Archive for the ‘Gene Therapy Research’ Category

Over the next 10 years, gene therapies are expected come into their own as a treatment option for a variety of diseases. So far, two such therapies have snagged regulatory approval, Novartis Zolgensma for spinal muscular atrophy, and Sparks Luxturna for a rare form of genetic blindness. More are waiting their turn.

Multiple companies are delving into gene therapy research with hopes of developing a one-time treatment for devastating genetic diseases. Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks. Over the past year, several clinical studies have been halted or scrapped due to safety concerns.

Bay Area-based Audentes Therapeutics had a temporary hold placed on the gene therapy under development for X-linked myotubular myopathy following reports of several patient deaths. That hold has since been lifted by the U.S. Food and Drug Administration. Uniqure also saw a hold placed on its hemophilia B trial after a patient in the study developed liver cancer. The hold was placed weeks after the company announced promising Phase III results at a conference in December.

Despite those risks, hundreds of millions of dollars in research dollars are being invested in gene therapies because of the potential near-curative capabilities the technology could offer. In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Eli Lilly also dove into the field in December with the acquisition of Prevail Therapeutics. That deal was expected to extend Eli Lillys 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.

This week, German scientists reported they were able to use gene therapy to help paralyzed mice run again. The researchers were able to genetically engineer a unique protein dubbed hyper-interleukin-6, which was then able to stimulate the regeneration of nerve cells in the visual system. A few weeks after the treatment, the injured animals were able to walk again.

Scientists in China announced the development of a gene therapy that could potentially reverse the effects of ageing. Initial research was conducted with mice, but if it is proven to be safe, human testing could begin. As Reuters reported, the method involved inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing. Researchers used CRISPR/Cas9 to screen thousands of genes for those which were particularly strong drivers of cellular senescence, the term used to describe cellular ageing, Reuters said.

Earlier this month, a public-private partnership in Boston formed to open a new facility to boost advances in cell and gene therapies. This creation of this new facility is being helmed by Harvard University and the Massachusetts Institute of Technology. Those prestigious universities are partnering with industry members such as Fujifilm Diosynth Biotechnologies, Cytivia and Alexandria Real Estate Equities, as well as multiple research hospitals. Part of the goal of this new institute, which is still unnamed at this point, is to boost the supply of materials for research and early clinical studies, provide space for some research and also offer training in equipment used for gene therapies, The Harvard Gazette reported this week.

On Monday, Curadigm, a subsidiary of France-based Nanobiotix, forged a collaboration with Sanofi to assess if that companys Nanoprimer technology is a promising option to significantly improve gene therapy development. 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, Nanobiotix said in its announcement. 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.

The Nanoprimer is a combination product candidate that does not alter or modify the therapies it is paired with, which means if the research with Sanofi is successful, Curadigm could seek out other opportunities for its technology.

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Are Gene Therapies the Medicine of the Future? - BioSpace

News Release

Thursday, January 28, 2021

Approach could provide new path for difficult-to-treat forms of Leber congenital amaurosis.

Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood. A form of Leber congenital amaurosis, the disease is caused by autosomal-dominant mutations in the CRX gene, which are challenging to treat with gene therapy. The scientists tested their approach using lab-made retinal tissues built from patient cells, called retinal organoids. This approach, which involved adding copies of the normal gene under its native control mechanism, partially restored CRX function. The study report appears today in Stem Cell Reports. NEI is part of the National Institutes of Health.

Our treatment approach, which adds more copies of the normal gene, could potentially treat autosomal-dominant LCA caused by a variety of mutations, said Anand Swaroop, Ph.D., chief of the NEI Neurobiology, Neurodegeneration and Repair Laboratory and senior author of the report.

The U.S. Food and Drug Administration approved Luxturna in 2017 for the treatment of LCA patients with mutations in a gene called RPE65. Although hailed as a major advance in gene therapy, Luxturna is ineffective against other forms of LCA, including those caused by autosomal-dominant mutations in CRX.

The CRX gene encodes a protein (also called CRX) that binds to DNA and instructs the retinas photoreceptors to make light-sensitive pigments called opsins. Without functional CRX protein, photoreceptors lose their ability to detect light and eventually die.

Disorders like autosomal-dominant LCA are tricky to treat with gene therapy, because adding more of the normal gene does not always restore function. People with autosomal-dominant mutations still have one normal copy of the gene, but the mutant version of the protein interferes with the normal protein. Sometimes, instead of restoring normal function, simply adding more of the normal protein can enhance the disease in unpredictable ways.

