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LATEST NEWS
FDA Proposes One Approval for Many Mutations in Personalized Gene Editing
A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.
Oct 294 min read
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.
Oct 224 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.
Oct 224 min read
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GENE TECH TIMES
The Future Has Arrived
CRISPR Gene-Editing Therapies Make Strides, but Cost and Accessibility Remain Key Challenges
As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their mark. One of the most exciting developments is Casgevy, a gene-editing therapy that has shown remarkable success in treating sickle-cell disease and β-thalassaemia. Approved in both the UK and US, Casgevy works by reactivating fetal hemoglobin, offering patients a chance at long-term relief with fewer blood transfusions and hospital visits. W
Dec 19, 2024
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