SNIPR Biome, a Danish biotech combining CRISPR gene editing and microbiome-based technology, has achieved good initial results in a Phase I trial of its therapy to target Escherichia coli infections in vulnerable cancer patients. Currently known as SNIPR001, the therapy being tested combines four CRISPR-armed bacteriophages that selectively target and…

Ray Therapeutics, an optogenetics start-up founded in 2021, has announced a $100M Series A financing round led by Novo Holdings A/S. The funding will advance multiple programs targeting blinding diseases of the eye through clinical development. Ray’s lead candidate is RTx-015 in retinitis pigmentosa, a degenerative retinal disease. It is…

On Friday, an FDA advisory committee voted 8-6 that the benefits of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy (DMD) outweighs the treatment’s risks. If it is approved, SRP-9001 (delandistrogene moxeparvovec), which is being developed with Roche, will be the first gene therapy approved for this disease. The vote…

Bluebird bio has submitted to the FDA for approval of its sickle cell gene therapy. It’s a one-time treatment that adds a functional β-globin gene into the patient’s own hematopoietic (blood) stem cells. Bluebird’s move comes soon after Vertex and CRISPR Therapeutics submitted for their gene editing therapy in the…

REGENXBIO is launching a Phase I/II trial of its gene therapy, RGX-202, for Duchenne muscular dystrophy. RGX-202 delivers a transgene for a novel microdystrophin protein that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 is designed for delivery and targeted expression of genes…

Charles River Laboratories (CRL) International and Rznomics Inc. have announced a contract development and manufacturing organization (CDMO) partnership around liver cancer. CRL has recently taken major steps, including investing about $1B, to be able to offer a comprehensive cell and gene therapy development platform. A South Korea-based biotech, Rznomics specializes in…

Researchers at Oregon Health & Science University have applied lipid nanoparticles previously used to deliver COVID-19 vaccines as a new approach to gene therapy which could improve the treatment of inherited types of blindness. Five years ago, the FDA approved the first adeno-associated virus (AAV) based gene therapy to treat…

Neurocrine Biosciences and Voyager Therapeutics have announced a new collaboration around multiple gene therapies for the treatment of neurological diseases that could be worth over $1B.  Under the agreement, Neurocrine Biosciences will pay Voyager $175 million up front, and Voyager will be eligible for up to $985 million in total…

Pfizer today announced very positive results from a Phase III study of fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult 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…

iECURE, a gene editing startup focused on liver disorders, this week announced a $65 million Series A-1 financing. The company is collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, MD, PhD, who led the gene-therapy trial that ended with teenager Jesse Gelsinger’s death…

Researchers at University College London (UCL) have developed a new gene therapy approach for epilepsy and potentially other neurological and psychiatric diseases that works by reducing the excitability of overactive brain cells. The new closed-loop gene therapy approach enables the on-demand expression of a gene that tamps down the activity…

Applied Genetic Technologies Corporation (AGTC), a developer of adeno-associated virus (AAV)-based gene therapies, is being acquired by Syncona Limited. AGTCs initial focus has been on inherited retinal diseases (IRDs). Syncona will pay $23.5 million ($0.34 per share) in cash at the closing of the transaction plus potential future aggregate cash payments…

Xcell Biosciences and aCGT Vector have announced a collaboration for manufacturing and analysis around personalized cell and gene therapies for cancer patients. aCGT Vector will provide its point-of-care GMP-licensed manufacturing platform to validate Xcellbio’s manufacturing and analytical AVATAR AI technology for use in precision cancer treatment. The partnership pairs aCGT’s…

Eli Lilly and Company announced today that it intends to acquire Akouos, a developer of gene therapies focused on treating hearing loss, for $12.50 per share, or a total of $487 million payable at the closing of the deal. The agreement also includes one non-tradeable contingent value right per share…

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