baseline elevated transaminases noted with a transient increase. This child is clinically stable at six months after dosing with plateaued development. Importantly, no seizure activity and no exaggerated startle responses were observed. By contrast, the patients untreated, two older siblings with Tay-Sachs disease exhibited rapid disease progression, clinical regression and seizure onset at 12 to 18 months of age. In addition, brain MRIs taken three and six months after administration (at 10 and 13 months of age, respectively) demonstrated no damage to the thalamus and normal new myelin deposition. By contrast, commonly reported MRI findings in infantile Tay-Sachs disease at this age include demyelination and cerebral and cerebellar atrophy. The CHOP INTEND score, a 16-item scale of motor function that has been validated in infants with neuromuscular disorders, was 58 out of 64 at baseline, increasing to a total score of 60 at month three following gene transfer and declining to a total score of approximately 52 at month 12 following gene transfer. Total CHOP INTEND scores sustained at levels greater than 40 points indicate a clinically meaningful improvement.

With the stock trading at $4.72, AXGT sports a market cap of $198M and an EV of $143M thanks to the $55M it had as cash on the balance sheet at the end of June.

(source)

For a company with a multi-billion dollar opportunity in front of it, these seem to be low valuations.

The risk however is continued dilution and/or failed or stalled trials which would mean that the company either doesn't succeed or the share count is much higher by the time the company is generating revenues. Indeed, despite having $55M in cash on the balance sheet, the company is operating under a going concern warning.

From the 10Q with my emphasis:

As of June 30, 2020, the Companys cash and cash equivalents totaled $55.5 million and its accumulated deficit was $767.2 million. For the three months ended June 30, 2020 and the fiscal year ended March 31, 2020, the Company incurred net losses of $8.6 million and $72.6 million, respectively. The Company expects to continue to incur significant operating and net losses, as well as negative cash flows from operations, for the foreseeable future as it continues to develop its gene therapy product candidates and prepares for potential future regulatory approvals and commercialization of its products, if approved. The Company has not generated any revenue to date and does not expect to generate product revenue unless and until it successfully completes development and obtains regulatory approval for at least one of its product candidates, and its current cash and cash equivalents balance will not be sufficient to complete all necessary development activities and commercially launch its products. The Company anticipates that its current cash and cash equivalents balance will not be sufficient to sustain operations beyond nine months following the date that these unaudited condensed consolidated financial statements and notes were issued, which raises substantial doubt about the Company's ability to continue as a going concern.

The company also has an ATM in force, from the 10Q:

During the three months ended June 30, 2020, the Company engaged SVB Leerink LLC as its agent to sell the Company's common shares from time to time through an at-the-market equity offering program. SVB Leerink LLC receives compensation for its services in an amount equal to 3% of the gross proceeds of any of the Company's common shares sold. As of June 30, 2020, the Company sold approximately 1.4 million common shares for total proceeds of approximately $4.3 million, net of brokerage fees, under this program, and subsequent to June 30, 2020, the Company has sold approximately 1.1 million common shares for total proceeds of approximately $3.3 million, net of brokerage fees (see Note 12).

AXGT has a promising gene therapy for Parkinson's Disease which will have more safety and efficacy data released next quarter. Should the data continue to substantially improve on current standard of care, I think investors will begin to take into account the huge potential market opportunity and the stock will rise impressively despite the risks mentioned above. With this in mind, I may take a starter position in the near future, but in doing this research I've also become intrigued by the idea of using OXBDF as a way to participate without taking the full risks that AXGT engenders. I will write up my findings if I do decide to go that way.

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Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in AXGT over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: I actively trade around core positions.

Originally posted here:
Axovant Offers An Interesting Risk-Reward Proposition - Seeking Alpha