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 ...

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 ...
Ophthalmology Times

FDA grants RMAT designation to RTx-015 optogenetic gene therapy for retinitis pigmentosa

RMAT designation enables intensified FDA engagement and potential accelerated pathways, implying preliminary clinical evidence for RTx-015 despite absent peer-reviewed efficacy details, endpoints, and ...

Rocket’s gene therapy Kresladi wins FDA approval in rare immune disease

Upon approval, Rocket has been awarded a priority review voucher, which currently fetch around $200m on the secondary market.
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 ...

FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...
Healio

Retinitis pigmentosa candidate receives regenerative medicine advanced therapy designation

The FDA granted regenerative medicine advanced therapy designation to RTx-015 for the treatment of retinitis pigmentosa.

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 affecting young children ...
UPI

FDA tightens Duchenne gene therapy after deaths of two teens

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...
BioSpace

Rocket’s Gene Therapy Wins FDA Greenlight, Clearing Way for Future Products

While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a ...
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), ...