gene therapy
Rocket Pharmaceuticals' Leukocyte Adhesion Deficiency-I Gene Therapy Under FDA Priority Review
The drugmaker submitted data from a Phase I/II study in which all nine LAD-I patients who received the one-time RP-L201 infusion were alive after one year.
PTC Therapeutics Cuts 25 Percent of Workforce, Downsizes Gene Therapy R&D
The workforce reduction primarily affects staff in early-stage research programs and those at a gene therapy manufacturing facility.
GenSight Biologics Unveils Preliminary Plans for New Inherited Vision Loss Gene Therapy Trial
The firm, which withdrew its EMA application for Lumevoq earlier this year, is working with the agency to refine the design of a new Phase III trial.
Abeona Therapeutics Submits BLA for Recessive Dystrophic Epidermolysis Bullosa Cell Therapy
The company submitted safety and efficacy data on the autologous, engineered cell therapy, EB-101, from the pivotal Phase III VIITAL trial.
Kriya Therapeutics Gains Access to Everads' Suprachoroidal Drug Delivery Platform
Kriya made an undisclosed upfront payment to gain field-based exclusive rights to use Everads' platform for certain therapeutic targets including for ophthalmic diseases.
Sep 25, 2023