The U.S. FDA approved Orchard Therapeutics plc’s BLA for gene therapy atidarsagene autotemcel, making it the first treatment option for metachromatic leukodystrophy in the U.S. The one-time treatment, branded Lenmeldy, is indicated for children with presymptomatic late infantile, presymptomatic early juvenile or early symptomatic early juvenile disease.
The University of California, Berkeley has announced the launch of the UC Berkeley Molecular Therapeutics Initiative (MTI) designed to accelerate drug discovery. The aim is to bridge fundamental research in rare neurological and metabolic diseases with drug discovery to identify and accelerate novel therapeutic modalities into the clinic.