Archive for the ‘Gene Therapy Research’ Category

Among the logistical challenges facing public health agencies that are struggling to vaccinate the masses against COVID-19 is that the two mRNA shots on the market, from Moderna and Pfizer, need to be stored at ultra-cold temperatures. Now, an alternative technology for shielding patients from the novel coronavirusone that doesnt pose that storage challengeis showing early promise.

Two vaccine candidates built from gene-therapy technology and developed by Mass General Brigham scientists elicited strong immune responses in mouse and nonhuman primate models, the researchersreported on the journal preprint site bioRxiv. The team received a grant of up to $2.1 million from the Bill & Melinda Gates Foundation to further develop the vaccine technology, called AAVCOVID.

The vaccines, which remain stable when stored at room temperature, use an adeno-associated virus (AAV) vector to deliver genetic sequences of SARS-CoV-2, the virus that causes COVID-19. That generates antigens of the viruss signature spike protein, in turn prompting an immune response.

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A single injection of either of the AAVCOVID vaccine candidates induced neutralizing antibodies in one mouse model of obesity and another of agingtwo conditions that have been linked with poor COVID-19 outcomes. The response lasted for at least three months, according to the study. In the monkey models, the immune response lasted for five months.

All animals also showed memory T-cell responses, which are believed to be critical for maintaining long-term immunity to COVID-19.

RELATED: Biopharma tackles COVID-19, HIV and other viruses with gene and cell therapies

Although the demand for the mRNA vaccines from Pfizer and Moderna is strong, the shots present some hurdles. The need for cold storage will make distributing the vaccines in developing countries difficult. And both vaccines require two doses to confer full immunity, which only adds to the logistical challengesand the costthe Mass Gen researchers argued in their study.

Because AAVs are already widely used to deliver gene therapies like Novartis Zolgensma for spinal muscular atrophy, the Mass Gen team was able to ramp up its preclinical studies quickly. To accelerate the effort, the researchers formed early partnerships with gene therapy specialists at the University of Pennsylvania and with Novartis, which agreed to manufacture the AAVCOVID vaccines for clinical trials.

The fact that theres an established industry out there around AAV made it easy for us to step into the existing [manufacturing] capacity, rather than having to build it, said lead investigator Luk Vandenberghe, Ph.D., an associate professor at Harvard Medical School and director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear, in an interview with Fierce Biotech last year.

Vandenberghes team is now planning to start clinical trials of the vaccines overseas.

The researchers acknowledged in the study that several other vaccine candidates are nearing approval. But they believe the AAVCOVID shots will prove valuable because of other attributes that will likely be critical to achieving the desired long-lasting herd immunity at a global population scale, they wrote. Many of these considerations are logistical in nature and seek to reduce the cost, time, and complexity of vaccine distribution using available infrastructure around the world.

Excerpt from:
One-dose COVID-19 vaccine candidate that can be stored at room temperature prompts immunity in animals - FierceBiotech

Dr. Li has over 30 years of experience in basic and applied biomedical research. She joins Neurophth from the University of Louisville School of Medicine, where she was a professor in the department of Ophthalmology and Visual Sciences for over 14 years. Her research focuses on the role of Hippo/YAP1 signaling pathway on different stages of ocular development, NF-kB/IKK2 inhibition of neovascularization, and gene discovery screening for eye diseases using mouse models.

Throughout her career, Dr. Li has contributed to more than 45 publications in journals including Investigative Ophthalmology & Visual Science (IOVS), Proceedings of the National Academy of Sciences of the United States of America(PNAS), Nature Review Immunology, and Science. She is currently the editorial board member of Scientific Reportsand Source Journal of Ophthalmology. Dr. Li holds a Ph.D. in cell biology from the Washington University in St. Louis and obtained both her Bachelor's and Master of Science degrees in Genetics from Beijing University.

"We are thrilled to have Dr. Li on our team, bringing over 3 decades of her diverse experience in basic and applied biomedical research," said Bin Li, M.D., Ph.D., Founder and Chairman of the Board of Neurophth. "Given her prior experience at Baylor College of Medicine mentored by Dr. Savio Woo, an internationally recognized expert in molecular human genetics and gene therapy, and Dr. Mark Kay, a leading researcher in the fields of AAV gene therapy and the current Head of Division of Human Gene Therapy at the Stanford University School of Medicine, Dr. Li has extensive knowledge in gene therapy for hepatic deficiencies, ocular diseases, and viral vector reconstruction."

"We are excited to have Qiutang join and expand our exceptional research and development team. She brings a wealth of experience in gene therapies for ocular diseases to Neurophth," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer at Neurophth. "Her deep understanding of viral vector design and animal models in the inhibition of neovascularization for ocular diseases, such as age-related macular degeneration and diabetic retinopathy, further bolsters our ability to deliver on our growing pipeline of clinical programs and platform capabilities."

"It has been captivating to watch the scale, scope, and speed with which Neurophth has successfully transformed itself into an innovative and diversified gene therapy company," said Dr. Li. "I look forward to being a part of Neurophth team as the company executes the next stage of its growth strategy and expands its pipeline of gene therapy candidates focused on ocular and non-ocular diseases, building a brighter future for patients worldwide."

About Neurophth

Neurophth is China's first gene therapy company in ophthalmic diseases.Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou, and the U.S., Neurophth, a fully integrated company, is striving to discover and develop gene therapies for patients suffering from blindness and other eye diseases globally. Our AAV validated platform which has been published in Nature - Scientific Reports, Ophthalmology, and EBioMedicine, successfully delivered proof-of-concept data with investigational gene therapies in the retina. Our most advanced investigational candidate, NR082 (rAAV2-ND4), in development for the treatment ofND4-mutated LHON, has received orphan drug designations in theU.S. The pipeline also includesND1-mutated LHON, autosomal dominant optic atrophy, glaucoma, wAMD/DME, and other preclinical candidates. Neurophth has initiated the scaling up in-house process in single-use manufacturing technologies to support future commercial demand at the Suzhou facility. To learn more about us and our growing pipeline, visitwww.neurophth.com.

SOURCE Neurophth Therapeutics, Inc.

http://www.neurophth.com

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Neurophth Therapeutics Further Expands Ocular Gene Therapy Expertise with Appointment of Qiutang Li, Ph.D., as Chief Scientific Officer - PRNewswire

PARIS & CAMBRIDGE, Mass.--(BUSINESS WIRE)--NANOBIOTIX (Paris:NANO) (NASDAQ:NBTX) (Euronext: NANO NASDAQ: NBTX the Company), a clinical-stage biotechnology company focused on developing first-in-class product candidates that use proprietary nanotechnology to transform cancer treatment, today announced a new collaboration agreement secured by its wholly-owned subsidiary, Curadigm.

Pursuant to the selection of a project involving Curadigms Nanoprimer technology as a promising option to significantly improve gene therapy development, Curadigm will enter into a one-year agreement with the pharmaceutical company inclusive of direct funding and scientific exchanges. The goal of the project is to establish proof-of-concept for the Nanoprimer as a combination product that could improve treatment outcomes for gene therapy product candidates.

Many promising nucleic acid-based therapeutics administered intravenously are limited in their efficacy due to rapid clearance in the liver, which prevents these therapies from reaching the necessary accumulation in target tissues to generate their intended outcomes. Additionally, accumulation in the liver, rather than in the target tissues, can lead to dose-limiting hepatic toxicity.

The Nanoprimer is proprietary technology invented at Nanobiotix and licensed to Curadigm for development and commercialization. The Nanoprimer is designed to precisely and temporarily occupy therapeutic clearance pathways in the liver. Delivered intravenously, immediately prior to the recommended therapy, the technology acts to prevent rapid clearancethereby increasing bioavailability and subsequent accumulation of therapeutics in the targeted tissues.

Given that the Nanoprimer is a combination product candidate that does not alter or modify the therapies it is paired with, Nanobiotix expects that the team at Curadigm will continue to seek partnerships across drug classesparticularly with RNA-based therapies. The Company believes that the ongoing expansion of Curadigms development pipeline could create new pathways for the significant improvement of treatment outcomes for patients around the world.

***

About CURADIGM: http://www.curadigm.com

Curadigm, a Nanobiotix SA subsidiary, is an early-stage nanotechnology company dedicated to improving outcomes for patients by shifting the therapeutic delivery paradigm. Curadigms Nanoprimer platform is designed to increase drug bioavailability while decreasing unintended off-target effects, specifically liver toxicity. The platform can be used with most intravenous (IV) therapeutics across multiple drug classes. Curadigm is dedicated to advancing therapeutic development based on our deep understanding of how drugs interact with the body, to impact both known and novel drugs across multiple clinical indications.

About NANOBIOTIX: http://www.nanobiotix.com

Incorporated in 2003, Nanobiotix is a leading, clinical-stage nanomedicine company pioneering new approaches to significantly change patient outcomes by bringing nanophysics to the heart of the cell.

The Nanobiotix philosophy is rooted in designing pioneering, physical-based approaches to bring highly effective and generalized solutions to address unmet medical needs and challenges.

Nanobiotixs novel, potentially first-in-class, proprietary lead technology, NBTXR3, aims to expand radiotherapy benefits for millions of cancer patients. Nanobiotixs Immuno-Oncology program has the potential to bring a new dimension to cancer immunotherapies.

Nanobiotix is listed on the regulated market of Euronext in Paris (Euronext: NANO / ISIN: FR0011341205; Bloomberg: NANO: FP) and on the Nasdaq Global Select Market (Nasdaq: NBTX). The Companys headquarters are in Paris, France, with a U.S. affiliate in Cambridge, MA, and European affiliates in France, Spain and Germany.

About Sanofi: http://www.sanofi.com

Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe. Sanofi, Empowering Life.

Disclaimer

This press release contains certain forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as at this time, anticipate, believe, expect, intend, on track, plan, scheduled, and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management, include statements about the timing and progress of Curadigms development program, the prospects of Curadigms collaboration with Sanofi, the timing of proof-of-concept data, the potential of Curadigms platform to achieve clinical and commercial success, and Curadigms relationship with, and the performance of, its collaboration partners. Such forward-looking statements are made in light of information currently available to us and based on assumptions that Nanobiotix considers to be reasonable. However, these forward-looking statements are subject to numerous risks and uncertainties, including with respect to the duration and severity of the COVID-19 pandemic and governmental and regulatory measures implemented in response to the evolving situation. Furthermore, many other important factors, including those described in our prospectus filed with the U.S. Securities and Exchange Commission on December 11, 2020 under the caption Risk Factors and those set forth in the universal registration document of Nanobiotix registered with the French Financial Markets Authority (Autorit des Marchs Financiers) under number R.20-010 on May 12, 2020 (a copy of which is available on http://www.nanobiotix.com), as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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Nanobiotix Subsidiary Curadigm Secures New Collaboration Agreement With Sanofi Focused on Gene Therapy Pipeline - Business Wire

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space.

Cutting edge is, for once, a truly apt description when it comes to gene editing both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome.

This has, of course, garnered immense excitement Doctors Emmanuelle Charpentier and Jennifer Doudna were named winners of the Nobel Prize for chemistry in recognition of their discovery of CRISPR/Cas9 gene editing technology.

Since that discovery, a flurry of gene-editing focused biopharma companies have launched including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences and the first drug therapies based on the technology are now in human testing for diseases like cancer.

