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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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
Casgevy, the First CRISPR Medicine, Expands to Children as Young as 2
The FDA expanded Vertex's CRISPR therapy Casgevy to children aged 2 and older with sickle cell disease or transfusion-dependent beta-thalassemia. Here's how it works and why the approval for toddlers is notable.
Jul 29
FDA Approves Stem Cell Therapy, Regenecyte, Shown to Help Chemotherapy Patients Recover Blood Count
The FDA has approved Regenecyte, an innovative therapy made from umbilical cord blood stem cells, to help rebuild blood and immune systems in patients with blood disorders or those recovering from intense treatments like chemotherapy. This breakthrough offers a promising option for patients needing healthy blood cell regeneration. Regenecyte uses stem cells collected from umbilical cords, which are highly adaptable and can grow into essential blood and immune cells. The treat
Nov 29, 2024
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