Investigators at Boston institutions believe the regimen could target immunosuppressive lipid macrophages and improve ...

A sponsor will have to demonstrate success with "with several consecutive patients with different bespoke therapies" to ...

Studies of germline genetic testing programs provide a snapshot of challenges at academic and community practices at the NSGC annual meeting.

The agency is cautioning patients about the risk of serious, possibly fatal, liver injuries and has limited the gene therapy's use to ambulatory patients.

CTX310 showed promising safety and efficacy in a small Phase I trial, according to data presented at the American Heart ...

The agency reviewed data from a Phase II study, in which around 21 percent of patients had a complete response to the menin inhibitor.

NEW YORK – Fondazione Telethon said Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for its gene therapy ...

NEW YORK – BostonGene on Wednesday said it has partnered with Kyoto University to identify biomarkers that can guide personalized treatment decisions for patients with esophageal squamous cell ...

The agency is establishing a network of care centers to expand access to regenerative medicine beyond regions served by major ...

Genetic testing could improve cancer outcomes for patients, but historical research abuses and access issues have slowed ...

The UK regulator is creating a new framework for how it will approve rare disease treatments, which it intends to publish in ...