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntingtons disease and Amyotrophic lateral sclerosis (ALS).

But when I spoke to CEO Sandy Macrae during the JP Morgan Health Care Conference 2021, he stressed that companies cannot be successful in the area unless they are wise about the hype, and understand that focusing purely on in vivo editing is unlikely to be financially viable for some time.

Zinc fingers

Macrae had previously worked at GSK and Takeda before he was recruited by Sangamo.

Maybe in 50 years time well be using gene editing to lower cholesterol, but it wont replace statins in anyone but those with life threatening mutations for a long time

They wanted someone who had lots of experience in drug development, was a molecular biologist, and was stubborn enough to take on CRISPR! he jokes.

Since Macrae joined the company just four years ago, Sangamo has more than tripled its staff and raised $1.6 billion in funding. It has also built its own manufacturing site and launched partnerships with six big pharma companies.

This growth reflects the continued and increasing interest in gene therapy and with stock prices rising for editing companies across the board, Macrae says there has never been a more interesting time to be in genomic medicine.

When I started in 2016 it was still a very academic field without much industrial interest. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.

And Macrae notes that we still dont even know the full potential for the field.

At the moment its mostly being applied to ultra-rare diseases. That can be incredibly effective, but it doesnt allow for a sustainable business model. Thats why companies like ours have decided to move into larger unmet medical needs such as transplant, multiple sclerosis, or inflammatory bowel disease.

The companys primary technology is its zinc finger (ZF) platform. ZFPs can be engineered to make zinc finger nucleases, or ZFNs, which are proteins that can be used to edit genomes by knocking select genes in or out to specifically modify DNA sequences.

ZFPs can also be engineered to make ZFP transcription-factors, or ZFP-TFs, which are proteins that can be used to regulate genomes by selectively increasing or decreasing gene expression.

Zinc fingers are the most common control gene in the body, Macrae explains. We can place them near the promoter of a gene and repress or upregulate it.

The exact mechanism depends on the disease in question. For example, the company is working on repressing promoter genes in tauopathies in collaboration with Biogen, but its partnership with Novartis is focused on upregulating genes related to autism, both leveraging the ZFP-TF platform.

The genomic medicine journey

Genome editing and regulation are still in their early stages, though, and Macrae says the fields evolution is likely to come in waves.

First of all it will be used for ultra-rare monogenic disease. Then itll be used for common monogenic disease, then polygenic disease or diseases where theres a genetic component. And ultimately we will be able to add genetic influences to diseases that dont have a genetic cause. Hypertension is one example there are probably 20-30 genes that control your hypertension, and perhaps one day well be able to identify which ones we can turn up or down.

Thats some way off, but it could be a whole new way of treating people.

That said, Macrae notes that the industry needs to be cautious about this hype.

We have to be thoughtful and prudent, because the worst thing that could happen is that gene editing is used in the wrong kind of patient, where theres a risk without a benefit. That would just slow the whole field down.

This is still a new area of medicine, and every company is realising that we dont always know as much about some of these rare diseases as we thought we did. Weve never had treatments for these conditions before, and now that we do we often find that we need to know a lot more about the physiology and the pathology of the disease than we imagined.

Many companies in this area tell wonderful stories about preclinical potential, but once youre in a clinical trial it doesnt matter how clever your science is what matters is whether the patient gets better, and because of that you really need to understand the potential risk.

Gene editing, he says, still has to go through a long journey to truly reach this potential.

That involves collecting as much safety data and uncovering as much about the benefit-risk profile as we can, Macrae says. The benefit-risk for a child thats going to die without treatment is unquestioned. The benefit for lowering your cholesterol, when there are other tools you can use, is more uncertain. We shouldnt go there until we have enough data to be sure that its safe.

Maybe in 50 years time well be using gene editing for things like that but while many patients might benefit from gene editing for lowering cholesterol, its not going to replace statins for anyone but those with life threatening mutations for a long time.

On top of this, there are the well-documented manufacturing challenges that come with such a new field.

I think weve all learnt that we need to spend more time earlier on in developing the industrial processes, Macrae says.

The call I get most often from headhunters is, Do you know anyone that can do manufacturing in cell therapy? The field has grown so rapidly that there are very few people with experience in it. There is also a shortage of manufacturing sites.

This is part of the reason Sangamo has built its own manufacturing site in California and is building a European site in France.

Owning your own fate in manufacturing is really important, says Macrae. The process of gene editing needs lots of care and attention, and were at an early stage of the science where we dont know all the answers. Thats why its so important to have your own people in-house who know how to do it well.

Pragmatic genomics

As such, while Sangamo strongly believes in the potential of in vivo genome editing and regulation, Macrae says that early on the company made a pragmatic decision that it shouldnt depend on the field becoming financially viable anytime soon, and required a near-term strategy that would bring in revenue and benefit patients.

That is why the company is also working on gene therapy and ex vivo gene-edited cell therapy.

If youre working in gene editing, you can also work in gene therapy, because you already know a lot about delivery, vectors, molecular biology etc., Macrae explains. So it seemed like a sensible decision for us to work on that while gene editing is still an evolving field.

The companys gene therapy pipeline now includes treatments for PKU, Fabry disease and hemophilia A (in partnership with Pfizer).

The next easiest area for the company to take on with its existing capabilities was ex vivo gene-edited cell therapy.

In this area, Macrae says he is most excited about the companys CAR-Treg platform, from its acquisition of French company TxCell.

Tregs travel to the site of the inflammation and release mediators to calm it. We can put our localising CAR onto the Treg, which takes it specifically where we want it to go. For example, for multiple sclerosis you can use a CAR that takes the Treg to the myelin sheath.

You dont need to know the cause of the disease, you just need to know where the disease is.

Sangamo still anticipates, though, a time when in vivo genome editing and regulation is just as key to the business as these other two pillars and in fact Macrae anticipates that over time, Sangamo will shift its development focus to genome engineering as the field and science mature.

Gene therapy can ultimately only take you into the liver, he explains. There are 7,000 liver diseases, and only 10-20 of them that are big enough to run large clinical trials. Most of them are rare mutations.

Everyone is going to the liver and doing the same disease, and what was already a small population gets sliced and diced between several companies. We therefore dont see it as a long-term sustainable opportunity.

We have the advantage of also being able to edit cells in vivo, and eventually we will be able to do fundamental once-and-done editing in other tissues. Its just a matter of getting the field there.

About the interviewee

Sandy Macrae has served as Sangamos president and chief executive officer and as a member of the Board of Directors since June 2016. He has twenty years of experience in the pharmaceutical industry most recently serving as the global medical officer of Takeda Pharmaceuticals. From 2001 to 2012, Dr Macrae held roles of increasing responsibility at GlaxoSmithKline, including senior vice president, Emerging Markets Research and Development (R&D).

About the author

George Underwood is pharmaphorums Deep Dive magazine editor. He has been reporting on the pharma and healthcare industries for seven years and has worked at a number of leading publications in the UK.

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Gene editing: beyond the hype - - pharmaphorum

LAS VEGAS, Jan. 25, 2021 /PRNewswire/ -- The AAV Vector-Based Gene Therapy market size is anticipated to shoot up exponentially attributing to an increase in the approval of a growing number of gene therapies and readily adoption on approval, ability to treat a broad array of conditions, increasing prevalence of diseases, convenient one-time dosing approach and curative treatment options.

DelveInsight's Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Marketreport offers a clear picture of the market position of the AAV vectors in the Gene therapy, emerging pipeline therapies, AAV vector-based Gene Therapy market share occupied by individual diseases, current and forecasted market share in the 7MM (the US, EU5 (the UK, Germany, France, Italy and Spain) and Japan) for the study period 2017-30.

The Report highlights the drivers and constraints shaping the present AAV Vector-Based Gene Therapy marketalong with the unmet medical needs that offer opportunities to the key players to explore the underlying potential of the market.

Key Highlights of AAV Vector-Based Gene Therapy Market:

Know more about the report highlights @ Adeno-associated virus (AAV) Vector-Based Gene Therapy Market Landscape

Adeno-Associated Virus (AAV)

Adeno-associated virus hails from the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae. They are small viruses (25-nm) with a genome of single-stranded DNA (~4.7kb) that can either be the plus (sense) or minus (anti-sense) strand. AAV is replication-defective and depends on a helper virus for effective and productive replication in mammalian cells. However, for choosing AAV as a gene delivery vector depends on:

Adeno-associated virus (AAV) Vector-Based Gene Therapy

With advances in bioengineering and genetics, the horizon of the medical approaches giving rise to novel treatment options, such as Gene therapy has also widened. Gene therapyhas emerged out as a promising treatment approach for a number of inherited disorders, certain types of cancer, and certain viral infections. The delivery of gene therapy involves "vectors" which can be either viral or non-viral vectors. Out of the several viral vectors, Adeno-associated virus (AAV) vectors are currently among the most frequently used, safest and effective viral vectors for gene therapy delivery. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.

Key Indications:

Haematology

Ophthalmology

Lysosomal Storage Disorders

Neurology

Neurological Disorders

Musculoskeletal

Request for sample @ Adeno-Associated Virus Vector-Based Gene Therapy Market

Epidemiology Segmentation

In the year 2020, the total prevalent cases of selected indications in which AAV Gene Therapies were administered were estimated to be 2,863,103 in the 7MM. DelveInsight's Adeno-Associated Virus Vector-Based Gene Therapy MarketReport provides historical as well as forecasted epidemiological analysis for the study period 2017-30 for the 7MM segmented into:

Adeno-Associated Virus Vector-Based Gene Therapy Market

At the moment, the AAV Vector-Based Gene Therapy Markethas only two FDA-approved AAV vector-based gene therapies approved.

Spark Therapeutics' Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. It is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease and the first AAV vector gene therapy approved in the United States.

Another therapy occupying the market share is Novartis/AveXis's Zolgensma, an AAV-delivered gene therapy indicated for the treatment of paediatric patients <2 years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Currently, it is being investigated in the global Phase III STR1VE clinical program (consisting of STR1VE-US, STR1VE-EU, and STR1VE-AP) to evaluate the intravenous (IV) formulation of AVXS-101 in patients who have SMA Type 1, and the multinational Phase III SPR1NT trial in presymptomatic patients who have a genetic diagnosis of SMA with two or three copies of the SMN2 gene.

However, before Luxturna and Zolgensma managed to steal the spotlight in the AAV Vector-Based Gene Therapy Market,there was another candidate, Glybera, which got the nod from the EMA to treat an ultra-rare, hereditary lipoprotein lipase deficiency(LPLD); and became the first Gene therapy to get launched. However, the decision of the company to not renew its market authorization after it expired in October 2017 stresses on the cost of the maintenance and development of the gene therapies even if they prove to be outright cures.

DelveInsight estimates that the Adeno-Associated Virus Vector-Based Gene Therapy Market,is anticipated to pick up momentum as companies across the globe are thoroughly working toward the development of new AAV-gene therapies to treat a spectrum of diseases. Key pharmaceutical and biotech companies such as Biomarin Pharmaceutical, Roche (Spark Therapeutics), Sangamo, Pfizer among several others are involved in exploring the AAV vector-based gene therapy for a wide range of indications such as Hemophilia A and B, MPS, and others.

