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Five Clinical-Stage Cell and Gene Therapy Startups Leading the Pack
After decades of research, cell and gene therapies are gaining traction in the biotech industry. Here are five investor-favored private startups with products already...
SNIPR Biome Reports Good Initial “In-Human” Results for CRISPR Gene Therapy
SNIPR Biome achieves good interim Phase I results in a trial to combat antibiotic resistant E. coli in blood cancer patients.
Ray Therapeutics Raises $100M Series A to Fuel Gene Therapy for Retinitis Pigmentosa
Ray Therapeutics has raised $100M to support the start of clinical trials for a gene therapy aimed at retinitis pigmentosa.
Sarepta Gets Adcomm Backing for Duchenne Gene Therapy
Despite some skepticism, an FDA advisory committee deemed the benefits of Sarepta's gene therapy for Duchenne muscular dystrophy were greater than the risks.
Bluebird Tightens Sickle Cell Race with BLA for Gene Therapy
Bluebird bio's submission for FDA approval of its one-time sickle cell gene therapy puts them in direct competition with Vertex and CRISPR Tx.
REGENXBIO Launches Early Phase Trial of Its Gene Therapy for Duchenne Muscular Dystrophy
REGENXBIO is launching a Phase I/II trial of its gene therapy, RGX-202, for Duchenne muscular dystrophy
Charles River and Rznomics Ink RNA-Based Anticancer Gene Therapy Manufacturing Deal
Charles River Laboratories International and Rznomics have announced a viral vector contract development and manufacturing partnership around mRNA-based gene therapy for liver cancer.
Lipid Nanoparticle Technology May Advance Gene Therapy for Blindness
Oregon researchers develop lipid nanoparticle technology to deliver vision-revising genes to photoreceptors in the retina, possibly treating inherited blindness.
Neurocrine and Voyager Ink New Potential Over $1 Billion Neuro Gene Therapy Deal
Neurocrine Biosciences and Voyager Therapeutics have announced a new collaboration worth potentially $1B around multiple gene therapies for the treatment of neurological diseases.
In Another Hemophilia Advance, Pfizer Reports Positive Results for Gene Therapy
Pfizer reported very positive results from a Phase III study of an investigational gene therapy in men with moderately severe to severe hemophilia B. This caps off a breakthrough year for hemophilia B treatment, as the first gene therapy for this blood clotting disorder (CSL Behring’s Hemgenix) received FDA approval last month.