In a first for adults in Singapore, scientists are conducting gene editing trials on heart patients to correct defects at ...

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

The U.S. government is divesting from mRNA vaccines, but will other uses of the technology be spared? In a time of ...

Liz Wood, who lives in Beacon, is the founder of The MYT1L Project ( myraregene.org ), a foundation that supports families whose children are affected by a rare genetic disorder.

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

In honor of World Blindness Awareness Month, "GMA" is spotlighting a breakthrough gene therapy that's helping some blind ...

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

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 ...