Visit, Adeno-Associated Virus Vector-Based Gene Therapy Market, for more information

Key Pipeline Therapies:

There is not a shred of doubt that clinical successes in AAV-mediated gene replacement have helped Adeno-Associated Viruses to get recognized as an ideal therapeutic vector for delivery of gene therapies. With two AAV vector-based gene therapies having won regulatory approval,their popularity as the predominant vectors that deliver genes of interest to target tissues with improved specificity, efficiency, and safety in the Gene therapy market further fuelled. However, the production and formulation of AAV products require specified conditions so as to ensure good stability and yield, and even after caution, some of its processing methods (filtration or lyophilization) may lead to aggregation or loss of AAV titer. Further, storing the AAV products can also prove to be quite challenging.

Conclusively, Gene therapy has proved to be an ingenious tool in the healthcare sector and with dramatic advancements being made in the domain, its potential is unfolding at a rapid pace. Thus, it is not wrong to say that Gene therapy is set to emerge as a novel therapeutic option.

Scope of the report:

Table of Content

1

Key Insights

2

Executive Summary of Adeno-Associated Virus Vectors in Gene Therapy

3

Competitive Intelligence Analysis for AAV Vector-Based Gene Therapy Market

4

AAV Vector-Based Gene Therapy Market SWOT Analysis

5

Adeno-Associated Virus Vector-Based Gene Therapy Market Overview at a Glance

6

Adeno-Associated Virus Vector-Based Gene Therapy: Disease Background and Overview

7

Patient Journey

8

Adeno-Associated Virus Vectors in Gene Therapy Epidemiology and Patient Population

9

AAV Vector-Based Gene Therapy Market: Treatment Algorithm, Current Treatment, and Medical Practices

10

AAV Vector-Based Gene Therapy Market Unmet Needs

11

Key Endpoints of Adeno-Associated Virus Vectors in Gene Therapy Treatment

12

Marketed Products in AAV Vector-Based Gene Therapy Market

13

Emerging Adeno-Associated Virus Vector-Based Gene Therapies

14

Adeno-Associated Virus Vectors in Gene Therapy: Seven Major Market Analysis

15

Attribute analysis

16

Adeno-Associated Virus Vector-Based Gene Therapy Market Outlook: 7MM

17

Access and Reimbursement Overview of Adeno-Associated Virus Vector-Based Gene Therapy Market

18

KOL Views

19

Adeno-Associated Virus Vector-Based Gene Therapy Market Drivers

20

Adeno-Associated Virus Vector-Based Gene Therapy Market Barriers

21

Appendix

22

DelveInsight Capabilities

23

Disclaimer

24

About DelveInsight

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Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market Expected to Grow at a Significant CAGR of 43.4% During the Study Period 2019-30 -...

Eric Pastor joins Spirovant Sciences as SVP of Technology Development and Operations.

PHILADELPHIA, PA, Jan. 26, 2021 (GLOBE NEWSWIRE) -- Spirovant Sciences, a gene therapy company developing treatments and cures for respiratory diseases including cystic fibrosis, todayannounced the leasing of its new, expanded headquarters and laboratories in the robust gene and cell therapy epicenter of Philadelphias University City in Wexford Science + Technologys uCity Square at 3675 Market Street. The Company also announced the appointments of Eric Pastor as Senior Vice President of Technology Development and Operations and Maria Limberis, PhD, as Vice President of Research.

In 2020, Spirovant advanced our pipeline and generated data that reaffirmed our vision to change the course of cystic fibrosis, said Joan Lau, Ph.D., Chief Executive Officer of Spirovant Sciences. As evidenced by our new team members Eric and Maria, we are focused on hiring leaders in gene therapy who share our values of collaboration, diversity, empowerment, and scientific rigor in our relentless pursuit of developing life-altering medicines for those in need. Additionally, our new, modern laboratories will energize the team in its pursuit to discover and develop advancements in the field.

In his new role, Eric Pastor brings more than 20 years of CMC and technical operations and experience will be responsible for expanding Spirovants technology development and manufacturing capabilities. He was most recently Vice President, CMC Operations, for Vedere Bio. Previously, Pastor served in a number of scientific positions at Sanofi, mostly recently as Head of Gene Therapy Development, as well as at AMRI and Targeted Genetics. He earned a Bachelor of Science degree at the University of Northern British Columbia.

Maria Limberis has more than 15 years of gene therapy research experience and will be responsible for applying Spirovants gene therapy vectors to therapy candidates. Previously, she was Associate Professor in the Department of Medicine and Director, Program of Excellence in Cystic Fibrosis in the Orphan Disease Center at the University of Pennsylvania, leading pre-clinical R&D and translational gene therapy programs in cystic fibrosis and airborne infectious diseases. She earned a PhD in molecular biology from the University of Adelaide.

The Company was assisted in its search for its new headquarters by Joseph Fetterman and Clifford Brechner of Colliers International.

About Spirovant Sciences, Inc.Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company's current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis. Spirovants lead programs are in development for cystic fibrosis. Spirovant is a wholly owned subsidiary of Sumitovant Biopharma Ltd., which is itself a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Spirovant is located inPhiladelphia, PA.More information is available athttps://www.spirovant.com/.

About Sumitovant BiopharmaLtd.Sumitovant is a global biopharmaceutical company with offices inNew York CityandLondon. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant and Urovant, and wholly owns Enzyvant, Spirovant andAltavant. Sumitovant'spipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need. For further information about Sumitovant please visithttps://www.sumitovant.com/.

About Sumitomo Dainippon Pharma Co., Ltd.Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies inJapan, operating globally in major pharmaceutical markets, includingJapan, the U.S.,Chinaand the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website athttps://www.ds-pharma.com/.

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Spirovant Continues Rapid Growth with New Headquarters and Expanded Leadership - GlobeNewswire

INDIANAPOLIS, Jan. 29, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) today announced financial results for the fourth quarter and full year of 2020.

$ in millions, exceptper share data

Fourth Quarter

%

Full Year

%

2020

2019

Change

2020

2019

Change

Revenue

$

7,440.0

$

6,114.0

22%

$

24,539.8

$

22,319.5

10%

Net Income Reported

2,116.8

1,495.7

42%

6,193.7

8,318.4

(26)%

EPS Reported

2.32

1.64

41%

6.79

8.89

(24)%

Net Income Non-GAAP

2,509.0

1,583.3

58%

7,235.9

5,568.2

30%

EPS Non-GAAP

2.75

1.73

59%

7.93

6.04

31%

Certain financial information for 2020 and 2019 is presented on both a reported and a non-GAAP basis. Some numbers in this press release may not add due to rounding. Reported results were prepared in accordance with U.S. generally accepted accounting principles (GAAP), include all revenue and expenses recognized during the periods, and reflect Elanco Animal Health (Elanco) as discontinued operations during the first quarter of 2019. Non-GAAP measures reflect adjustments for the items described in the reconciliation tables later in the release, and assume that the disposition of Elanco occurred at the beginning of 2019 (including the benefit from the reduction in shares of common stock outstanding). The company's 2021 financial guidance is being provided on both a reported and a non-GAAP basis. The non-GAAP measures are presented to provide additional insights into the underlying trends in the company's business.

"Lilly closed a complex year by delivering impressive results in the fourth quarter of 2020. We finished the year with strong momentum in our core business areas, as volume-based revenue growth for our newest medicines and initial sales of our COVID-19 antibody therapy, coupled with our ongoing productivity agenda, drove robust margin expansion and solid earnings growth," said David A. Ricks, Lilly's chairman and CEO. "I am also encouraged by exciting recent data readouts for three of our most important pipeline assets: tirzepatide, LOXO-305 and donanemab. Each of these potential medicines has a chance to significantly improve patient outcomes in areas of high unmet medical need, and, should they go on to receive approvals, reinforce our growth prospects for the decade ahead."

Key Events Over the Last Three Months

COVID-19

Regulatory

Clinical

Business Development/Other Developments

Fourth-Quarter Reported Results

In the fourth quarter of 2020, worldwide revenue was $7.440 billion, an increase of 22 percent compared with the fourth quarter of 2019, driven by a 24 percent increase in volume and a 1 percent increase due to the favorable impact of foreign exchange rates, partially offset by a 4 percent decrease due to lower realized prices. The company recognized worldwide revenue of $871.2 million in the fourth quarter of 2020 for bamlanivimab, its COVID-19 antibody therapy. Excluding bamlanivimab revenue, worldwide revenue grew by 7 percent. Key growth products launched since 2014, consisting of Trulicity, Verzenio, Taltz, Tyvyt, Olumiant, Jardiance, Emgality, Cyramza, Retevmo, Baqsimi and Basaglar contributed nearly 12 percentage points of revenue growth and represented approximately 48 percent of total revenue for the quarter, or 55 percent of total revenue excluding bamlanivimab.

Revenue in the U.S. increased 31 percent, to $4.598 billion, driven by a 36 percent increase in volume, partially offset by a 5 percent decrease due to lower realized prices. The company recognized U.S. revenue of $850.0 million in the fourth quarter of 2020 for bamlanivimab. Excluding bamlanivimab revenue, revenue in the U.S. grew by 7 percent. Increased U.S. volume for key growth products, including Trulicity, Taltz, Verzenio, Emgality, Retevmo, Cyramza, Olumiant, Jardiance and Baqsimi, was partially offset by lower volume for certain other products, including Forteo and Tradjenta. Inventory stocking in the fourth quarter of 2020 was approximately $120 million higher than in the fourth quarter of 2019 due to lower than typical year-end stocking in 2019. The decrease in realized prices in the U.S. in the fourth quarter of 2020 was primarily driven by increased rebates to gain and maintain broad commercial access across the portfolio, partially offset by modest list price increases, largely for diabetes, and, to a lesser extent, by changes to estimates for rebates and discounts, most notably for Taltz. Segment mix was not a major driver of U.S. price performance in the fourth quarter of 2020, as increased utilization in more highly-rebated government segments was offset by lower utilization in the 340B segment, primarily for Trulicity and Humalog.

Revenue outside the U.S. increased 10 percent, to $2.842 billion, driven by a 9 percent increase in volume and a 3 percent increase due to the favorable impact of foreign exchange rates, partially offset by a 2 percent decrease due to lower realized prices. The increase in volume outside the U.S. was driven primarily by increased volume for key growth products, including Tyvyt, Trulicity, Olumiant, Taltz, Verzenio, Jardiance, Cyramza, Basaglar, Emgality and Baqsimi, as well as volume gains for Alimta, partially offset by decreased volume for Cialis, Forteo and Trajenta. In addition, revenue outside the U.S. in the fourth quarter of 2019 benefited from a milestone from Bayer Consumer Care AG resulting from its exclusive development and commercialization license for Vitrakvi. The decrease in realized prices outside the U.S. was driven primarily by the inclusion of Tyvyt in the government reimbursement programs in China.

Gross margin increased 18 percent, to $5.720 billion, in the fourth quarter of 2020 compared with the fourth quarter of 2019. Gross margin as a percent of revenue was 76.9 percent, a decrease of 2.1 percentage points compared with the fourth quarter of 2019. The decrease in gross margin percent was primarily due to unfavorable product mix driven by bamlanivimab sales, higher amortization of intangibles expense related to Retevmo, the unfavorable effect of foreign exchange rates on international inventories sold, and the impact of lower realized prices on revenue, partially offset by greater manufacturing efficiencies.

