Audentes Therapeutics UK Ltd.

Dive Brief:Japanese drugmaker Astellas will invest $50 million into Taysha Gene Therapies, gaining access to the Dallas, Texas-based biotechnology companys pipeline of experimental treatments in a deal announced Monday.In return, Astellas will gain a 15% stake in Taysha, as well as options to license rights to two of the companys gene therapies currently in clinical testing. Astellas will also hold an observer seat on Tayshas board of directors.While a minority shareholder, Astellas has certain preferred negotiation rights should Taysha be acquired by another company, or there be a change of control.Dive Insight:The latest investment gives Astellas a larger presence in gene therapy, an area its invested heavily in since 2019, when it bought Audentes Therapeutics for $3 billion. More recenty, it built a gene therapy plant in North Carolina to supply its clinical trials as well as marketing of any therapies its cleared to sell.But Astellas has faced setbacks in its research expansion. In the months following the Audentes acquisition, researchers reported the deaths of three boys given Audentes lead gene therapy, for X-linked myotubular myopathy, causing testing to be suspended. The trial later resumed, but was halted again after the death of a fourth boy. All four had signs of liver damage following treatment with the gene therapy.The study remains on clinical hold, as of Aug. 1 this year.Tayshas research is much earlier stage, as the company was spun out of the University of Texas-Southwesterns medical school in 2020 with $30 million. The startup quickly went public, raising over $150 million in an initial public offering later that year.Since then, however, Taysha shares have lost almost all of their value. In April, the company was forced to reduce its workforce by 35% to save cash. Shares in the company were worth about $1.50 apiece at Mondays close.The company is advancing gene therapies for giant axonal neuropathy and Rett syndrome, both rare central nervous system disorders. The two candidates Astellas now holds an option to license TSHA-102 for Rett syndrome and TSHA-120 are both in clinical testing.Taysha is expecting to receive regulatory feedback on the latter, as well as disclose initial clinical data on the former by the end of this year. '

AviadoBio Appoints Graeme Fielder as Chief Operating Officer and Alex Bloom as Chief Technical Officer London, UK; October 4, 2022 — AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, today announced the promotions of Graeme Fielder, Ph.D. to Chief Operating Officer and Alex Bloom, Ph.D. to Chief Technical Officer. Graeme Fielder, part of the founding management team at AviadoBio, has served as Vice President and Head of Operations since 2020. Graeme has deep experience in the life sciences industry, holding roles in strategy, business development, and program and alliance management over the past 10 years. He joined AviadoBio from Audentes Therapeutics (now Astellas Gene Therapies), a neuromuscular gene therapy company, where he oversaw the company’s corporate development activities including the acquisition of Astellas Pharma. Graeme holds a BSc (Hons) degree in Biotechnology and a Ph.D. from the University of Auckland, New Zealand. Graeme also has an MBA from Stanford University, USA. Since joining AviadoBio in September 2021, Alex Bloom has served in the role of Senior Vice President Technical Operations, heading up all technical operations and enabling functions across CMC, quality assurance and regulatory affairs. His experience includes more than 15 years of operational excellence in biologics, with over 10 years spent dedicated to cell and gene therapy development. Previously, he led quality and regulatory activities at Gyroscope Therapeutics, supporting the development of gene therapies and innovative delivery devices for retinal disease. Alex holds a Ph.D. and MSc from Sheffield University, UK. AviadoBio also noted today that Co-Founder and Chief Scientific and Clinical Advisor Professor Chris Shaw will be taking on an additional role. He has been named the Hugh Green Foundation Chair in Translation Neurology at the Centre for Brain Research at the University of Auckland, New Zealand. Professor Shaw retains his roles at AviadoBio and King’s College London. “Alex and Graeme have been integral members of the AviadoBio team and their promotions further validate their great work as well as the incredible growth of the company. I know that both Alex and Graeme, alongside the rest of the management team, will continue to drive AviadoBio forward as we work to bring our gene therapies for frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) into the clinic,” said Lisa Deschamps, Chief Executive Officer. – ENDS – About Frontotemporal Dementia (FTD) and FTD with GRN mutations (FTD-GRN) Frontotemporal dementia (FTD) is of the second most common form of dementia in people under the age of 65 after Alzheimer’s disease. It affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U. Approximately one third of FTD cases are familial and linked to autosomal dominant mutations in three genes including the granulin gene (GRN) and FTD-GRN represents 5-10% of all patients with FTD. Progressive degeneration of the frontal and temporal lobes of the brain is characteristic of FTD, and is associated with progressive decline of behaviour, decision-making, language and emotion, typically leading to death within 7-10 years of diagnosis. There are currently no approved treatments to stop or slow the progression of FTD or FTD-GRN. About AviadoBioAviadoBio’s mission is to develop and deliver potentially transformational gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, we are working relentlessly to overcome the challenges of delivering the right drug to the right place in time. Our innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential to halt or potentially reverse neurodegenerative diseases. The company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. The lead program, AVB-101, is an investigational AAV gene therapy designed to slow or stop FTD disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Lifesciences, and LifeArc. For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio. Contact: For media enquiries: Farah SpeerSVP, Head of Communications and External RelationsFspeer@aviadobio.com+1-312-543-2881 Consilium Strategic CommunicationsChris Gardner, Angela Gray, Sukaina Virji+44 (0) 20 3709 5700AviadoBio@consilium-comms.com