To explore how gene augmentation adding copies of the normal gene would affect autosomal-dominant LCA, Swaroops team, developed retinal organoids from two volunteers with LCA and from their unaffected family members. Led by Kamil Kruczek, Ph.D., a postdoctoral fellow in Swaroops lab, they built the complex retina-like tissues in several stages, starting with skin cells, inducing the production of mature photoreceptors and other retinal cells with the genetic profile of each volunteer. As expected, patient organoids made far less light-sensing opsin than the organoids made from unaffected family members.

To carefully control how much CRX gene would be expressed by the recipient photoreceptors, the team re-engineered the CRX promoter so it could be delivered with the CRX gene as part of the gene therapy. A promoter is a neighboring sequence of DNA that controls when and how genes are expressed. The researchers packed the gene and their engineered promoter inside a virus that shuttled them into the organoid photoreceptors.

The teams gene augmentation strategy restored some CRX protein function for organoids from both patients, driving expression of opsins in both types of photoreceptors: rods and cones.

The fact that this strategy worked for both CRX mutations was pretty exciting, said Swaroop. Gene augmentation may be a viable therapy for LCA caused by other autosomal-dominant mutations.

This proof-of-concept gene therapy study is the first step toward a potential treatment for a rare form of LCA, said Brian Brooks, M.D., NEI clinical director and co-author on the study. Its a great example of bench-to-bedside science, when researchers in basic and clinical science collaborate.

The current study was funded through the intramural programs of the NEI and the National Institute of Allergy and Infectious Diseases, both part of NIH. Patient samples were collected at the NIH Clinical Center, clinical trial number NCT01432847.

NEI has protected intellectual property around this technology which is available for licensing and or co-development. Details can be found on the NIH OTT Licensing website: Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies | Office of Technology Transfer, NIH or by contacting NEI Office of Translational Research mala.dutta@nih.gov

Additional authors include: Zepeng Qu, James Gentry, Benjamin Fadl, Linn Gieser, Suja Hiriyanna, Zacahry Batz, Mugdha Samant, Ananya Samanta, Colin Chu, Laura Campello, and Zhijian Wu.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Kruczek K. Qu Z, Gentry J, Fadl BR, Gieser L, Hiriyanna S, Batz Z, Samant M, Samanta A, Chu CJ, Campello L, Brooks BP, Wu Z, and Swaroop A. Gene therapy of dominantCRX-Leber congenital amaurosis using patient stem cell-derived retinal organoids.Stem Cell Reports, January 28, 2020.https://doi.org/10.1016/j.stemcr.2020.12.018

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Researchers use patients' cells to test gene therapy for rare eye disease - National Institutes of Health

NOIDA, India, Jan. 29, 2021 /PRNewswire/ -- A comprehensive overview of the gene therapy market is recently added by UnivDatos Market Insights to its humongous database. The gene therapy market report has been aggregated by collecting informative data of various dynamics such as market drivers, restraints, and opportunities. This innovative report makes use of several analyses to get a closer outlook on the gene therapy market. The gene therapy market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth. Furthermore, this statistical market research repository examines and estimates the gene therapy market at the global and regional levels. The Global Gene therapy Market is expected to grow at a CAGR of 29.7% from 2021-2027 to reach US 20.9 billion by 2027.

Market Overview

Gene therapy is the next trend of curative transformation in the life sciences industry. Globally, around 2,600 clinical trials in gene therapy have been performed, are underway, or have been approved to date. More than ever, the field of gene therapy seeks to identify a route to the clinic and the market. Approximately 20 gene therapies have now been licensed and over two thousand clinical trials of human gene therapy have been published globally. Aging populations worldwide and socio-economic risk factors are among the primary influences driving this surge.

As per Alliance for Regenerative Medicine (ARM) Quarterly Regenerative Medicine Global Data Report Q12019, 372 gene therapy clinical trials were in progress as of the end of Q1. Remarkably, a margin (217 or 58%) were studies in Phase II, followed by Phase I (123 or 33%), and Phase III (32 or 9%). The number of gene therapy clinical trials edged up by 10 from the 362 recorded as of the end of 2018.