Total operating expenses in the fourth quarter of 2020, defined as the sum of research and development and marketing, selling, and administrative expenses, increased 3 percent to $3.392 billion compared with the fourth quarter of 2019. Research and development expenses increased 16 percent to $1.838 billion, or 24.7 percent of revenue, driven primarily by approximately $265 million of development expenses for COVID-19 antibody therapies and baricitinib. Excluding these COVID-19 expenses, research and development expenses remained flat. Marketing, selling, and administrative expenses decreased 8 percent to $1.554 billion, primarily due to lower marketing expenses, reflecting reduced promotional activity.

In the fourth quarter of 2020, the company recognized acquired in-process research and development charges of $366.3 million related to the previously-announced business development transactions with Innovent Biologics, Inc., Disarm Therapeutics, Inc., and Fochon Pharmaceuticals, Ltd. There were no acquired in-process research and development charges in the fourth quarter of 2019.

In the fourth quarter of 2020, the company recognized income for asset impairment, restructuring and other special charges of $30.1 million, reflecting adjustments to prior period estimates for asset impairment and severance costs. In the fourth quarter of 2019, the company recognized asset impairment, restructuring and other special charges of $151.7 million. These charges were primarily related to the decision to close and sell a research and development facility located in the United Kingdom, as well as severance costs incurred as a result of actions taken to reduce the company's cost structure.

Operating income in the fourth quarter of 2020 was $1.992 billion, compared to $1.400 billion in the fourth quarter of 2019. The increase in operating income was primarily driven by higher gross margin, lower asset impairment, restructuring and other special charges, and lower marketing expenses, partially offset by higher acquired in-process research and development charges and higher research and development expenses. Operating margin, defined as operating income as a percent of revenue, was 26.8 percent.

Other income was $477.0 million in the fourth quarter of 2020, compared with other income of $262.9 million in the fourth quarter of 2019. The increase in other income was driven primarily by higher net gains on investment securities.

The effective tax rate was 14.3 percent in the fourth quarter of 2020, as compared with 10.1 percent in the fourth quarter of 2019. The effective tax rates for both periods were impacted by net discrete tax items.

In the fourth quarter of 2020, net income and earnings per share were $2.117 billion and $2.32, respectively, compared with net income of $1.496 billion and earnings per share of $1.64 in the fourth quarter of 2019. The increase in net income and earnings per share in the fourth quarter of 2020 was primarily driven by higher operating income and higher other income, partially offset by higher income tax expense.

Fourth-Quarter Non-GAAP Measures

On a non-GAAP basis, fourth-quarter 2020 gross margin increased 20 percent, to $5.848 billion compared with the fourth quarter of 2019. Gross margin as a percent of revenue was 78.6 percent, a decrease of 1.3 percentage points. The decrease in gross margin percent was primarily due to unfavorable product mix driven by bamlanivimab sales, the unfavorable effect of foreign exchange rates on international inventories sold, and the impact of lower realized prices on revenue, partially offset by greater manufacturing efficiencies.

Operating income on a non-GAAP basis increased $850.5 million, or 53 percent, to $2.456 billion in the fourth quarter of 2020 compared with the fourth quarter of 2019, due primarily to higher gross margin and lower marketing expenses, partially offset by higher research and development expenses. Operating margin was 33.0 percent on a non-GAAP basis.

The effective tax rate on a non-GAAP basis was 14.4 percent in the fourth quarter of 2020, as compared with 12.6 percent in the fourth quarter of 2019. The effective tax rates for both periods were impacted by net discrete tax items.

On a non-GAAP basis, in the fourth quarter of 2020 net income increased 58 percent, to $2.509 billion, while earnings per share increased 59 percent, to $2.75, compared with $1.583 billion and $1.73, respectively, in the fourth quarter of 2019. The increase in net income and earnings per share was driven primarily by higher operating income and higher other income, partially offset by higher income tax expense.

For further detail on non-GAAP measures, see the reconciliation below as well as the "Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information" table later in this press release.

Fourth Quarter

2020

2019

% Change

Earnings per share (reported)

$

2.32

$

1.64

41%

Acquired in-process research and development

.35

Amortization of intangible assets

.11

.05

Asset impairment, restructuring and other special charges

(.03)

.14

Gain on sale of China antibiotics business

(.26)

Charge related to repurchase of debt

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Lilly Reports Strong Fourth-Quarter and Full-Year 2020 Financial Results - BioSpace

Scientists have identified a protein that can slow or stop some signs of Parkinsons disease in mice.

The team found that the bone morphogenetic proteins 5 and 7 (BMP5/7) can have these effects in a mouse model of the disease.

This research, which appears in the journal Brain, may be the first step toward developing a new treatment for Parkinsons disease.

This type of brain disorder typically affects people over the age of 60, and the symptoms worsen with time.

Common symptoms include stiffness, difficulty walking, tremors, and trouble with balance and coordination.

The disease can also affect the ability to speak and lead to mood changes, tiredness, and memory loss.

Parkinsons Foundation report that about 1 million people in the United States had the disease in 2020, with about 10 million affected globally.

Despite this prevalence, scientists are still unsure why Parkinsons disease affects some people and not others, and there is currently no cure.

The National Institute on Aging note that some cases of Parkinsons disease seem to be hereditary. In other words, the disease can emerge in different generations of a family but for many people with the disease, there appears to be no family history.

Researchers believe that multiple factors may affect a persons risk, including genetics, exposure to environmental toxins, and age.

Since there is currently no cure for Parkinsons disease, treatments typically focus on alleviating its symptoms.

Existing treatments can help alleviate of Parkinsons disease, such as stiffness. However, they may work less well, or not work, for others, such as tremors or a loss of coordination.

Though researchers are still unsure why some develop the disease and others do not, they understand what occurs in the brain of a person with Parkinsons.

The disease causes the neurons in the part of the brain that controls movement to stop working or die. The brain region, therefore, produces less of the chemical dopamine, which helps a person maintain smooth, purposeful movement, as the National Institute of Neurological Disorders and Stroke observe.

Also, Lewy bodies occur in the brains of some people with Parkinsons disease. These bodies are clumps primarily made up of misfolded forms of the protein alpha-synuclein.

In their recent study paper, the scientists refer to research suggesting that neurotrophic factors molecules that help neurons survive and thrive could, in theory, restore the function of neurons that produce dopamine. However, the clinical benefit of these factors had yet to be proven.

The team focused on bone morphogenetic proteins 5 and 7 (BMP5/7). They had previously shown that BMP5/7 has an important role in dopamine-producing neurons in mice.

In the latest study, the scientists wanted to see whether BMP5/7 could protect the neurons of mice against the damaging effects of misfolded alpha-synuclein proteins.

To do this, they injected one group of mice with a viral vector that caused misfolded alpha-synuclein proteins to form in their brains. They used other mice as a control group. The scientists then injected the mice with the BMP5/7 protein.

The researchers found that the BMP5/7 protein had a significant protective effect against the misfolded alpha-synuclein proteins.

According to senior study author Dr. Claude Brodski, of the Israel-based Ben-Gurion University of the Negevs Department of Physiology and Cell Biology, We found that BMP5/7 treatment can, in a Parkinsons disease mouse model, efficiently prevent movement impairments caused by the accumulation of alpha-synuclein and reverse the loss of dopamine-producing brain cells. He continues:

These findings are very promising, since they suggest that BMP5/7 could slow or stop Parkinsons disease progression. Currently, we are focusing all our efforts on bringing our discovery closer to clinical application.

The universitys technology transfer company, BGN Technologies, is currently looking to bring the development to the market.

Dr. Galit Mazooz-Perlmuter, the companys senior vice president of bio-pharma business development, notes that There is a vast need for new therapies to treat Parkinsons disease, especially in advanced stages of the disease.

Dr. Brodskis findings, although still in their early stages, offer a disease-modified drug target that will address this devastating condition. We are now seeking an industry partner for further development of this patent-pending invention.

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Protein identified that may help treat Parkinsons disease - Medical News Today

Jan. 26, 2021 12:00 UTC

Timothy J. Douros, J.D., former General Counsel at Bluebird Bio, joins as Chief Legal Officer and Corporate Secretary, brings over 25 years of legal experience in the biotech industry

Tracy M. Porter, M.Ed., SPHR, former Head of Human Resources at Audentes Therapeutics, joins as Chief People Officer, brings nearly 30 years of human resources experience

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced new additions to its leadership team with the appointments of Timothy J. Douros, J.D., as Chief Legal Officer and Corporate Secretary and Tracy M. Porter, M.Ed., SPHR, as Chief People Officer. Mr. Douros will lead all aspects of the companys legal organization. Ms. Porter will oversee all aspects of human resources, including operations, talent acquisition and employee development.

We are thrilled to welcome Tim and Tracy to the Taysha leadership team, said RA Session II, President, Founder and CEO of Taysha. Tim brings significant gene therapy experience from Bluebird Bio where he led a global team, in cross functional collaboration with all corporate functions. His experiences include a wide range of legal and corporate matters which will be invaluable as we continue to advance our programs and evolve as a company. With her vast experience in strategic and operation roles in human resources, Tracy has successfully led and scaled global organizations quickly, most recently at Audentes where she helped grow the base of global gene therapy professionals by more than 50% in less than a year. Tracy will be instrumental in helping us continue to build a successful organization, scale thoughtfully and maintain our culture. Importantly, both Tim and Tracys experiences working in companies at various stages of the biotech life cycle will be essential to our future growth strategy.

Mr. Douros brings over 25 years of legal experience in biotech specializing in intellectual property, strategic licensing and contracting, litigation and dispute resolution, as well as international and healthcare compliance while building and managing high-performing teams. Prior to joining Taysha, he served as Senior Vice President, General Counsel at Bluebird Bio, Inc., where he led a global team of attorneys and staff and was a strategic partner to all internal clients, including business development, research and development, clinical operations and regulatory affairs. Prior to that, he was Vice President, Deputy General Counsel and Chief IP Counsel at Bluebird Bio where he provided counsel and managed all legal services related to the companys European infrastructure build to support product launches as well as provided counsel on all areas of compliance. Mr. Douros also led the corporate intellectual property function and managed all corporate dispute resolution matters. Before joining Bluebird, he spent nearly 13 years at Cubist Pharmaceuticals, Inc., holding positions of increasing responsibility, most recently serving as Vice President, Chief International Counsel & Chief IP Counsel. Mr. Douros received an A.B. in Chemistry from Dartmouth College and a J.D. from Boston College Law School.

I could not be more excited to join such an accomplished and energetic team of gene therapy trailblazers, said Mr. Douros. With its potentially de-risked approach to gene therapy development, Taysha is well-positioned to be a leader in developing potentially disease-modifying gene therapies for patients with monogenic CNS diseases and I look forward to contributing to the companys growth.

Ms. Porter joins Taysha with nearly 30 years as a human resources executive focused in entrepreneurial global business environments and specializing in pharmaceuticals and biotech. She has guided organizations from start-up and early-stage biotech to initial product launches and has provided human resources support through manufacturing expansion, acquisitions, divestitures, strategic business reorganizations and integrations. She most recently served as Vice President, Head of Human Resources at Audentes Therapeutics, where she developed, implemented and sustained its human capital strategy to rapidly scale and support the companys growth. Prior to joining Audentes, she spent time at Medivation Inc., and Bayer Healthcare LLC. Ms. Porter received a B.S. in Human Development and Counseling from the University of North Carolina at Greensboro and an M.Ed. in Global Human Resource Development from the University of Illinois at Urbana-Champaign.

