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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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GENE TECH TIMES
The Future Has Arrived
Sickle Cell Disease Gene Therapy is Now COVERED under Medicare and Medicaid
Sickle cell disease (SCD), which affects over 100,000 people in the U.S., is about to see a major shift in treatment accessibility. With the staggering prices of $2.8 million for LYFGENIA and $2.1 million for CASGEVY, these treatments were inaccessible to many patients. On November 4th, 2024, The Biden-Harris Administration announced that these treatments would now be covered under Medicare and Medicaid, following agreements with the Centers for Medicare and Medicaid Services
Dec 5, 2024
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