Ana de Barros, PhD, managing science editor —

Abeona Therapeutics has announced the opening of The Elisa Linton Center for Rare Disease Therapies, a commercial manufacturing facility for advanced gene and cell therapies in Cleveland, Ohio. The new GMP (good manufacturing practices) facility will have the capability to manufacture clinical and commercial grade products over Abeona’s multiple programs, including …

Lysogene’s Phase 2/3 trial on LYS-SAF302 gene therapy, a clinical candidate for Sanfilippo syndrome type A, will begin in the second half of 2018 due to manufacturing delays. LYS-SAF302 is a viral vector that carries a healthy SGSH gene. This gene gives instructions to produce an enzyme called sulfamidase, which…

The U.S. Food and Drug Administration granted its regenerative medicine advanced therapy (RMAT) designation to Abeona Therapeutics‘ investigative gene therapy ABO-102, under development for the treatment of Sanfilippo syndrome type A. Also known as MPS IIIA (mucopolysaccharidosis type IIIA), Sanfilippo syndrome type A is a severe and progressive disorder caused…

The Nuffield Council on Bioethics, an independent advisory group on ethical issues in bioscience in the United Kingdom and abroad, recently published briefing material on the ethical issues raised by the whole genome sequencing of newborns and small infants. This sequencing is now being used in the U.K.’s National Health…