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Covid-19 Impact:

The COVID-19 pandemic has dislocated global management attempts across gene therapies. The manufacture and delivery of treatments, research and clinical development, and commercial operations are the three areas within the gene therapy sector that have been most interrupted amid the COVID-19 crisis. The development of gene therapies has been less affected. For instance, Peter Marks, Director of FDA's Center for Biologics Evaluation and Research (CBER) stated that with the arrival of therapies for cell and gene therapies over the last five years, it should have doubled in size while it is only modestly larger, 15-20% larger in size.

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Global Gene therapy Market report is studied thoroughly with several aspects that would help stakeholders in making their decisions more curated.

By Vector, the market is primarily bifurcated into

The viral vector segment dominated the gene therapy vector market in 2019 and will grow at 29.2% CAGR to reach US$ 17.9 billion by the year 2027.

By Viral Vector, the market is primarily sub-segmented into

Amongst viral vector types, adeno-associated virus accounted for the largest share and is expected to grow at 29.3% CAGR during the forecast period 2021-2027. In 2019, the adeno-associated virus segment accounted for a revenue share of almost 34%.

By Gene Type, the market is primarily studied into

In 2019, the antigen segment dominated the global gene therapy market with nearly 19.2% of the market share and it is anticipated by 2027, the segment will garner US$ 3.9 billion of the market.

By Indication, the market is primarily studied into

In 2019, the oncology segment dominated the global gene therapy market by indication with nearly 48.6% of the market share and it is anticipated to grow at 27.6% CAGR during the forecast period 2021-2027.

By Delivery Method, the market is primarily segmented into

Amongst delivery method, In vivo accounted for the largest share and is expected to grow at 28.6% CAGR during the forecast period 2021-2027. In 2019, the ex vivo segment accounted for a revenue share of 12.5%.

Gene therapy Market Geographical Segmentation Includes:

Based on the estimation, the North America region dominated the gene therapy market with almost US$ 1.7 billion revenue in 2019. At the same time, the Asia-Pacific region is expected to grow remarkably with a CAGR of 28.7% over the forecast period on account of owing increasing government initiative to improve healthcare infrastructure and rise in healthcare expenditure and surging cancer incidence rate in the region.

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The major players targeting the market includes

Competitive Landscape

The degree of competition among prominent global companies has been elaborated by analyzing several leading key players operating worldwide. The specialist team of research analysts sheds light on various traits such as global market competition, market share, most recent industry advancements, innovative product launches, partnerships, mergers, or acquisitions by leading companies in the gene therapy market. The leading players have been analyzed by using research methodologies for getting insight views on global competition.

Key questions resolved through this analytical market research report include:

We understand the requirement of different businesses, regions, and countries, we offer customized reports as per your requirements of business nature and geography. Please let us know If you have any custom needs.

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Gene Therapy Market to Reach US$ 20.9 Billion by 2027, Globally |CAGR: 29.7%|UnivDatos Market Insights - PR Newswire India

Newswise ROCHESTER, Minn. In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition.

Researchers demonstrated its potential therapeutic efficacy in two in vitro model systems using beating heart cells reengineered from the blood samples of patients with 1 long QT syndrome. They targeted the whole KCNQ1 gene rather than specific LQT1-causative mutations, making this study applicable to all patients with long QT syndrome 1, regardless of their specific disease-causing variant.

The team consisted of Mayo Clinic researchers from Mayo Clinic's:

The prevalence of long QT syndrome is about 1 in 2,000. When untreated, high-risk patients have an estimated 10-year mortality of 50%.

Long QT syndrome is a genetic heart rhythm condition that can potentially cause fast, chaotic heartbeats. These rapid heartbeats might trigger people to suddenly faint. Some people with the condition have seizures. In some severe cases, long QT syndrome can cause sudden cardiac death. The most common subtype, type 1 long QT syndrome, or LQT1, is caused by pathogenic variants in the KCNQ1 gene.

"Gene therapy is an emerging area of interest for treating a variety of genetic heart diseases in general and long QT syndrome in particular," says Michael Ackerman, M.D. Ph.D., a Mayo Clinic genetic cardiologist and director of Mayo Clinic's Windland Smith Rice Comprehensive Sudden Cardiac Death Program. "We designed and developed the first suppression and replacement KCNQ1gene therapy approach for the potential treatment of patients with type 1 long QT syndrome." Dr. Ackerman is senior author of this study.

According to Dr. Ackerman, over the past two decades, substantial improvements have been made to manage long QT syndrome, but current therapies, such as beta blockers and defibrillators, a more invasive therapy, still have limitations, risks and an array of unwanted side effects.

