Last May, people were thrilled to learn that a 7-month-old baby who became the first in the world to receive a personalized ...

Thousands of Californians live with sickle cell disease, a painful blood disorder. But new treatments, including a promising form of gene therapy, are offering hope to ...

Ocugen Inc. is currently conducting a Phase 3 clinical study titled ‘A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the ...

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 ...

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 ...

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 ...