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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.
2 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.
2 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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Orforglipron Weight-Loss Pill Trial Results: Eli Lilly Reports 12.4% Body Weight Reduction, Shares Fall 15%
Eli Lilly’s once-daily oral weight-loss pill, orforglipron, helped patients lose 12.4% of their body weight in a Phase 3 trial, but fell short of rival Wegovy’s results. The announcement sent Lilly shares down 15%, the company’s steepest drop in 25 years, despite meeting all primary and secondary endpoints and showing improvements in heart health markers.
Aug 11, 2025
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