Combined company to operate as Remix Therapeutics and advance Remix’s pipeline of novel small molecule therapies designed to reprogram RNA ...

Researchers at work in Sangamo Therapeutics' laboratory. The pioneer gene editing biotech company has filed for Chapter 11 bankruptcy protection, and has agreed to sell some of its programs and ...

A new gene-editing company emerged from stealth on Tuesday with $230 million in committed financing, a reverse merger with a defunct oncology biotech, and a lead drug candidate licensed from a Chinese ...

Unlike retroviral and lentiviral gene-therapy vectors, adeno-associated virus (AAV) vectors generally do not integrate into the recipients’ genome, so they are preferred in gene-therapy development.

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Capsida Biotherapeutics said Tuesday that ...

Collaboration leverages AAV manufacturing expertise to advance a preclinical-stage gene therapy program for a rare pediatric neurological disorder. SAN SEBASTIÁN, Spain and LONDON, May 5, 2026 ...

Financing extension led by 8VC to advance LTS-201 as a potential significant commercial opportunity in Huntington’s disease (IND expected in 3Q 2026) Potential clinical proof-of-concept of Latus ...

Physician and molecular biologist Katherine High remembers sitting at a staff meeting of the gene therapy company Spark Therapeutics on November 15, 2018, waiting to hear from a guest speaker, when ...

Regeneron will make its newly approved gene therapy Otarmeni for a rare, genetic form of hearing loss available for free in the U.S. The company hasn't decided how it will price the therapy overseas, ...

A new therapy for genetic hearing loss has just been approved by the Food and Drug Administration (FDA). The treatment, Otarmeni (lunsotogene parvec-cwha), is the first-ever dual adeno-associated ...

You might like the sound of this news. In a landmark move, the Food and Drug Administration on Thursday approved the first gene therapy for a rare, inherited form of hearing loss. And while these ...