Gene therapy company Kriya has acquired Tramontane Therapeutics, which is developing such therapies for metabolic and neurodegenerative diseases. Kriya has prioritized Tramontane’s lead program that uses an adeno-associated virus (AAV) vector targeting Fibroblast Growth Factor 21 (FGF2), which has been established as a clinically-validated biological target in Non-alcoholic steatohepatitis (NASH). “People with NASH…

Investigators led by a researcher from the Ohio State University Wexner Medical Center and College of Medicine report that a one-time administration gene therapy shows potential as a treatment for serious alcohol addiction. The study, published today in the journal Nature Medicine, used an accepted primate model to show that…

Astellas Pharma and 4D Molecular Therapeutics (4DMT) just announced a license agreement giving Astellas rights to 4DMT’s intravitreal retinotropic R100 vector for use in gene therapy for a rare monogenic ophthalmic disease, with options to add up to two additional targets after paying additional option exercise fees. 4DMT will receive…

Sickle cell disease (SCD) and beta thalassemia could be cured by gene therapy, but which approach would be most effective and safest? A team from St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard may have the answer. They compared five gene editing strategies in CD34+ hematopoietic stem…

The first gene therapy for hemophilia A has finally reached the U.S. This week the FDA approved BioMarin’s Roctavian (valoctocogene roxaparvovec), an adeno-associated virus (AAV) vector-based gene therapy for the treatment of adults with severe hemophilia. The treatment reduced means annualized bleeding rate from 5.4 bleeds per year at baseline…

Kate Therapeutics (KateTx) today emerged from stealth mode with a $51 million Series A round and a deal with Astellas Pharma to develop a gene therapy for X-linked myotubular myopathy (XLMTM). “KateTx is applying novel capsid and cargo technology platforms to enable skeletal and cardiac muscle targeting and liver de-targeting. We believe…

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 in clinical development. While conventional medications are often effective at controlling symptoms or modifying a disease, there are many conditions where their benefits are limited, such…

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…