Gene therapy is a technique that treats diseases caused by defective genes by altering genes in a patient's cells rather than using drugs or surgery. Genes contain DNA the code that controls the body's form and function. Gene therapy replaces faulty genes or adds a new gene to try to treat a disease.

According to Dr. Ackerman in this case, this is the first time that hybrid gene therapy (simultaneous out with the old, in with the new) has been created for any form of genetic heart disease.

"If the therapeutic efficacy of this 'disease-in-the-dish' gene therapy trial with KCNQ1 can be replicated in a nonhuman, animal model of long QT syndrome, then suppression-replacement (hybrid) gene therapy may be a promising strategy for long QT syndrome in general and in theory almost any sudden death-predisposing autosomal dominant genetic heart disease," says Dr. Ackerman. "Of course, we still have a long way to go from nearly curing a patient's heart cells in the dish to effectively treating the whole person. Nevertheless, we are excited by this first critical milestone and look forward to the next step."

In addition to heart disease, researchers at Mayo Clinic's Center for Individualized Medicine are investigating an approach to replace and fix mutated genes in a wide range of genetic disorders.

Funding This work was supported by Mayo Clinic's Windland Smith Rice Comprehensive Sudden Cardiac Death Program (MJA), the Dr. Scholl Foundation (MJA), Mayo Clinic's Center for Individualized Medicine High-Definition Therapeutics grant (MJA), and Mayo Clinic's Department of Molecular Pharmacology and Experimental Therapeutics training grant under National Institutes of Health award T32GM072474 (SMD and MJA).

Disclosures Dr. Ackerman is a consultant for Abbott, Armgo Pharma Inc., Audentes Therapeutics, Boston Scientific Corporation, Daiichi Sankyo Co. Ltd., Invitae Corporation, LQT Therapeutics Inc., Medtronic, MyoKardia, and UpToDate Inc. Dr. Ackerman and Mayo Clinic are involved in an equity/royalty relationship with AliveCor Inc.

About Mayo Clinic Mayo Clinic is a nonprofit organization committed to innovation in clinical practice, education and research, and providing compassion, expertise and answers to everyone who needs healing. Visit the Mayo Clinic News Network for additional Mayo Clinic news and Mayo Clinic Facts for more information about Mayo.

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First hybrid gene therapy shows early promise in treating long QT syndrome - Newswise

With the rising demand for cell and gene therapies, the need for manufacturing innovation has never been higher. A surge of deals and expansions in the last year is fuelling the push to truly make these therapies widely available and affordable.

Cell and gene therapies offer huge potential to treat a wide range of diseases including cancer, neurological, and genetic diseases. They have even shown promise to treat the symptoms of Covid-19.

The amount of academic and early-stage biotech research in this area has skyrocketed over the last few years. According to the Alliance for Regenerative Medicine, there are currently 1,220 ongoing clinical trials in this space, 152 of which are at phase III. Despite the global pandemic, investment in this area is also at a record high around the world, with the equivalent of 15.7B invested in 2020, a figure double that of 2019.

But research alone cannot get these complex treatments to patients. The sharp discrepancy between the high number of products in early-stage development and the still very small number that have made it onto the market, as well as their cost, speaks to the impact and importance of cost-effective and scalable manufacturing, Ryan Cawood, CEO of Oxgene (previously Oxford Genetics), told me. Oxgene is a UK biotech aiming to improve manufacturing for cell and gene therapies.

To meet this challenge, cell and gene therapy producers are exploding into motion. With 2021 only just getting started, weve seen manufacturing deals between Vigeneron and Daiichi Sankyo, Sirion Biotech and Cellectis, and Cevec and Biogen. The giant Thermo Fisher Scientific absorbed the Belgian viral vector producer Henogen for 724M. And CDMO heavyweights like Cognate BioServices and Polyplus Transfection have announced expansions to their manufacturing capacity.

Thedifficulties with manufacturing the recently approved Covid-19 mRNA vaccines in high enough quantities has really highlighted the importance of having a solid manufacturing strategy in place. This lesson applies equally to companies trying to take cell and gene therapies to market.

Stuck in the first generation

Despite the huge increase in development of cell and gene therapies over the past couple of years, manufacturing technology for these therapies is largely still at the first-generation stage. This can make scaling up a challenge.

