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LATEST NEWS
Estuary Launches With $103 Million to Bring Gene Therapy to Polycystic Kidney Disease
Estuary Biotherapeutics launched with $103 million to develop one-time, kidney-directed AAV gene therapies for ADPKD, the most common inherited kidney disease. Here is how restoring polycystin proteins could stop, or even reverse, kidney cysts.
3 days ago4 min read
Should Gene Therapy Be Used for Common Diseases? Two Cardiologists Propose Five Questions
One-time genetic treatments are moving from rare diseases to common ones like high cholesterol. In JAMA Internal Medicine, two University of Michigan cardiologists explain why permanence, rare side effects and cost call for a new framework.
3 days ago4 min read
Optogenetic Gene Therapy Gives Some Blind Patients Back a Sense of Light
In the NEJM-published PIONEER trial, an algae-derived light-sensing gene plus amber-light goggles improved light sensitivity in 6 of 10 people with late-stage retinitis pigmentosa, and 4 of 8 got better at finding objects. Here is how optogenetics works.
Oct 115 min read
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GENE TECH TIMES
The Future Has Arrived
Two Years In, Elevidys Shows Functional Benefit in Older Boys With Duchenne
Boys with Duchenne who received Elevidys between ages 8 and 12 kept more motor function over two years than matched untreated boys, Sarepta reported at the World Muscle Society Congress. Here is what the data show, and what they don't.
Oct 8
Sarepta Pauses Elevidys Gene Therapy Trial After Second Death
Sarepta Therapeutics has paused its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, after a second patient death due to acute liver failure. The pause affects ongoing trials and therapy shipments, raising safety concerns—especially for non-ambulatory patients. Approved under the FDA’s accelerated pathway, Elevidys aimed to slow DMD progression, but recent events have sparked debate over gene therapy risks, regulatory oversight, and treatment decisions for affected f
Jul 1, 2025
FDA Expands Approval of ELEVIDYS: A Breakthrough Gene Therapy for Duchenne Muscular Dystrophy
On June 20th, 2024, the FDA expanded its approval of ELEVIDYS, an innovative AAV-based gene therapy developed by Sarepta Therapeutics, for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged 4 years and older. This landmark decision marks a significant advancement in the management of DMD, a serious neuromuscular condition primarily affecting young boys and severely impacting their quality of life and lifespan. Understanding Duchenne Muscular Dystrophy DMD is
Jun 20, 2024
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