A GROUNDBREAKING treatment based on research by Spanish scientists has helped children born with incurable deafness hear for ...

In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the ...

Cathy Tie, who launched her first biotech in SF's IndieBio, now leads Manhattan Genomics in controversial push to edit human ...

A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...

Medication has long been the cornerstone of treatment for people with epilepsy, but it doesn’t stop seizures for everyone and ...

As of May 2025, RGX-202 displayed favourable safety, with no serious adverse events (SAEs) and adverse events of special ...

Nonfactor therapies may reduce costs associated with bleeding-related hospitalizations and use of bypassing agents. One study ...

Duke Eye Center has become the first academic medical center in the country to implant a new cell-based gene therapy for a ...

Intellia Therapeutics, Inc. is rated Hold due to uncertainty from dosing pause in its MAGNITUDE program for ATTR. Learn more ...

The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...

Specialty drugs, especially biologics, will constitute a significant portion of global drug spending by 2029, driven by cancer care costs. Autoimmune diseases and multiple sclerosis are key short-term ...

Seven years after the first gene-edited babies were revealed, biotech startup Manhattan Genomics is reviving the idea of ...