Alicia Bigica

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.

Typically treated with stem cell transplantation, this lentiviral vector-based gene therapy approach may resolve challenges related to donor availability and post-transplantation complications.

The novel B-cell therapy has been assigned a PDUFA date of June 2020.

This is the third safety incident in the IGNITE DMD clinical trial that has resulted in a clinical hold since its inception in 2017.

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.