Deafness reversed: Scientists restore hearing in 10 patients with a single-injection gene therapy breakthrough

A groundbreaking gene therapy trial has successfully restored hearing in individuals with a rare form of congenital deafness.

UF scientist develops safer gene therapy to restore vision

UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin this year.
BioPharma Dive

Rocket gene therapy cleared by FDA for rare immune disorder

Though the therapy, Kresladi, isn’t seen as a big seller, its clearance is a step forward for a company that’s lost most of ...
European Medical Journal

EBMT 2026: Gene Therapy Adoption Gaps in Sickle Cell Care

Gene therapy for sickle cell disease shows strong clinician support but faces barriers in referral practices, knowledge, and ...
CT Insider on MSN

CT researchers explore gene therapy that shows promise to slow aging, fight cancer

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients.

UCLA-led gene therapy clinical trial leads to FDA approval for rare immune disorder

A groundbreaking gene therapy tested through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...

Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease ...
Fierce Pharma

Rocket plots measured trajectory for new gene therapy Kresladi after clearance to launch from FDA

With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit.  | Rocket ...
Medscape

Gene Therapy to Be Studied for Treating Type 1 Diabetes

A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.

Saudi Hospital KFSH Successfully Implements Gene Therapy for Sickle Cell Disease

The milestone reflects KFSH’s ongoing efforts to advance its gene therapy program, bridging clinical care with translational ...
BioSpace

Sarepta Pushes Past Gene Therapy Debacle as RNA Therapies Show Early Promise

Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong ...
STAT

FDA approves Rocket gene therapy for rare immune disorder

The Food and Drug Administration approved on Thursday a gene therapy for severe leukocyte adhesion deficiency type 1 (LAD-1), ...