top of page
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.
1 day 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.
1 day 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
LEARN MORE
LEARN MORE
GENE TECH TIMES
The Future Has Arrived
FDA Launches New Pathway to Fast-Track Treatments for Ultra-Rare Diseases
The FDA unveils a “plausible mechanism pathway” to speed approval of innovative therapies for ultra-rare diseases, paving the way for personalized and gene-based treatments.
Feb 25
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
bottom of page