Often cell and gene therapy manufacturing processes are highly manual stemming from the early academic or process development stage and, without adequate technology solutions available currently, these processes often remain this way through clinical trials and then into commercial manufacturing, said Jason Foster, CEO of Ori Biotech, a London- and New Jersey-based company focusing on cell and gene therapy manufacturing.

These first-generation processes cause manufacturing to be expensive, highly variable and low-throughput, which reduces the ability of patients to access these potentially life-saving therapies.

Another problem common to all bio-based therapeutics is that any product sourced from a live cell or a component of one is subject to a lot more variation than a simpler pharmaceutical product.

Most gene therapies are built on viruses found in nature. They have not evolved for very high productivity in a large-scale, animal component-free bioreactor, said Cawood.

The more complicated the biologic becomes, the more parts of it require optimization, and the more analytics you require.

According to Kevin Alessandri, the cofounder and CEO/CTO of the French company TreeFrog Therapeutics, there is also a lot of waste in cell therapy manufacturing.

Yields are impaired by high cell death at every passage, and genetic alterations inevitably arise, said Alessandri. When it comes to producing commercial batches to treat thousands of patients, scaling out 2D cell culture processes is far too expensive and poses batch-to-batch reproducibility issues.

While many in the industry are now turning to bioreactors to produce cells on a bigger scale, this is also not without problems. Impeller-induced shear stress is damaging the cells, thus negatively impacting cell viability and triggering undesired genetic mutations, explained Alessandri.

Taking manufacturing up a gear

What are companies in this space doing to make scaling up cell and gene therapies easier, quicker, and cheaper?

Ori Biotech raised24.8M in Series A funding in October last year to develop an automated and robotic manufacturing system to minimize the number of manual steps needed to produce a given cell or gene therapy. This speeds up the process as well as making it more accurate. Another advantage of the technology is that it can tailor the production capacity according to demand.

This is impossible to do in most current processes, which involve manual tube welding and transfers from flask to bag to bigger bag to bioreactor, said Foster, adding that this increases cost and variability while constraining throughput. Oris technology, in contrast, could take years off the production timeline and cut costs by as much as 80%.

London-based Synthace is one of several companies trying to improve advanced therapeutic manufacturing by developing software and computer systems to optimize the process, rather than industrial machinery.

Peter Crane, Corporate Strategy Manager for the company, said that in-depth data analysis and planning before starting the manufacturing process can make a big difference to outcomes, and that connected software can help make this task easier.

The best way to remove some of the risk associated with biomanufacturing of these products is to solve as many problems as possible before manufacturing.

In addition to making the process quicker, cheaper, and more accurate, computing tools can also help with quality control and tracking. In cell therapy manufacturing, especially autologous products, line of sight around electronic batch records, as well as the vein-to-vein supply chain, is incredibly important, emphasized Crane.

Another company specifically focusing on logistics and quality control is the Cardiff- and San Francisco-based TrakCel, which nailed deals with Ori Biotech in February and the UKs National Health Service in November.

The company TreeFrog Therapeutics works with cell encapsulation technology to improve quality and reduce waste, albeit from a more mechanical viewpoint. The company launched an industrial demonstration plant in June last year, followed by two co-development deals with undisclosed big pharma partners.

Encapsulated stem cells spontaneously self-organize in an in vivo-like 3D conformation promoting fast and homogeneous growth, as well as genomic stability, said Alessandri. The resulting 3D stem cell colony can then be differentiated in the capsule into functional microtissues ready for transplantation.

With our technology, which is based on high-throughput microfluidics capable of generating over 1,000 capsules per second, it becomes possible to expand and differentiate stem cells at a large scale, in industrial bioreactors, with best-in-class cell quality and reduced operating costs.

Oxgene has a focus on scaling up production for manufacturers. In September, the company launched a technology to scale up manufacturing of viral vector production with less contamination and a 40-fold improvement in yield compared to current methods. Oxgenes expertise with viral vectors also prompted a collaboration deal in April with the CDMO Fujifilm Diosynth Biotechnologies.

Innovation in new manufacturing technologies just hasnt kept pace with the level of discovery around genetic disease and potential avenues open to treat them, or even development of the viral vectors themselves, said Cawood. This is definitely changing though.

Enter the second generation of manufacturing

Cell and gene therapy manufacturing is definitely hot right now, boosted by increased needs from biotech and pharma companies developing Covid-19 vaccines and therapies, and by notable increases in investment.

Huge advances in gene and cell therapies over the last few years, such as the approval of the eye gene therapy Luxturna and the first CAR T-cell therapies, mean the demand for new manufacturing technologies has also increased exponentially.

