Both Solid Biosciences Inc. and Pfizer Inc. got lifts to their Duchenne muscular dystrophy (DMD) programs from the FDA as the agency released a clinical hold and awarded a fast track designation to their respective adeno-associated viral (AAV) programs.
DUBLIN – Dyno Therapeutics Inc., an early stage gene therapy firm applying artificial intelligence to advanced capsid engineering, has entered partnerships with Novartis AG and Sarepta Therapeutics Inc., in ophthalmic indications and muscle diseases, respectively, which have over $2 billion in biobucks attached.
Weeks after raising an oversubscribed $60 million series A, Affinia Therapeutics Inc., of Waltham, Mass., is collaborating with Vertex Pharmaceuticals Inc. in a far larger deal, one potentially worth more than $1.6 billion.
Privately held Swanbio Therapeutics Inc., of Bala Cynwyd, Pa., completed a $52 million expanded series A financing designed to boost the company’s R&D of adeno-associated virus (AAV)-based gene therapies to treat adrenomyeloneuropathy (AMN) and other rare, monogenic neurological diseases.
While many companies use viruses and viral vectors to deliver gene therapy and to modify cells for CAR T treatments, others have shunned adeno-associated viruses (AAV) and lentiviral vectors for other methods to deliver DNA and RNA into the cells.
