UniQure plans to seek FDA accelerated approval for its Huntington's disease gene therapy, using three-year data from a Phase 1/2 study. This follows a recent FDA meeting that reversed previous criticisms of the evidence, with shares soaring 70%.
A gene therapy for Huntington's disease, AMT-130, has a renewed path to FDA approval after the departure of Vinay Prasad, who had previously blocked the therapy's accelerated approval process. The FDA has once again agreed that UniQure can pursue approval with trial data using standard care patients as controls, avoiding sham surgeries.
Scientists at ShanghaiTech University have developed CS-101, a base-editing gene therapy that successfully cured five Chinese patients with beta-thalassaemia. The treatment restored haematopoietic function, raising total haemoglobin and fetal haemoglobin levels, freeing patients from lifelong blood transfusions. China has 30 million thalassaemia gene carriers while the global total reaches 350 million.
Regeneron will provide its newly approved gene therapy Otarmeni for free in the US, treating a rare genetic hearing loss caused by faulty OTOF gene. In clinical trials, 80% of patients experienced hearing improvements. CEO Leonard Schleifer said other countries have not yet been priced but should contribute their fair share. About 50 babies annually are born with this condition in the US.
Regeneron agreed to lower U.S. drug prices for some Americans under a deal with President Trump, part of his 'most favored nation' effort to align U.S. prices with lower international rates. The biotech company will also offer its newly FDA-approved hearing-loss gene therapy Otarmeni for free to eligible patients. The agreements exempt companies from tariffs for three years, including planned up to 100% levies on pharmaceuticals. The FDA approved Otarmeni under its National Priority Voucher program for an ultra-rare genetic condition causing deafness.
The FDA approved the first gene therapy to restore hearing for people born deaf, developed by Regeneron Pharmaceuticals. The treatment targets a rare genetic defect affecting about 50 U.S. children annually, caused by a defective OTOF gene. In clinical trials, 80% of patients achieved significant hearing restoration and 42% gained normal hearing, with effects lasting at least two years.
Six-year-old Saffie Sandford from Stevenage has received life-changing Luxturna gene therapy for Leber's Congenital Amaurosis (LCA) at Great Ormond Street Hospital. The treatment, the first of its kind for one genetic cause of LCA, involved injections into each eye in April and September 2025. Without treatment, Saffie would have been blind by age 30. Her mother Lisa described it as "like someone waved a magic wand and restored her sight in the dark."
Saffie's mum says Luxturna therapy at Great Ormond Street has been like "someone waved a magic wand".
A six-year-old girl has had her sight restored thanks to a life-changing gene therapy on the NHS.
Researchers at Mass Eye and Ear reported that an experimental gene therapy restored hearing in about 90% of 42 patients born with DFNB9, a rare genetic deafness caused by OTOF gene mutations. The treatment uses an adenovirus to deliver a healthy gene to the inner ear, with results lasting more than two years in some patients. A Regeneron-developed treatment could become the first FDA-approved gene therapy for deafness.