Tayshas community of talented, diverse and passionate people is exceptional and I am honored to be a part of the team, said Ms. Porter. Taysha has a remarkable vision to develop and commercialize innovative therapies to potentially transform patients lives. I look forward to helping build the team and capabilities to realize that vision.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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Taysha Gene Therapies Continues to Add Significant Gene Therapy Expertise to Leadership Team with the Appointment of Chief Legal Officer and Chief...

Jan. 29, 2021 12:00 UTC

Program to screen for congenital, monogenic hearing loss in children diagnosed with auditory neuropathy

BOSTON--(BUSINESS WIRE)-- Decibel Therapeutics, a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced a partnership with Invitae, a leading medical genetics company, to launch AmplifyTM, a no-charge genetic testing program to screen for the genetic cause of congenital hearing loss in children diagnosed with auditory neuropathy.

We are pleased to collaborate with Invitae to introduce AmplifyTM, which is designed to bring patients one step closer to molecular diagnosis and clinical management of auditory neuropathy, a disorder that affects approximately 10 percent of children who are born with hearing loss, said Jonathon Whitton, Au.D., Ph.D., Vice President of Clinical Research at Decibel. This program seeks to provide much-needed answers to patients and families of patients who experience congenital, monogenic hearing loss. We hope that AmplifyTM will provide those patients with a better understanding of their diagnosis and their treatment options.

Auditory neuropathy is a hearing disorder in which the cochlea, the hearing organ located in the inner ear, receives sound normally, yet the transmission of sound to the brain is interrupted. The most common genetic cause of auditory neuropathy is insufficient production of a protein called otoferlin, which facilitates communication between the inner ear sensory cells and the auditory nerve. When this protein is lacking, the ear cannot communicate with the auditory nerve and the brain, resulting in profound hearing loss. Decibels lead investigational gene therapy program, DB-OTO, is designed to treat congenital, monogenic hearing loss caused by a deficiency in the otoferlin gene.

Amplify Program Eligibility

AmplifyTM is available to individuals who meet the following criteria:

AmplifyTM is a no-charge program that offers genetic testing for those who qualify. Although genetic testing can confirm a potential diagnosis, the absence of a genetic alteration does not preclude a diagnosis of genetic hearing loss. For more information about the program, please visit the Amplify program page.

About DB-OTO

DB-OTO is Decibels investigational gene therapy to restore hearing in children with congenital hearing loss due to a deficiency in the otoferlin gene. The program, developed in collaboration with Regeneron Pharmaceuticals, uses a proprietary, cell-selective promoter to precisely control gene expression in cochlear hair cells. DB-OTO is in preclinical studies, and Decibel expects to initiate clinical testing in 2022.

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website.

About Decibel Therapeutics

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibels pipeline, including its lead investigational gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow @DecibelTx.

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Decibel Therapeutics and Invitae Announce Launch of Amplify Genetic Testing Program - BioSpace

MIAMI, Jan. 25, 2021 (GLOBE NEWSWIRE) -- Ambulero, Inc., a biotechnology company developing cell and gene therapy treatments for patients suffering from vascular disease, today announced that it raised up to $5.5M in funding from Orphinic Scientific (Orphinic). As part of the investment, Ambulero and Orphinic formed a Polish subsidiary (Ambulero Sp. z o.o.) that will lead clinical testing of a novel gene therapy for a serious vascular disease in Europe. The disease is rare in the US but more common in certain parts of Central and Eastern Europe.

Ambulero holds an exclusive license from the University of Miami to develop and commercialize research from the laboratories of Drs. Omaida C. Velazquez and Jun Zhao-Liu. That research demonstrated in relevant animal models that an important cell adhesion molecule (E-selectin) can help promote vascular repair.

Ambulero is delighted to partner with Orphinic as they share our commitment to advance gene therapies for the benefit of patients suffering from vascular diseases, said Robert L. Buchanan, Co-founder and CEO of Ambulero.

We are excited to participate in a global development of Ambuleros breakthrough gene therapy that has a potential to reduce risk of limb ulcers and amputation in patients affected by vascular diseases, said Dr. Artur Plonowski, Partner and CMO at Orphinic.

About Ambulero

Ambulero is a biotechnology company dedicated to advancing a platform of cell and gene therapies to treat serious vascular diseases. The company is a 2019 spin-out of the University of Miami co-founded by Robert L. Buchanan, Randy Berholtz, Omaida C. Velazquez and Jun Zhao-Liu. Ambuleros cell therapy program uses stromal cells engineered to express E-selectin to promote tissue repair. The companys gene therapy program uses established approaches to administer E-selectin directly to injured tissues. Ambulero is financially backed by Ventac Holdings, LLC and is currently sourcing investors for a Series A round. See http://www.ambulero.com

About Orphinic With offices in Palo Alto, CA and Warsaw, Poland, Orphinic is an innovative drug development and investment company focused on mid-market opportunities and orphan drugs in late preclinical research and Phase 1/2. See http://www.orphinic.com

Follow Ambulero, Inc.Twitter: @ambulero1LinkedIn: http://www.linkedin.com/company/ambulero

ContactAmbulero, IncConverge Miami1951 NW 7th Street, STE. 600Miami, FL 33136Dr. Carlton AndersonChief Operation OfficerEmail: canderson@ambulero.com

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Ambulero Raises Up To $5.5 Million from Orphinic ScientificMiami Cell and Gene Therapy Company Opens European Subsidiary To Advance Gene Therapy For...

Those most famous of disruptive innovators, the team at ARK, has published its annual Big Ideas report for 2021, highlighting an extraordinarily broad range of themes detailing the latest developments in innovation and offering what it calls some of their most provocative research conclusions for the year.

First out of the box is Deep Learning, which the team believes could be the most important software breakthrough of our time.

Until recently, humans programmed all software. Deep learning, a form of artificial intelligence (AI), uses data to write software, the ARK team writes. By automating the creation of software, deep learning could turbocharge every industry. The ARK teams research shows that deep learning will add USD30 trillion to the global equity market capitalisation during the next 15-20 years.

Up next for causing huge change is the Reinvention of the Data Centre, according to ARK, which observes a revolution occurring in the sector. Cheaper, faster, and more power efficient processors are starting to displace Intelwhich traditionally had captured over 90 per cent of all processor revenue, the firm says. It believes that ARM, RISC-V, and graphics processing units (GPUs) are likely to emerge as the new powerhouse processors. Together they could scale at a 45 per cent annual rate to USD19 billion in revenue by 2030. In the data centre, we believe accelerators, dominated by GPUs, will become the dominant processors for new workloads, growing 21 per cent at an annual rate to USD41 billion by 2030.

The next big theme for ARK is Virtual Worlds, consisting of video games, augmented reality and virtual reality. ARK writes: A virtual world is defined as a computer-simulated environment that can be accessed by anyone at any time.

Society interacts daily with virtual worlds which today are in their infancy. According to our research, revenue from virtual worlds will compound 17 per cent annually from roughly USD180 billion today to USD390 billion by 2025. Today, virtual worlds are independent from each other, but in the future they could become interoperable, culminating in what futurists have deemed 'The Metaverse.

Next up is Digital Wallets, which, ARK says, could represent a USD4.6 trillion opportunity in your pocket, and upend traditional banking.

More overthrowing of the traditional comes in the firms predictions and observations on bitcoin - the firm estimates that if all S&P 500 companies were to allocate 1 per cent of their cash to bitcoin, its price would increase by approximately USD40,000.

ARK believes bitcoins rapid growth has positioned it for an allocation in investment portfolios. We believe bitcoin offers one of the most compelling risk-reward profiles among assets. As our analysis suggests, it could scale from roughly USD500 billion to USD1-5 trillion in network capitalisation during the next five to 10 years.

Electric vehicles are also part of the changing world for ARK which has famously supported Tesla throughout its volatile run. The firm says that electric vehicles are approaching sticker price parity with gas-powered cars.

Robotics and automation come next with ARK commenting that while the robots are coming they will help to create jobs. Autonomous ride hailing comes next with ARKs research suggesting that autonomous ride-hailing platforms will generate more than USD1 trillion in profits per year by 2030. In addition, automakers and fleet owners could enjoy profits of USD250 billion and USD70 billion, respectively. ARKs research on Drone Delivery, another big theme for disruptive innovation according to the firm, finds that drone delivery platforms will generate roughly USD275 billion in delivery revenues, USD50 billion in hardware sales, and USD12 billion in mapping revenue by 2030.

Orbital aerospace comes next with ARKs prediction that the orbital aerospace opportunity including satellite connectivity and hypersonic flight will exceed USD370 billion annually. Back on earth, 3D printing is another potential disruptor, with ARK estimating that 3D printing will revolutionise manufacturing, growing at an annual rate of roughly 60 per cent from USD12 billion last year to USD120 billion in 2025.

Long-Read Sequencing, that could provide a more complete picture of the human genome is also covered in the report, with ARK estimating that long-read revenues will grow 82 per cent at an annual rate, from USD250 million in 2020 to roughly USD5 billion in 2025.

Turning to health care, ARK believes that going forward, Multi-Cancer Screening using liquid biopsies could prevent more cancer deaths that any other medical intervention in history.

According to ARKs research, the convergence of innovative technologies has pushed the cost of multi-cancer screening down by 20-fold from USD30,000 in 2015 to USD1,500 today and it should drop another 80 per cent+ to USD250 in 2025.

As a result, the multi-cancer screening market should scale to USD150 billion in the US. A multi-cancer screening protocol could avert 66,000 cancer deaths per year in the US, saving 1.4 million human life years, the firm says.

Cell and Gene Therapy: Generation 2 is the final outing in the report, with ARK predicting that the second generation of cell and gene therapies could increase the total addressable market for oncology therapeutics by more than 20-fold.

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ARK's Big Ideas for 2021 lists the firm's 'most provocative research conclusions for the year' - ETF Express

This will address an accelerating gene therapy market that is expected to grow globally by 16.6 percent from 2020-2027.

"The Gene Therapy Ready network demonstrates our commitment to empowering sites and supporting our industry partners as they pursue advanced genetic engineering to find cures for the world's most pressing health conditions," said Scott Uebele, President and Chief Research Services Officer at Advarra. "Our commitment to efficient study activation is unwavering, and this is another example of how Advarra bringslife sciences companies,CROs, research sites, investigators,andacademiatogether at the intersection of safety,compliance,technology, and collaboration."

All Gene Therapy Ready sites stand ready to help industry sponsors conduct clinical trials that advance cures, develop vaccines, and find treatments for rare disease. By placing clinical trials with a Gene Therapy Ready site, research sponsors can save significant time during study startup.

"This innovative network is truly the first of its kind. We constantly look for ways to support our sponsors in rapidly starting trials in a safe, compliant, and quality manner. With the Gene Therapy Ready network, we can improve study startup times by a month or more, potentially placing cures in the hands of patients faster," said James Riddle, Vice President of Research Services and Strategic Consulting at Advarra. "The Gene Therapy Ready site network charts a course to success by providing our sponsor clients with a clear choice for IBC review services."

