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enGene's Non-Viral Gene Therapy for Bladder Cancer Heads to the FDA
enGene's non-viral gene therapy detalimogene cleared detectable cancer in 56% of evaluated patients with BCG-unresponsive bladder cancer, and 86% kept their bladders. The company plans to start its FDA application by the end of 2026.
25 minutes ago3 min read
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.
2 days ago4 min read
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.
2 days ago4 min read
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The Future Has Arrived
enGene's Non-Viral Gene Therapy for Bladder Cancer Heads to the FDA
enGene's non-viral gene therapy detalimogene cleared detectable cancer in 56% of evaluated patients with BCG-unresponsive bladder cancer, and 86% kept their bladders. The company plans to start its FDA application by the end of 2026.
25 minutes ago
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.
2 days 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.
2 days 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.
5 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.
5 days ago
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