December 4th 2024
EXO Biologics and ExoXpert, an EXO Biologics subsidiary, have received GMP certification of a European manufacturing facility for exosomes and have successfully loaded mRNA and DNA payloads into GMP-grade exosomes for drug delivery.
bluebird bio’s One-Time Gene Therapy for CALD Receives Positive Opinion from CHMP
May 24th 2021The Committee for Medicinal Products for Human Use of the European Medicines Agency presented a positive opinion of bluebird bio’s SKYSONA (elivaldogene autotemcel, Lenti-D), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy, an X-linked metabolic disorder.
Biogen and Capsigen to Develop Novel AAV Capsids for up to $1.37 Billion
May 12th 2021Biogen and Capsigen have entered into a strategic research collaboration to engineer novel AAV capsids to deliver gene therapies that address the underlying genetic causes of various central nervous system and neuromuscular disorders.
Catalent Enters into Gene Therapy Manufacturing Collaboration with AavantiBio
April 28th 2021Under the terms of the agreement, Catalent will handle the process development and CGMP manufacturing of AavantiBio’s adeno-associated viral vector-based therapeutic candidate for use in clinical trials in the US and Europe.
Vertex Pharmaceuticals and CRISPR Therapeutics Amend Agreement for $1.1 Billion
April 21st 2021Vertex and CRISPR have amended their collaboration agreement to include the development, manufacture, and commercialization of CTX001, an investigational CRISPR/Cas9-based gene editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia.