Novel RNA-Targeting Gene Therapy Shows Promise for Neuromuscular, Retinal Diseases
The CEO of Locanabio shares details of 2 presentations the company is making this week at the 24th Annual Meeting of the American Society of Gene & Cell Therapy.
Lentiviral-Based Gene Therapy for LAD-I Improves Clinical Course, CD18 Expression
Typically treated with stem cell transplantation, this lentiviral vector-based gene therapy approach may resolve challenges related to donor availability and post-transplantation complications.
Ofatumumab sBLA Is Accepted for Treatment of Relapsing Multiple Sclerosis
The novel B-cell therapy has been assigned a PDUFA date of June 2020.
Duchenne Muscular Dystrophy Gene Therapy On Hold After Safety Incident
This is the third safety incident in the IGNITE DMD clinical trial that has resulted in a clinical hold since its inception in 2017.
FDA Approves Gene Therapy Zolgensma for Spinal Muscular Atrophy
Zolgensma, an adeno-associated virus vector-based, one-time gene therapy administered via intravenous infusion, is the first and only FDA-approved gene therapy for SMA.