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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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
One CRISPR Infusion Cut Triglycerides 63% in People With Severe Hypertriglyceridemia
At AHA 2026, CRISPR Therapeutics reported that one fixed dose of CTX310 lowered ANGPTL3 by 65% and triglycerides by 63% in 10 people with severe hypertriglyceridemia, with no treatment-related serious side effects.
1 hour ago
Beam's Base-Edited Sickle Cell Therapy Hits Its Pivotal Goal, Setting Up the First Base-Editing BLA
Beam Therapeutics says risto-cel met its pivotal endpoint of at least 12 months free of severe pain crises in the BEACON trial, and plans to seek FDA approval as early as the end of 2026.
1 hour ago
FDA Investigates Cancer Risk With Zolgensma After Vector DNA Found Next to a Tumor Gene
The FDA confirmed that Zolgensma's AAV vector inserted next to the GLI1 cancer gene in two children who developed tumors. Here's what was found and what it means for SMA families and AAV gene therapy.
3 days ago
FDA Finalizes Potency Assurance Guidance for Cell and Gene Therapies: What Changes
FDA's final potency assurance guidance replaces its 2011 potency test guidance and asks cell and gene therapy makers to build a risk-based strategy so every lot works as intended.
3 days ago
FDA Grants Priority Review to uniQure's Huntington's Gene Therapy AMT-130, Decision Due May 2027
The FDA accepted uniQure's BLA for AMT-130 under accelerated approval with Priority Review and a May 1, 2027 decision date. It could become the first gene therapy for Huntington's disease.
3 days ago
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