rare disease
BridgeBio Discontinues Development of Gene Therapy for Congenital Adrenal Hyperplasia
The company said newly released Phase I/II study data for BBP-631 indicated the program did not merit further capital investment.
Muscular Dystrophy Association Announces First Project Under Kickstart Program
The first project in this program to advance gene therapies for very rare conditions will focus on CHAT-mutated congenital myasthenic syndrome.
Ionis Pharmaceuticals Prices $500.3M Stock Offering
The antisense oligonucleotide developer plans to use the funds raised to support its independent commercial launches, late-stage clinical programs, and more.
BioMarin Announces New Strategy, Structure Aimed at Reaching $4B Revenue Goal
The drugmaker's strategy centers on three business units: skeletal conditions, enzyme therapies, and its hemophilia A gene therapy.
Promising 12-Month Data on Atsena's LCA1 Gene Therapy Bolsters Case of Pivotal Trial
The latest safety and efficacy Phase I/II data on ATSN-101 support moving the gene therapy into a randomized Phase III trial.
Jul 24, 2024