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DNA structure

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...
Bacteria inside the small intestine, concept, representation to indicate problems with the gut microbiome

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.
Human dark brown eyes close-up.  Side look

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.
Motor neuron connecting to muscle fiber to illustrate myasthenia gravis

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.
Blood clot made of red blood cells, platelets and fibrin protein strands. Thrombus

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.
Genetic research at the laboratory

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
Human liver anatomy. Liver cancer symptoms

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.
Treatment with lipid Nanoparticles, Genetic therapy, Fluorescent staining

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.
Brain MRI Scan of Healthy Male  ( Magnetic Resonance Imaging) High Resolution

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.
Blood vessel with platelets, white blood cells and red blood cells.

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.


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