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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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.
3 days ago4 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
Positive Phase 2 Results for Gene Therapy in Parkinson’s Disease
In exciting news for Parkinson’s disease research, MeiraGTx has announced positive results from a Phase 2 clinical bridging study of AAV-GAD, an innovative gene therapy. The study, which involved 26 weeks of evaluation included three participant groups: high dose, low dose, and a sham control group. The clinical trial aimed to assess both safety and effectiveness, showing promising results in both areas. Key Findings: AAV-GAD demonstrated a strong safety profile, with no seri
Oct 17, 2024
Magnets: The New Way to Control the Brain?
Imagine switching brain circuits on and off using only magnetic fields. Researchers have just developed a cutting-edge gene therapy that does exactly that—without any implants or invasive surgery. Traditionally, methods like optogenetics required fiber-optic implants to control neurons with light. Recently, a team from Weill Cornell Medicine, Rockefeller University, and the Icahn School of Medicine has created a simpler, non-invasive solution. This new approach, called "magne
Oct 14, 2024
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