Otarmeni, a surgically-delivered drug developed by Regeneron, recently received accelerated FDA approval to treat a rare, ...

Silver Spring, MD, June 02, 2026 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration (FDA) today issued draft guidance ...

UCSD study says the therapy increased expression of a neuroprotective protein to help preserve brain functions, despite the ...

FDA’s draft guidance allows sponsors to leverage public and platform knowledge for CMC, nonclinical, and clinical support of ...

Northwestern Medicine scientists have discovered that suppressing excitatory synaptic transmission in a small group of ...

The Allen Institute in Seattle says scientists have now learned enough about how the brain works to start fixing it when it ...

The FDA's decision follows data from a Phase Ib study on salanersen.

In a worldwide first, a one-and-done gene therapy has been approved to treat a form of hereditary deafness. Many of the children treated can hear normally and speak.

The Kleefstra Syndrome Foundation, a leading patient advocacy organization, today announced a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to ...

A key claim by the company MedTherapy is a major reduction in production timelines. While gene therapies typically take weeks ...

All Evaluable Patients Achieved Improvements in Multiple Hallmarks of MYBPC3-Associated HCM Benefits Among Cohort 1 Patients ...

The FDA’s press release noted that the guidance supports development of an array of cell and gene therapy products, including ...