A lot of very promising programs are now in the pipeline, and patients are waiting for their approval, said Alessandri. Industry urgently needs robust manufacturing technology, capable of serving millions of patients.

European biotechs are busy developing second-generation technologies to allow easier and cheaper scale up, producing higher quality products with less waste. They could start to phase out first-generation methods very soon.

The realm of cell manufacturing in industrial and food biotech is also likely to see big breakthroughs in the coming years. Earlier this month, for instance, the nutrition and health giant Royal DSM set up a lab in the Netherlands dedicated to applying artificial intelligence (AI) to the challenge of growing microbial strains at a commercial scale.

Rapid improvements in advanced computing options such as AI and machine learning technology, as well as robotics, are already having an effect on the industry, but this will only get bigger as time goes on.

Cover image from Elena Resko. Body text image from Shutterstock

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Cell and Gene Therapy Firms Gear up to Revolutionize Manufacturing - Labiotech.eu

Vancouver, British Columbia, Jan. 26, 2021 (GLOBE NEWSWIRE) -- The global cell and gene therapy market is projected to reach a market size of USD 7,250.0 Million by 2028 at a rapid and steady CAGR of 16.3% over the forecast period, according to most recent analysis by Emergen Research. The growing demand for cell and gene therapy can be attributed to increasing investments in production capacity expansion for cell and gene therapy. Several contract development & manufacturing organizations and contract manufacturing organizations are making huge investments in the expansion of cell and gene therapy production capacity, anticipating a rise in demand for their services from biopharmaceutical companies that emphasize the development and production of emerging therapeutic technologies.

For instance, in May 2019, CDMO Catalent invested USD 1.20 billion in Paragon Bioservices, a contract development & manufacturing organization involved in developing and producing viral vector development for gene therapy. In April 2019, Paragon Biosciences had commenced its second good manufacturing practices (GMP) gene therapy production facility in Harmans, Maryland, the US, to provide customized manufacturing set-ups to manage the specific requirements for gene therapy products.

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Cell and Gene Therapy Market Size to Reach USD 7,250.0 Million by 2028 | Increasing Investments in Production Capacity Expansion for Cell and Gene...

RED BANK, N.J., Jan. 28, 2021 /PRNewswire/ --Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated diseases, today reported results from a pre-clinical proof-of-concept study for PRV-3279, a DART(bispecific antibody-based molecule) targeting the B cell surface proteins CD32B and CD79B, conducted in a murine model of gene therapy for Pompe disease. A PRV-3279 mouse surrogate was tested in mice transgenic for human CD32B, which received gene therapy with an adeno-associated virus (AAV) vector AAV9 encoding for the enzyme acid-alpha-glucosidase (GAA) gene. Errors in the GAA gene cause the serious human glycogen storage disease type II (Pompe disease).

In the study, the PRV-3279 surrogate reduced anti-AAV9 vector antibody levels in a dose-dependent fashion. Anti-AAV9 antibodies have been linked to reduced efficacy, safety concerns and the inability to re-dose patients, and thus, based on these and other study data, we believe PRV-3279 co-administration with gene therapy products has the potential to improve the safety and efficacy of this therapeutic modality. The PRV-3279 surrogate in combination with sirolimus increased skeletal muscle levels of GAA enzyme expression. Consistent with prior results from clinical trials in healthy human subjects, the PRV-3279 surrogate decreased IgM production and was well tolerated.

"As the field of gene therapy advances, patients' immune responses to the viral vectors and the transgene products remain a key challenge negatively impacting the safety, efficacy and ability to deliver additional courses systemically," stated Francisco Leon, M.D., Ph.D., chief scientific officer, Provention Bio. "One of the current mitigation strategies to overcome these immune responses is pharmacological modulation of the patients' antibody immune responses with the B cell depleting agent rituximab in combination with the immune-suppressive agent sirolimus. Prolonged use of rituximab has been associated with certain adverse events. The use of PRV-3279, a non-depleting B cell inhibitor, is a potential strategy to address this unmet need in serious genetic diseases."

"A critical challenge for the success of gene therapy is the host immune responses to both the vector capsid and transgene product, which pose ongoing concerns regarding the safety, longevity, extent of gene expression and ability to re-dose," stated Professor Barry Byrne, director of the Powell Gene Therapy Center at the University of Florida. "PRV-3279's mechanism of action, inhibiting B cell activation without depleting these important cells, has the potential to provide a unique opportunity to be used as an adjunctive therapy with gene therapy products. We look forward to collaborating with Provention Bio and other potential partners in forthcoming clinical studies."

