Gene Therapy

1st Patient Dosed in Trial of Gene Therapy, OTL-201, for Sanfilippo Type A Orchard Therapeutics announced the dosing of a first patient in a clinical trial evaluating its investigational cell-based gene therapy OTL-201 for Sanfilippo syndrome type A. OTL-201’s safety, tolerability, and efficacy is being investigated in the open-label, Phase 1/2 study (NCT04201405) taking place at Manchester University NHS Foundation Trust in the…

Nationwide Children’s Hospital chooses Mayo Pujols, most recently of Novartis Pharmaceuticals, for its new biotech subsidiary Andelyn. As the new CEO for Andelyn Biosciences, a Nationwide Children’s Hospital-owned gene-therapy startup, Mayo Pujols will begin to assemble a diverse team of up to 200 scientists, biochemists, engineers and technicians. The portfolio of gene-therapy products to be…

A new biotech startup in Dallas aims to speed the development of treatments for rare neurological diseases that disproportionately affect children. Taysha Gene Therapies, launched Tuesday with $30 million in seed financing, has partnered with UT Southwestern researchers whose mission is to eradicate genetic diseases that can deprive children of the ability to walk, talk,…