About Advarra

Advarra advances the way clinical research is conducted: bringing life sciences companies, CROs, research sites, investigators, and academia together at the intersection of safety, technology, and collaboration. With trusted IRB and IBC review solutions, innovative technologies, experienced consultants, and deep-seated connections across the industry, Advarra provides integrated solutions that safeguard trial participants, empower clinical sites, ensure compliance, and optimize research performance. Advarra is advancing clinical trials to make them safer, smarter, and faster. For more information, visit advarra.com.

SOURCE Advarra

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Advarra Announces New Gene Therapy Ready Site Network - PRNewswire

London, UK-based integrated cell and gene therapy company Ixaka formerly Rexgenero has launched with additional financing from existing shareholders totalling over 40m.

Ixaka is focused on developing cell therapies to treat serious diseases including cancer and chronic limb-threatening ischaemia (CLTI).

The company is continuing to develop its proprietary technology using concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

These technologies aim to bolster the naturally therapeutic power of cells by targeting curative cells at the site of disease, or by modifying cells within the body to improve disease targeting and boost their restorative function.

Ixakas lead MCT product REX-001 is in clinical development for the treatment of CLTI and is currently being evaluated in the Phase III SALAMANDER trial across multiple sites in Europe.

Ixakas broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies, said Joe Dupere, chief executive officer of Ixaka.

Our focus is now on accelerating progress to help realise the potential for durable and curative cell and gene therapies. By exploring multiple therapies across oncology and cardiovascular, genetic, neurological and autoimmune diseases, we are well positioned to bring life-changing treatments to multiple patient populations with critical unmet needs, he added.

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UK biotech Ixaka scores additional funding for cell and gene therapy research - PharmaTimes

NEW YORK--(BUSINESS WIRE)--Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, and University of Edinburgh, a world leader in biomedical and translational research for neurodevelopmental diseases, today announced a research collaboration to advance development of multiple platform approaches to enable next generation gene therapies.

The collaboration provides comprehensive research capabilities to Neurogene, enabling the company and the University to expedite a multiple platform approach to improve upon existing gene therapy technologies. Under the terms of the collaboration, Neurogene will provide financial support for Dr. Stuart Cobbs laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property at agreed-upon economic terms. Neurogene will be responsible for late stage preclinical and all clinical development of any products generated under the collaboration.

This partnership provides Neurogene with preeminent neurological research expertise and capabilities. Dr. Cobbs lab has contributed significant scientific expertise to improve the quality of our current rare disease pipeline and generated promising early data to allow us to tackle complex neurological diseases not addressable with conventional gene therapy, said Rachel McMinn, Ph.D., Founder and CEO of Neurogene. With this collaboration, I look forward to advancing our mission to provide safe and effective genetic therapies to patients and families as quickly as possible.

Neurogene is a science-driven company committed to investing in innovation and is the right partner for us to build upon the early successes in gene therapy technology, Stuart Cobb, Ph.D., Simons Fellow and Reader in Neuroscience at the Patrick Wild Centre and Centre for Discovery Brain Sciences, University of Edinburgh, stated. We are excited to collaborate with Neurogene on the critically-important endeavor of improving upon current gene therapy technologies. Gene therapy is a very promising yet complex development area, and we are privileged to help address the unmet needs that exist within rare neurological diseases.

In addition to Dr. Cobbs position at the University of Edinburgh, he serves as Chief Scientific Officer of Neurogene.

This Collaboration has been supported by Edinburgh Innovations, the University of Edinburghs commercialization service.

About Neurogene Inc.

Neurogene Inc. is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring therapies to patients that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options currently exist. Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address. For more information, visit http://www.neurogene.com.

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Neurogene and University of Edinburgh Announce Research Collaboration to Advance Next Generation Gene Therapies - Business Wire

SUZHOU, China, Jan. 18, 2021 /PRNewswire/ -- Cure Genetics announced a collaboration with Boehringer Ingelheim to develop novel Adeno-Associated Virus (AAV) vectorsleveraging Cure Genetics' proprietary VELPTM platform to develop next-generation gene therapies. This new collaboration combines Boehringer Ingelheim's experience in disease biology and gene therapy development with Cure Genetics' AAV expertise in library construction and highly efficient in vivo AAV screening. The aim is to provide potential new AAV serotypes for patients.

The clinical applications of existing AAV serotypes are limited by some of their features, such as low transduction efficiency, low tissue specificity and immunogenicity. Therefore, finding new AAV serotypes to overcome these challenges becomes critical for the majority, if not all, AAV-based gene therapies.

Comparing to other traditional vector engineering technologies, Cure Genetics' proprietary VELPTM platform encompasses key methodical innovations, including a comprehensive strategy of engineering a plasmid library with high complexity and an effective ratio. the optimized AAV production protocol ensures high genome-capsid correspondence and world-class production capacity, and the most physiologically relevant models for vector selection and validation. It enables a significantly shorter process to find the "right" AAV vectors with almost all possibility effectively covered.

Boehringer Ingelheim aspires to develop the next generation of medical breakthroughs and gene therapy is one of the focuses under exploration by the team of Research Beyond Borders. The advanced VELPTM technology platform may provide effective solutions in increasing the efficiency of novel AAV screening and help further expand our efforts in the area of gene therapy development.

"This is the very first time that a global pharmaceutical group is collaborating with a Chinese biotech in the cutting-edge field of AAV vector engineering. We appreciate the recognition of Boehringer Ingelheim's recognition of our VELPTM platform. Novel AAV vectors enlarging the therapeutic window is key to unfolding the potential of gene therapy, which is also Cure Genetics' innovative focus . We believe, together with visionary partners like Boehringer Ingelheim, the quality of life for more patients in need can be improved by next-generation gene therapy." stated Dr. Qiushi Li, Cure Genetics' Chief Operating Officer.

The collaboration with Cure Genetics was initiated by Boehringer Ingelheim China External Innovation Hub. It consists of three business units: Research Beyond Borders, Business Development and Licensing, and Venture Fund. The hub is committed to becoming the preferred partner of China's biopharmaceutical industry and bringing more Chinese innovative partnership projects to enrich Boehringer Ingelheim's global R&D pipeline, thereby ultimately benefiting more patients. So far, Boehringer Ingelheim China External Innovation Hub has established various partnerships with reputable research institutions and biotech companies in China.

About Cure Genetics

Cure Genetics is a biotech company founded in 2016, committed to expanding the frontier of gene therapy via its innovative technology of gene editing and gene delivery. With the world-leading AAV manufacturing capability, Cure Genetics' proprietary VELPTM platform enables a fast yet systematic design, selection and optimization of AAV vectors with special features and significantly better performance of in vivo gene delivery, which will empower AAV-based gene therapy to be applied in a much broader range of disease treatments.

View original content:http://www.prnewswire.com/news-releases/cure-genetics-collaborates-with-boehringer-ingelheim-to-develop-novel-aav-vectors-enabling-the-next-generation-liver-targeted-gene-therapy-301210036.html

SOURCE Boehringer Ingelheim; Cure Genetics

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Cure Genetics Collaborates with Boehringer Ingelheim to Develop Novel AAV Vectors Enabling the Next-generation Liver-targeted Gene Therapy - BioSpace

Jan. 20, 2021, FNF Research (fnfresearch.com) published the latest study on [2020-2026] Cancer Gene Therapy Market Report by Quantitative Research Incorporating Impact Of Economic And Non-economic Aspects was recently released. It uses exploratory techniques like qualitative and quantitative analysis to uncover and present data on the target market. Efficient sales strategies have been mentioned that would business and multiply customers in record time.

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Article content continued

They identified 100 genes out of around 10,000, and kat7 was the most efficient at contributing to senescence in cells, Qu said.

Kat7 is one of tens of thousands of genes found in the cells of mammals. The researchers inactivated it in the livers of the mice using a method called a lentiviral vector.

We just tested the function of the gene in different kinds of cell types, in the human stem cell, the mesenchymal progenitor cells, in the human liver cell and the mouse liver cell and for all of these cells we didnt see any detectable cellular toxicity. And for the mice, we also didnt see any side effect yet.

Despite this, the method is a long way from being ready for human trials, Qu said.

Its still definitely necessary to test the function of kat7 in other cell types of humans and other organs of mice and in the other pre-clinical animals before we use the strategy for human aging or other health conditions, she said.

Qu said she hopes to be able to test the method on primates next, but it would require a lot of funding and much more research first.

In the end, we hope that we can find a way to delay aging even by a very minor percentagein the future.

(Reporting by Martin Quin Pollard; Editing by Kim Coghill)

Continue reading here:
Chinese scientists develop new gene therapy that can delay the aging process - National Post

Reuters | Published: January 21, 2021 10:37:09 | Updated: January 21, 2021 14:27:09

Scientists in Beijing have developed a new gene therapy which can reverse some of the effects of ageing in mice and extend their lifespans, findings which may one day contribute to similar treatment for humans.

The method, detailed in a paper in the Science Translational Medicine journal earlier this month, involves inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing.

The specific therapy they used and the results were a world first, said co-supervisor of the project Professor Qu Jing, 40, a specialist in ageing and regenerative medicine from the Institute of Zoology at the Chinese Academy of Sciences (CAS).

These mice show after 6-8 months overall improved appearance and grip strength and most importantly they have extended lifespan for about 25%, Qu said.

The team of biologists from different CAS departments used the CRISPR/Cas9 method to screen thousands of genes for those which were particularly strong drivers of cellular senescence, the term used to describe cellular ageing.

They identified 100 genes out of around 10,000, and kat7 was the most efficient at contributing to senescence in cells, Qu said.

Kat7 is one of tens of thousands of genes found in the cells of mammals. The researchers inactivated it in the livers of the mice using a method called a lentiviral vector.

n liver cell and the mouse liver cell and for all of these cells we didnt see any detectable cellular toxicity. And for the mice, we also didnt see any side effect yet.

Despite this, the method is a long way from being ready for human trials, Qu said.

Its still definitely necessary to test the function of kat7 in other cell types of humans and other organs of mice and in the other pre-clinical animals before we use the strategy for human ageing or other health conditions, she said.

Qu said she hopes to be able to test the method on primates next, but it would require a lot of funding and much more research first.

In the end, we hope that we can find a way to delay ageing even by a very minor percentage...in the future.

Read more:
Gene therapy developed to delay ageing - The Financial Express

REGULATED INFORMATION

Clinical programs running on schedule, including Phase III trial of JTA-004 and Phase IIb trial of ALLOB

Extensive achievements in collaborations and partnerships to enhance development, manufacturing and commercialization capabilities

Strong financial position following fundraising, licencing agreement and optimisation of manufacturing assets

Gosselies, Belgium, 20January 2021, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today provides a business update for the fourth quarter, ending 31 December 2020 as well as a business outlook for 2021.