The IPO window has been closed to most biotech companies this year, but Third Harmonic Bio is pushing it open with a drug from Novartis that might work better than the wide selection of inflammation medicines already available to patients. The biotech has early clinical data showing signs of efficacy. Now the company wants to tap the public markets to finance further clinical development of the molecule in multiple inflammatory disorders. Cambridge, Massachusetts-based Third Harmonic did not set any financial terms for the stock sale outlined in the preliminary IPO paperwork filed with regulators on Tuesday. However, IPO research firm Renaissance Capital said the offering could reach up to $150 million. Third Harmonic has applied for a Nasdaq listing under the stock symbol “THRD.” Third Harmonic’s research focuses on the mast cell, a type of immune cell in the blood whose dysfunction is associated with the development of many allergic and inflammatory diseases. These cells are found throughout the body and are prominent on tissue with exposure to the external environment, such as the skin, respiratory tract, and gastrointestinal tract. Lead Third Harmonic drug THB001 is a small molecule designed to block KIT, a cell surface receptor whose role is to regulate mast cells. In the IPO filing, the company notes that KIT inhibition has shown signs of efficacy in mast cell-mediated diseases such as asthma. A wide range of therapies already treat asthma and other inflammatory disorders, but Third Harmonic notes in the filing that many of these drugs are insufficient because they target the compounds that are produced by mast cells, such as histamines. For many diseases, multiple compounds are involved. “As a result, we believe that targeting mast cells directly through highly selective inhibition of KIT is key to achieving the clinical efficacy needed for broad symptomatic relief across a range of allergic and other inflammatory disorders,” Third Harmonic said in the filing. So far, Third Harmonic has tested its lead drug in a Phase 1a clinical trial. Though this study only enrolled healthy volunteers, the drug flashed indications of efficacy. The company reported that blood tests showed dose-dependent declines in tryptase, an enzyme released when mast cells are activated. Third Harmonic now aims to test whether its drug can treat chronic urticaria, an inflammatory skin condition characterized by hives and an itchy rash. The company said in the filing that it has submitted a clinical trial application in Europe for a dose-escalation Phase 1b study intended to show proof-of-concept in this disease. Data are expected in the second half of next year. Asthma is the next disease target. Third Harmonic plans to start a Phase 1b study in the first half of 2023 and report data in the second half of 2024. The biotech is also eying the start of Phase 2 tests in chronic spontaneous urticaria. Those studies in the U.S. and Europe are planned for the first half of 2024. Third Harmonic describes its lead asset as a “pipeline in a product.” The company is exploring other diseases where stopping mast cell-driven inflammation may offer relief. Third Harmonic has competition in its pursuit of a better drug for urticaria, a condition whose first line of treatment is antihistamines. Blocking KIT is the objective of Celldex Therapeutics drug barzolvolimab, formerly known as CDX-0159. The Hampton, New Jersey-based biotech has reached early clinical development in urticaria and other inflammatory disorders. But as an antibody, that drug must be given as an intravenous infusion. Third Harmonic hopes that the oral formulation of its KIT-blocking drug gives it an edge. Third Harmonic is led by CEO Natalie Holles, who previously served as chief executive of gene therapy developer Audentes Therapeutics (now a part of Astellas Pharma). Venture capital firm Atlas Venture founded Third Harmonic in 2019. That year, it licensed THB001 from Novartis for $400,000 cash and an equity stake in the emerging biotech. Much more could be paid out depending on the progress of the research: $31.7 million is tied to in development milestones and up to $200 million in sales and commercialization milestones. Novartis would also earn royalties from the sale of any approved products. Third Harmonic raised $155 million in its start, most recently a $105 million Series B round that was announced in February. Atlas is the largest shareholder with a 37.9% stake, according to the filing. The Novartis Institutes for BioMedical Research owns 9.4% of the company. As of June 30, Third Harmonic’s cash position was $112.7 million, which the company said will be enough to fund operations for at least 12 months. Third Harmonic plans to use the IPO cash to continue clinical development of TBH100, specifically the Phase 1b study in chronic inducible urticaria and the Phase 2 test in chronic spontaneous urticaria. The cash will support the planned Phase 1b test in asthma. The biotech also states in the filing that the capital may be deployed toward the development or acquisition of other programs.