Bayer opens its new cell therapy manufacturing facility as it prepares a Parkinson’s disease cell therapy for Phase 2 testing. The new 100,000 square foot facility also has space for manufacturing other cell therapies in the pharma giant’s pipeline.
The Orchard Therapeutics acquisition comes as the biotech’s gene therapy for metachromatic leukodystrophy approaches a March 2024 FDA decision. The ex vivo gene therapy is already commercialized in Europe, marketed as Libmeldy.
The TSX Venture Exchange has a strong history of helping early-stage health and life sciences companies raise patient capital for research and development.
Gene therapy biotech Kriya Therapeutics has acquired Tramontane Therapeutics, a startup focused on metabolic diseases. Tramontane brings a gene therapy that’s a potential one-time treatment for the fatty liver disease NASH.
The FDA approved Roctavian for treating hemophilia A. The regulatory decision makes the BioMarin Pharmaceutical product the first gene therapy for this inherited bleeding disorder.
Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.
Multiple efforts are underway to improve cell therapies for cancer. During the MedCity News INVEST conference in Chicago, a panel discussed the future of cell therapies and what the industry doing to manage the financial risks of these new treatments.
At the Payer Insights sessions on Day 1 of ViVE 2024, a panel on prior authorization offered compelling insights from speakers who shared the positive developments in this area after years of mounting frustration. Speakers also shared challenges as they work with providers to figure out how policy developments and technology will work in practice.
Companies presenting their technology at the conference seek to support a diverse patient population spanning conditions such as inflammatory bowel disease, food allergies, and cancer. Cell, gene therapy and immunotherapies also form some of their treatment approaches in development.
By selling partial royalties to approved hemophilia gene therapy Hemgenix, uniQure gains immediate cash to support its pipeline, including a Huntington’s disease gene therapy already in the clinic . The royalty deal extends uniQure’s cash runway into 2026.
A panel discussion at the annual Abarca Forward conference in Puerto Rico last month sought to identify some of the innovative financial approaches that can be applied to managing the high price of novel therapies. Conversations also addressed what payers need for these financing models to be sustainable.
VintaBio manufactures AAV and lentiviral vectors for entities pursuing clinical development of cell and gene therapies. The contract development manufacturing organization’s launch comes as demand for viral vectors continues to outpace the supply of these crucial components of the therapies.
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BioMarin Pharmaceutical's submission of additional data for patients treated with its hemophilia A gene therapy, Roctavian, mean that the FDA will push out a regulatory decision to early summer. But it could be worth the wait as analysts anticipate FDA approval and project Roctavian becoming one of BioMarin’s biggest products.
AbbVie and Capsida Therapeutics are expanding their gene therapy R&D alliance to the eyes. Capsida is in line to receive $70 million now and up to $595 million later, depending on the progress of the eye programs.
Panelists at a recent pharmacy conference were optimistic about the future of value-based drug contracting. For example, one said that healthcare will be close to establishing a value-based formulary by 2030, and another said that longitudinal patient tracking will become a more important part the drug pricing process.
Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study—a challenge in any ultra-rare disease.
Vector BioMed has developed a technology platform for designing and manufacturing the lentiviruses used to deliver cell and gene therapies. The startup is launching as the field continues to experience shortages of these viral vectors.