The next generation of gene therapy for rare diseases forges ahead as developers weather hurdles Bayer to pay $2B upfront for AskBio to drive gene therapy expansion Wilson, Penn ink Regeneron pact to use gene therapy tech to deliver COVID-19 antibodies Novartis sharpens focus on vision-restoring gene therapy with $150M Vedere buyout Treating multiple sclerosis with an antigen-specific cell therapy Lilly, Precision Biosciences team up on Duchenne gene therapy in $135M deal UCB inks Handl buyout to boost nascent gene therapy unit Novartis licenses phase 3 COVID-19 cell therapy from Mesoblast Avrobio bags gene therapy to join Regenxbio and Sangamo in race EdiGene raises $67M to take gene-editing therapies into humans 4D hires a trio of area heads as it ramps up its gene therapy pipeline After boosting gene therapy focus, Bayer signs up to new cell therapy pact with Atara Featured Story | By Arlene Weintraub Even amid setbacks at the FDA, biopharma companies have been improving gene therapies to treat rare diseases like PKU, Fabry and gangliosidosis, with advances that range from new gene-insertion methods to technologies that can get the treatments to hard-to-reach areas of the body. read more |
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Top Stories By Nick Paul Taylor Bayer has struck a deal to acquire Asklepios BioPharmaceutical for $2 billion upfront. The deal, which features an additional $2 billion in milestones, will give Bayer control of an adeno-associated virus (AAV) gene therapy platform and a pipeline led by clinical-phase treatments for Parkinson’s, Pompe disease and congestive heart failure. read more By Ben Adams Gene therapy pioneer Jim Wilson and the University of Pennsylvania are teaming up with Regeneron to help deliver its COVID-19 antibody cocktail using AAV tech in the hope of curbing infection via a nasal spray. read more By Amirah Al Idrus Vedere Bio didn’t stand a chance. The Atlas Venture gene therapy startup was working under the radar on its vision loss candidates and contemplating another venture round when it got an offer from Novartis it couldn’t refuse: $150 million up front, plus $130 million more if it hit certain goals. read more By Angus Liu Scientists at Thomas Jefferson University said they have found a way to train the immune system to tolerate myelin self-antigens that trigger inflammatory responses in multiple sclerosis. It consists of tiny sacs called extracellular vesicles from cells known as oligodendrocytes, and the therapy showed promise in mice. read more By Amirah Al Idrus Once wary of inking deals around gene therapy, Eli Lilly is jumping headfirst into developing treatments that edit genes within the body. It’s teaming up with cell and gene therapy biotech Precision Biosciences to develop in vivo gene therapies for three gene targets, starting with Duchenne muscular dystrophy. read more By Nick Paul Taylor UCB has struck a deal to buy gene therapy startup Handl Therapeutics. The takeover, which was disclosed alongside a separate gene therapy collaboration, positions UCB to accelerate its push into genetic medicines. read more By Nick Paul Taylor Novartis has secured a global license to Mesoblast’s cell therapy remestemcel-L in the treatment of COVID-19. The deal lands Mesoblast $50 million upfront and the support of a partner that could help address critical quality attribute concerns raised by the FDA. read more By Nick Paul Taylor Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder. read more By Nick Paul Taylor EdiGene has raised around $67 million to advance work on gene-editing therapies. The Beijing-based biotech will use the series B funding to progress a pipeline led by a beta-thalassemia treatment and an allogeneic CAR-T therapy. read more By Amirah Al Idrus 4D Molecular Therapeutics raised $75 million in June to get several gene therapy programs into and through the clinic. Now, it’s adding a trio of executives to spearhead its work in heart, eye and lung diseases as it looks to shepherd treatments in those focus areas forward. read more By Ben Adams German pharma Bayer is moving into cell therapy R&D with its latest pact focusing on tough-to-treat lung cancers with Atara Biotherapeutics. read more Resources Sponsored by: Covance Download the whitepaper to learn how your safety organization can implement automation technologies to enable process and cost efficiencies, improve resource allocation and ensure compliance and quality. Sponsored By: Oracle Health Sciences New research indicates an accelerated adoption of decentralized clinical trials due to COVID-19. Sponsored By: BBK Worldwide The BBK Worldwide Study Voices 2020 survey gives voice to the healthcare consumer in the wake of COVID-19 and reveals important insights for clinical trial enrollment and engagement. 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