Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

The FDA has approved Capsida Biotherapeutics' investigational new drug (IND) application for CAP-003 to treat Parkinson’s ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007 for Stargardt disease.

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

With FDA platform technology designation for its viral vector, SRPT can fast-track other therapies using the same ...