EDIT-101
- Generic Name
- EDIT-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
EDIT-101 is a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene editing constructs.
- Associated Conditions
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- Associated Therapies
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Participants of pioneering CRISPR gene editing trial see vision improvement
79% of participants in the BRILLIANCE trial showed improvement after receiving CRISPR-based gene editing for a rare blindness form, LCA Type 10. EDIT-101, targeting the CEP290 gene mutation, was safe and effective, with 11 out of 14 participants experiencing vision and life quality improvements.
Editas Medicine, Inc. (NASDAQ:EDIT) Q4 2022 Earnings Call Transcript
Editas Medicine reported Q4 2022 earnings, highlighting clinical progress with EDIT-301 for sickle cell disease and beta thalassemia, strategic shifts towards in-vivo gene editing, and financial stability extending into 2025. The company aims to advance its gene editing technologies and clinical trials, focusing on delivering transformative medicines.
First CRISPR therapy dosed
An adult with congenital blindness received the first in vivo CRISPR-based therapy, EDIT-101, targeting a CEP290 gene mutation causing Leber congenital amaurosis. This therapy uses AAV5 with guide RNAs and Cas9 enzyme to correct the mutation, differing from Luxturna, which introduces a correct gene copy for RPE65 mutations.
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