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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.
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
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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
Eli Lilly Acquires Verve Therapeutics for $1.3 Billion to Advance Gene Editing for Heart Disease
Eli Lilly has announced its $1.3 billion acquisition of Verve Therapeutics, a leading biotech company specializing in gene editing for cardiovascular diseases. This strategic move aims to accelerate the development of one-time gene therapies using base editing technology, targeting high-risk heart disease patients. The deal marks a significant step forward in Eli Lilly’s expansion into precision genetic medicine.
Jul 20, 2025
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