Bone Therapeutics managed to achieve a highly productive period in the last few months. We have made major advances in nearly all aspects of our activities while executing on our business strategy, said Miguel Forte, Chief Executive Officer, Bone Therapeutics. We recently started treating patients in the Phase IIb study with our allogeneic bone cell therapy product ALLOB. We also completed patient recruitment in the pivotal Phase III trial with the enriched viscosupplement, JTA-004, ready to provide topline data in the third quarter of this year. This will be a critical milestone in the history of Bone Therapeutics. In addition to our clinical programs, we have strongly improved our manufacturing capabilities with a partnership with Catalent, a leading global cell and gene CDMO, and extended the geographic reach of our bone cell therapies into Asia thanks to the licensing agreement with our Chinese partners Link Health and Pregene. We also have explored new grounds in the collaborations with Rigenerand and BioWin consortium. These collaborations will further expand the application of our innovative cell therapy platform and broaden our advanced clinical pipeline with potential new breakthrough developments. Building on the strong foundation of these recent achievements and a strengthened cash position, we are confident for 2021, continuing the progress we have already made and moving our allogeneic cell therapy and advanced biological products through clinical development while exploring new innovations that meet critical needs of patients.

Clinical highlights Q4 2020 to date

Corporate highlights Q4 2020 to date

Financial highlights Q4 2020 (1)

Outlook for 2021

Financial Calendar 2021

29 April Full Year Results & Annual Report 202026 May Q1 2021 Business and Financial Highlights9 June Annual General Meeting 20218 September Half Year Results 202126 October Q3 2021 Business and Financial Highlights

The financial calendar is communicated on an indicative basis and may be subject to change.

(1) Unaudited number

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in Phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid - a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive Phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics provides fourth quarter 2020 business update and 2021 outlook - GlobeNewswire

Large pharmaceutical companies have made gene therapy a priority with a series of acquisitions over the past several years, a stamp of validation for a field that's pushed through decades of ups and downs.

One of the latest buyers is German healthcare conglomerate Bayer, which in October inked a $2 billion deal for North Carolina gene therapy developer Asklepios Biopharmaceuticals, also known as AskBio.

For Bayer, the acquisition is part of a broader effort to build a gene and cell therapy division. But the deal is just as noteworthy for AskBio, an unusually large, privately held biotech based on the work of one of gene therapy's pioneers, Jude Samulski.

AskBio chose the security of a wealthy backer over independence and the chance to go public like several of its peers. And the deal helped the biotech quickly hire Katherine High, one of the few executives with experience shepherding a gene therapy through to regulatory approval. All of which makes the efforts of AskBio, now operating as an independent arm of Bayer, worth watching.

"I think we have the right people, the right chemistry, and the right amount of experience to make a difference here," Samulski said in an interview.

As 2019 drew to a close, so did a chapter in High's career. A hematologist by training, High, 69, has spent three decades working in gene therapy, a large portion of which was as a founder, president and chief scientific officer of Philadelphia biotech Spark Therapeutics.

At Spark, High had helped make history by steering the development of Luxturna, a treatment for a genetic form of blindness. When cleared by the Food and Drug Administration in late 2017, Luxturna became the first gene therapy for an inherited disease approved in the U.S. Roche swooped in soon after to acquire Spark, and closed the deal in December 2019.

Katherine High, president of therapeutics at AskBio

Permission granted by AskBio

High decided to take a year off from biopharma. But the coronavirus pandemic dashed her plans to conduct research at Rockefeller University. The institution reduced its staff to essential personnel, and the Harvard Club of New York City, where High, a Philadelphia resident, planned to stay during the week, closed its doors.

"My sabbatical turned into a virtual event, which was good; I got a lot of things done review articles written, book chapters written, things like that," High said in an interview. "I really needed a break."

She spent time with her first grandchild, swam, and, fulfilling a longtime desire, remotely studied German at Middlebury College's storied language program.

But High couldn't keep away from drug research. During periodic visits to North Carolina, where she has family, High dropped in on Samulski and fellow AskBio co-founder Sheila Mikhail.

High has over the years both collaborated and competed with Samulski, a University of North Carolina researcher and expert in gene therapy delivery tools known as adeno-associated viruses, or AAVs. He formed AskBio in 2001 with another gene therapy researcher, Xiao Xiao, and CEO Sheila Mikhail, a life sciences attorney.

"Our paths have crossed, our students have crossed, our sciences [have] definitely cross-pollinated," Samulski said, describing High's academic work at University of Pennsylvania and his at UNC.

By the time of their meeting, AskBio had grown to become one of the gene therapy field's most unique. Originally bootstrapped with angel investing and backing from the Muscular Dystrophy Association, AskBio had spun multiple gene therapy programs into companies that were later acquired. The returns from those buyouts were then used by AskBio to build its own manufacturing capabilities, a crucial step for gene therapy products.

During High's visits, Samulski and Mikail shared some of the progress the company had made advancing its technology. Among them: the acquisition of a Scottish biotech whose technology may allow the company to more tightly control how much protein a gene therapy can produce. Doing so could help overcome a critical limitations of gene therapy, which can have widely varying effects from patient to patient.

"We have a roadmap, how to get from A to B," Samulski told High. "If you want to come in and champion that, we would love to have you."

As AskBio was courting High, Bayer was eyeing AskBio, which had put in motion plans for an initial public offering a typical step for a biotech of its size.

Bayer had already announced plans to develop a cell and gene therapy division, acquiring Bluerock Therapeutics, a maker of "off-the-shelf" cell-based treatments, in 2019.

But the large pharma didn't have an anchor for its gene therapy ambitions. Marianne De Backer, Bayer's head of business strategy and development, had assembled a list of developers to pursue. AskBio was at the top.

"If you look at the [gene therapy] assets that are on the market today, like Zolgensma from Novartis, part of the technology is based on technology from AskBio," she said in an interview, referring to the Swiss company's spinal muscular atrophy treatment. A Duchenne muscular dystrophy treatment in late-stage testing at Pfizer also originated within AskBio, as did a Takeda program being studied in hemophilia.

De Backer faced two obstacles, though. Bayer, for one, didn't know the AskBio team, and couldn't meet them in person because of the travel restrictions that began during the pandemic.

"It was really almost a cold call," she said.

Bayer was also competing against the draw of a deep market for public stock offerings, which helped a record number of biotechs to IPO in 2020. De Backer said she needed to show AskBio that she could get the deal done quickly. So she and Mikhail spent six weeks hammering out terms, including an agreement the company could continue to operate independently an "arm's length" arrangement like one Bayer made with BlueRock.

Such promises are often made, and eventually broken, when a larger company acquires a smaller one. But Samulski's concerns that AskBio's work might be stifled within such a massive company were eased after speaking with BlueRock CEO Emile Nuwaysir.

Jude Samulski, co-founder and chief scientific officer at AskBio

Permission granted by AskBio

"[Nuwaysir] said, they have left me alone, they've encouraged me to do what I'm doing,' and I said, OK, that's what I needed to hear,'" Samulski said.

The acquisition allows the company to spend more time on science and less on raising money, he added.

"If I go back and write a grant today, it'll be three years before we can start the project," Samulski said. "In this setting, when we have our meeting ... the decision-makers are at the table and the science starts that afternoon."

For High, AskBio represents a return to a similar role as the one she had left: helping run an advanced gene therapy business newly acquired by big pharma. At AskBio, she's been named president of therapeutics.

The role, however, lines up with High's current career ambitions. AskBio has the manufacturing capabilities, breadth of clinical-stage programs and financial backing to take on diseases like Parkinson's and congestive heart failure the types of complex, common conditions gene therapy hasn't yet been proven in.

"There are great strengths in pharma, and there are great strengths in biotech, and the ideal situation is one that will let you employ the strengths of both types of organizations," she said.

High considered other options, such as working with a different and unproven drugmaking technology. But as someone who's spent much of her life living the story of gene therapy, she knows more than most the challenges of pioneering a new technology and convincing regulators of its worth.

Sometimes people "may underestimate the amount of time it takes to build all the tools that you need to enable regulators to say 'yes, this is safe,'" she said.

By sticking with gene therapy, much of the groundwork has been laid. She's just looking to take it a step further.

"I'm probably not going to work for another three decades," High said, with a laugh.

Read this article:
How 2 scientific pioneers teamed up to run AskBio, Bayer's new gene therapy division - BioPharma Dive

However, the partners were not willing to disclose, as of today, which diseases exactly are being targeted under this alliance.

The collaborative project combines Neurogenes manufacturing and drug development capabilities with the University of Edinburghs novel platform and neurodevelopmental disease expertise.

Under the terms of the collaboration, the US company will provide financial support for Dr Stuart Cobbs laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property at agreed-upon economic terms.Neurogene will be responsible for late stage preclinical and all clinical development of any products generated under the collaboration.

Dr Cobbs lab uses a broad range of technologies to develop novel treatments for neurodevelopmental disorders based on a deep understanding of the molecular pathology.

In addition to Dr Cobbs position at the university, where he is a Simons fellow and reader in neuroscience, he is also Neurogenes chief scientific officer (CSO).

Neurogenes lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Its product pipeline of gene therapy candidates addresses distinct monogenic neurological diseases.

Neurogene is trying to find treatments for, among others, Batten disease - a group of rare, inherited diseases of the nervous system also called neuronal ceroid lipofuscinoses (NCLs).The company is focusing on CLN5 and CLN7, two rare, late infantile and rapidly progressive subtypes of Batten disease.Children with CLN5 or CLN7 typically develop signs and symptoms of the diseases at a young age, including seizures, progressive deterioration in intellectual and motor capabilities, and loss of vision.CLN5 is caused by a variant in the CLN5 gene, which leads to disruption of normal CLN5 protein function. The CLN7 subtype of Batten disease is caused by a variant in the CLN7 gene, also called the MFSD8 gene, which leads to disruption of normal CLN7 protein function.

Another disorder Neurogene is targeting is Charcot-Marie-Tooth disease (CMT) a group of inherited diseases that affect the peripheral nervous system (PNS). CMTs are the most common inherited motor and sensory nerve disorders - neuropathies.

It is also working to determine and address the root cause of diseases such as aspartylglucosaminuria (AGU) a rare, neurodegenerative lysosomal storage disorder (LSD).

In December 2020, Neurogene announced the completion of a US$115m Series B financing, which was led by EcoR1 Capital, with participation from existing investors Redmile Group, Samsara BioCapital, Cormorant Asset Management and an undisclosed leading healthcare investment fund.

New investors included funds and accounts managed by BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, and Alexandria Venture Investments.

The company said proceeds from the financing would be used to advance Neurogenes portfolio of multiple gene therapy programs into the clinic, as well as accelerate investment in novel gene therapy product designs and Neurogenes technology platform addressing key limitations in conventional gene therapy, while building out its AAV vector GMP manufacturing capabilities.

Oleg Nodelman, portfolio manager, EcoR1 Capital, said then: Neurogene is establishing itself as a leader in the gene therapy arena for neurological diseases. We are impressed by the companys innovation and accomplishments to date and are pleased to provide our support to Neurogene to advance medical research in this rapidly evolving area.

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Neurogene in tie up with university to advance gene therapy technologies - BioPharma-Reporter.com

Jan. 22, 2021, FNF Research (fnfresearch.com) published the latest study on [2020-2026] Cell and Gene Therapy Consumables Market Report by Quantitative Research Incorporating Impact Of Economic And Non-economic Aspects was recently released. It uses exploratory techniques like qualitative and quantitative analysis to uncover and present data on the target market. Efficient sales strategies have been mentioned that would business and multiply customers in record time.

