ATSN-101
- Generic Name
- ATSN-101
- Brand Names
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- Drug Type
- Biotech
- Chemical Formula
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- CAS Number
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- Unique Ingredient Identifier
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- Background
ATSN-101 is a adeno-associated virus serotype 5 encoding human retinal guanylate cyclase 1 gene (AAV5-GUCY2D)
- Associated Conditions
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- Associated Therapies
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Gene Therapy Reverses a Rare Cause of Vision Loss
Gene therapy, ATSN-101, improved vision 100 to 10,000 times in 15 LCA1 patients, reversing Leber congenital amaurosis caused by GUCY2D gene mutations. Vision improvements were rapid and lasting, with high-dose recipients experiencing significant enhancements. Side effects were minor and related to the injection procedure. A larger trial is planned for FDA approval.
Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/II Clinical
Atsena Therapeutics announced 12-month safety and efficacy data from its Phase I/II trial of ATSN-101, a gene therapy for Leber congenital amaurosis caused by biallelic mutations in GUCY2D, published in The Lancet. ATSN-101 demonstrated durable, clinically significant improvements in vision at the high dose and was well-tolerated. This marks the first gene therapy treatment for LCA1, with potential for a Phase III trial.
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