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LATEST NEWS
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.
59 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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GENE TECH TIMES
The Future Has Arrived
Five-Year Follow-Up Shows Sustained Visual Improvements in LHON Patients Treated with AAV Gene Therapy
A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide long-lasting visual improvements for patients with Leber hereditary optic neuropathy (LHON). This genetic condition, caused by a mutation in the MT-ND4 gene, typically leads to severe vision loss. The study followed patients who received the therapy and found significant improvements in vision even five years after treatment. The therapy involv
Dec 27, 2024
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