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The report also features information about significant market players across global regions that are North America, Europe, Latin America, Asia Pacific, and India. This further helps to enlighten the strong and effective business outlook of the industrial global expanse. Apart from paying attention to the present competitive current market scenario, the report also shares knowledge on the growth prospects of global Cell and Gene Therapy Consumables market during the forecast period of 2020-2026. The report also contains a circumstantiated description of various key vendors that are operating in the global regions. Showcasing a cosmopolitan landscape of Cell and Gene Therapy Consumables sector, the report marks the prevalent industry competition that is visible on both domestic as well as the global level.

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Major industry key players have been documented to study successful strategies employed by leading industries.

Amgen Inc.

ATLANTA BIOLOGICALS

bluebird bio Inc.

Cook

Dendreon Pharmaceuticals LLC

Fibrocell Science Inc.

General Electric

Kolon TissueGene Inc.

Orchard Therapeutics plc.

Pfizer Inc.

PromoCell GmbH

RENOVA THERAPEUTICS

Sibiono GeneTech Co. Ltd.

Spark Therapeutics Inc.

Vericel

Helixmith Co. Ltd.

Vitrolife

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Cell and Gene Therapy Consumables Market 2020 Key Manufacturers, Development Trends and Competitive Analysis 2026 KSU | The Sentinel Newspaper - KSU...

Researchers have implicated the pro-inflammatory cytokine interleukin-1 (IL-1) in a wide variety of diseases such as osteoarthritis, rheumatoid arthritis (RA), diabetes, and obesity. Steven Abramson, MD, the Frederick H. King Professor of Internal Medicine, professor of pathology, and chair of the Department of Medicine at NYU Langone Health, has long studied how IL-1 can propagate and exacerbate the disease process. That research effort has more recently expanded to include investigations into how the anti-inflammatory IL-1 receptor antagonist, IL-1Ra, can counter IL-1 and modulate the inflammatory response. Based on intriguing findings about how certain gene variants may influence osteoarthritis risk and severity, a new National Institutes of Health (NIH) research grant will help Dr. Abramson and collaborators seek out IL-1related targets for inflammatory disease prevention and treatment.

To help clarify the inflammatory process, Dr. Abramson and collaborators including Mukundan G. Attur, PhD, associate professor of medicine, and Jonathan Samuels, MD, associate professor of medicine, examined several variants of the IL-1Raencoding IL1RN gene in the knee joints and cells of osteoarthritis and rheumatoid arthritis patients. In particular, a haplotype designated TTG predicted which at-risk patients would go on to develop knee osteoarthritis and was associated with more severe radiographic osteoarthritis as well as new onset RA. Its a marker of both severity and increased risk for incident osteoarthritis, Dr. Abramson says.

Their 2019 study in osteoarthritis patients, published in Annals of the Rheumatic Diseases, suggested that the IL1RN TTG haplotype produced less IL-1Ra protein. So one explanation for the finding is that these people with the gene are deficient in the endogenous inhibitor of IL-1, which is driving the disease, Dr. Abramson says. Conversely, a separate haplotype called CTA yields more IL-1Ra protein production and may be protective.

In collaboration with Jef D. Boeke, PhD, professor of biochemistry and molecular pharmacology and director of the Institute for Systems Genetics, a new NIH grant may help clarify how each gene haplotype modulates inflammation, influences the associated gene regulatory networks, and contributes to the mechanics of disease pathogenesis. In particular, the research will focus on a haplotype block, or a section of DNA including multiple genes adjacent to the IL1RN gene. The researchers hope to learn whether any of the neighboring genes have inflammatory properties of their own, a synergistic effect on IL1RN, or even a more dominant effect on the underlying inflammatory pathway. One reason to do that is if youre developing a drug, you might find that one of these other genes is a better target than IL1RN, Dr. Abramson says.

One key to the unique research effort is Dr. Boekes expertise in using CRISPR-Cas9 gene editing technology to construct a series of what his lab calls assemblons, or precisely altered haplotype blocks. Led by Dr. Attur, the collaborators will then transfect embryonic stem cells with the manipulated DNA and use in vitro assays to gauge the effects of the putative risk and protective IL1RN haplotypes. The genetic manipulation is very technical. But if we can succeed, it allows us to really define the role of these haplotypes, not just in osteoarthritis but in other IL-1driven diseases, Dr. Abramson says.

After differentiating the engineered embryonic stem cells into macrophage cells, the researchers will measure production of the IL-1Ra protein. Well also be stimulating the macrophages in an inflammatory way and looking at the profile of inflammatory mediators that they produce, Dr. Abramson says. Experiments may reveal whether stimulated macrophages that carry the protective IL1RN CTA haplotype, for example, produce more IL1-Ra protein and fewer pro-inflammatory mediators such as IL-1, cyclooxygenase-2 (COX-2), and tumor necrosis factor (TNF). In the same way, sequential knockouts of other genes in the assemblon may clarify their own contributions to each haplotypes effects.

If the researchers can zero in on the principal drivers of disease through their in vitro experiments, they plan to inject the engineered embryonic stem cells into mice models of osteoarthritis and RA. The in vivo studies of the gene regulatory network may help determine how specific gene variants influence disease outcomes.

The research could have broad implications for understanding IL-1associated inflammatory diseases and for personalizing antiIL-1 therapies. It might be that in personalized medicine, antiIL-1 treatments will be more effective in patients who have a deficiency of IL-1 receptor antagonist, Dr. Abramson says. A patient who produces abundant IL-1Ra, on the other hand, may not benefit from receiving more of it as a therapy. Alternatively, the research may suggest that the IL1RN haplotypes are exerting their influence mainly by modulating other genes with key roles in the disease pathogenesis. It may be that they will emerge as targets that people hadnt even thought about in those diseases, he says.

Link:
New Research Grant Seeks to Clarify the Role Genes Play in Modulating Inflammation - NYU Langone Health

INDIANAPOLIS, Jan. 22, 2021 /PRNewswire/ --Eli Lilly and Company (NYSE:LLY) today announced the successful completion of its acquisition of Prevail Therapeutics Inc. (NASDAQ: PRVL). The acquisition establishes a new modality for drug discovery and development at Lilly, extending Lilly's research efforts through the creation of a gene therapy program that will be anchored by Prevail's portfolio of clinical-stage and preclinical neuroscience assets.

"We are pleased to complete the acquisition of Prevail and establish a gene therapy program at Lilly that has the potential to deliver transformative treatments for patients with neurodegenerative diseases such as Parkinson's, Gaucher and dementia," said Mark Mintun, M.D., vice president of pain and neurodegeneration research at Lilly.

The impact of this transaction will be reflected in Lilly's 2021 financial results according to Generally Accepted Accounting Principles (GAAP). There will be no change to Lilly's 2021 financial guidance for research and development expense or non-GAAP earnings per share as a result of this transaction.

The Offer and the MergerThe tender offer for all of the outstanding shares of common stock of Prevail at a price of (i) $22.50 per share, net to the seller in cash, without interest and less any applicable tax withholding, plus (ii) one non-tradable contingent value right (a "CVR"), which CVR represents the contractual right to receive a contingent payment of up to $4.00 per share, net to the seller in cash, without interest and less any applicable tax withholding, which amount (or such lesser amount, as further described below) will become payable, if at all, if a specified milestone is achieved prior to December 1, 2028 (the "Offer"), expired as scheduled at one minute past 11:59 p.m., Eastern time, on January 21, 2021. Computershare Trust Company, N.A., the depositary and paying agent for the Offer, has advised Lilly that27,374,689 shares of Prevail common stock were validly tendered and not properly withdrawn in the Offer, representing approximately79.8 percent of the shares of Prevail common stock outstanding. All of the conditions to the Offer have been satisfied, and on January 22, 2021, Lilly and its wholly-owned subsidiary, Tyto Acquisition Corporation, accepted for payment, and will promptly pay for, all shares validly tendered and not properly withdrawn in the Offer.

Following completion of the Offer, Lilly completed the acquisition of Prevail through the merger of Tyto Acquisition Corporation with and into Prevail, without a vote of Prevail's stockholders pursuant to Section 251(h) of the General Corporation Law of the State of Delaware, with Prevail surviving the merger as a wholly-owned subsidiary of Lilly. In connection with the merger, each share of common stock of Prevail not validly tendered in the Offer (other than (1) shares owned by Prevail (or held in Prevail's treasury) immediately prior to the effective time of the merger, (2) shares owned by Lilly, Tyto Acquisition Corporation or any other wholly owned subsidiary of Lilly immediately prior to the effective time of the merger or (3) shares held by any stockholder that was entitled to and has properly demanded statutory appraisal of such shares pursuant to, and who complied in all respects with, Section 262 of the Delaware General Corporation Law (the "DGCL") and who, as of the effective time of the merger, had neither effectively withdrawn nor lost its rights to such appraisal and payment under the DGCL with respect to such shares) has been cancelled and converted into the right to receive the same (i) $22.50 per share in cash, without interest and less applicable tax withholding, plus (ii) one CVR, as will be paid for all shares that were validly tendered and not properly withdrawn in the Offer. Prevail's common stock will be delisted from the NASDAQ Stock Market.

Under the terms of the agreement, Prevail stockholders were awarded one non-tradable CVR worth up to $4.00 per share in cash payable (subject to certain terms and conditions) upon the first regulatory approval for commercial sale of a Prevail product in one of the following countries: United States, Japan, United Kingdom, Germany, France, Italy or Spain. To achieve the full value of the CVR, such regulatory approval must occur by December 31, 2024. If such regulatory approval occurs after December 31, 2024, the value of the CVR will be reduced by approximately 8.3 cents per month until December 1, 2028 (at which point the CVR will expire). There can be no assurance any payments will be made with respect to the CVR.

About Eli Lilly and CompanyLilly is a global healthcare leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at http://www.lilly.com. C-LLY

Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements about Lilly's acquisition of Prevail Therapeutics Inc. ("Prevail"), regarding contingent consideration amounts and terms, regarding Prevail's product candidates and ongoing preclinical development, regarding Lilly's development of a potential gene therapy program, and regarding Lilly's expected 2021 financial guidance and the impact of the acquisition on research and development expense and non-GAAP earnings per share. It reflects current beliefs and expectations; however, as with any such undertaking, there are substantial risks and uncertainties in integration of acquisitions, in drug research, development and commercialization, and in Lilly's evaluation of its estimated financial results for 2021 and the impact of the acquisition. Actual results could differ materially due to various factors, risks and uncertainties. Among other things, there can be no guarantee that Lilly will realize the expected benefits of the acquisition, that product candidates will be approved on anticipated timelines or at all, that Lilly will be successful in building a gene therapy program, that any products, if approved, will be commercially successful, that all or any of the contingent consideration will become payable on the terms described herein or at all, that Lilly's financial results will be consistent with its expected 2021 guidance or that Lilly can reliably predict the impact of the acquisition on its 2021 financial guidance and results. For further discussion of these and other risks and uncertainties, see Lilly's most recent Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission (the "SEC"). Except as required by law, Lilly does not undertake any duty to update forward-looking statements to reflect events after the date of this press release.

Refer to:

Mark Taylor; mark.taylor@lilly.com; (317) 276-5795 (Media)

Kevin Hern; hern_kevin_r@lilly.com; (317) 277-1838 (Investors)

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Lilly Completes Acquisition of Prevail Therapeutics - BioSpace