"We believe PRV-3279 has the potential to intercept and prevent the immunogenicity of life-saving gene therapy products and other biotherapeutics," stated Ashleigh Palmer, CEO and co-founder, Provention Bio. "Administration of PRV-3279 has been well-tolerated and pharmacodynamically effective in Phase 1 studies, with linear PK and dose-dependent reduction in B cell activation in the absence of depletion. PRV-3279 has also been shown to reduce B cell responses to viral antigens using experimental vaccine challenge in Phase 1. Given these promising clinical data and the novel pre-clinical data in gene therapy, we look forward to opportunities to work with academic and industry experts to combine PRV-3279 with gene therapy products to further our mission of preventing and intercepting devastating immune-mediated conditions."

The company plans to submit the data from this study for presentation at an upcoming medical conference later in 2021.

About PRV-3279:

PRV-3279 is a humanized diabody (a bispecific DART molecule) targeting the B cell surface proteins, CD32B and CD79B.Simultaneous engagement of the CD32B and CD79B receptors triggers inhibition of B cell function and suppression of autoantibody production, thereby regulating B cells without causing depletion. Provention is initially developing PRV-3279 for the interception of systemic lupus erythematosus (SLE), a chronic autoimmune disorder characterized by an abnormal overactivation of B cells and subsequent pathologic production of auto-antibodies.PRV-3279 also has the potential to prevent or reduce the immunogenicity of biotherapeutics, including but not limited to gene therapy vectors and transgenes.

About Provention Bio, Inc.:

Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated diseases. The Biologics License Application (BLA) for teplizumab, its lead investigational drug candidate, for the delay or prevention of clinical type 1 diabetes in at-risk individuals has been filed by the U.S. Food and Drug Administration (FDA). The Company's pipeline includes additional clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. Visit http://www.ProventionBio.comfor more information and follow us on Twitter: @ProventionBio.

Internet Posting of Information:

Provention Bio, Inc. uses its website,www.proventionbio.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation F.D. Such disclosures will be included on the Company's website in the "News" section. Accordingly, investors should monitor this portion of the Company's website, in addition to following its press releases,SECfilings and public conference calls and webcasts.

Forward Looking Statements:

Certain statements in this press release are forward-looking, including but not limited to, statements relating to the Company's studies, the potential safety, health benefits of and planned research and development efforts for PRV-3279. These statements may be identified by the use of forward-looking words such as "anticipate," "believe," "forecast," "estimate," "expect," and "intend," among others. These forward-looking statements are based on Provention's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to delays in, or failure to obtain FDA approvals or clearances and noncompliance with FDA regulations; the potential impacts of COVID-19 on our business and financial results; changes in law, regulations, or interpretations and enforcement of regulatory guidance; uncertainties of patent protection and litigation; dependence upon third parties; substantial competition; our need for additional financing and the risks listed under "Risk Factors" in our annual report on Form 10-K for the year endedDecember 31, 2019, our quarterly reports on form 10-Q,and any subsequent filings with the Securities and Exchange Commission. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Provention does not undertake an obligation to update or revise any forward-looking statement. The information set forth herein speaks only as of the date hereof.

Partnering:Alex Rabiee, SVP, Business Development & Program Management[emailprotected]908-698-4612 (EXT-118)

Investor Contacts:Robert Doody, VP of Investor Relations[emailprotected] 484-639-7235

Sam Martin, Argot Partners[emailprotected]212-600-1902

Media:Lori Rosen, LDR Communications[emailprotected]917-553-6808

SOURCE Provention Bio, Inc.

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Provention Bio Announces Key Findings from Pre-clinical Proof-of-concept Study for PRV-3279 for the Prevention of Immunogenicity of Gene Therapy -...

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.

Blazing a Trail to Clinical Trial Diversity: Four-Part Webinar Series from Syneos Health, Featuring Pharma, Clinical Research and Community Health Leaders

This series will identify obstacles that stifle appropriate patient diversity in trials; unpack the organizational overhaul needed; share how sponsors, patients & investigators have come together to overcome hurdles; and explore how policy innovations can move the industry forward.

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.

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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.

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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

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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)

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Chinese scientists develop new gene therapy that can delay the aging process - National Post