Gene Therapy Trans-Splicing OPA1 Mutations
Summary
Centre National de la Recherche Scientifique has filed USPTO Patent Application US20260109985A1 (application No. 19116221, originally filed September 29, 2023) covering pre-mRNA trans-splicing molecules (RTMs) designed to correct mutations in the OPA1 gene, with claims extending to both ex vivo and in vivo gene therapy methods for treating diseases associated with OPA1 mutations. The application names five inventors: Yannick Le Dantec, Guy Lenaers, Olivier Baris, Arnaud Chevrollier, and Salim Khiati. CPC classifications include C12N 15/1137 (DNA/RNA therapeutic compositions) and C12N 15/86 (viral vectors). This publication makes the application publicly available for prior art and freedom-to-operate analysis; no patent rights are granted until the application is examined and allowed.
“The present invention provides pre-mRNA trans-splicing molecules (RTMs) that are useful for correcting mutations in the OPA1 gene.”
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GovPing monitors USPTO Patent Applications - Biotech (C12N) for new healthcare & life sciences regulatory changes. Every update since tracking began is archived, classified, and available as free RSS or email alerts — 264 changes logged to date.
What changed
The USPTO has published Patent Application US20260109985A1 by Centre National de la Recherche Scientifique, covering pre-mRNA trans-splicing molecules (RTMs) for correcting OPA1 gene mutations and methods of using those RTMs in gene therapy for associated diseases. The application claims both ex vivo and in vivo therapeutic approaches. Inventors named are Yannick Le Dantec, Guy Lenaers, Olivier Baris, Arnaud Chevrollier, and Salim Khiati.
Competitors and researchers in the gene therapy or rare disease space should treat this publication as a prior art reference and conduct freedom-to-operate analyses if developing similar trans-splicing technologies targeting mitochondrial or optic-nerve-related genetic disorders. No patent rights are granted at this stage; the application remains pending subject to USPTO examination.
Archived snapshot
Apr 23, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
THERAPY BY TRANS-SPLICING OF OPA1 PRE-MESSENGER RNAS FOR THE TREATMENT OF DISEASES ASSOCIATED WITH OPA1 GENE MUTATIONS
Application US20260109985A1 Kind: A1 Apr 23, 2026
Assignee
Centre National de la Recherche Scientifique
Inventors
Yannick LE DANTEC, Guy LENAERS, Olivier BARIS, Arnaud CHEVROLLIER, Salim KHIATI
Abstract
The present invention provides pre-mRNA trans-splicing molecules (RTMs) that are useful for correcting mutations in the OPA1 gene. Also provided are methods of using the RTMs as gene therapy (e.g., ex vivo and in vivo gene therapy) for the treatment or prevention of diseases or disorders associated with OPA1 mutations.
CPC Classifications
C12N 15/1137 C12N 15/86 C12Y 306/05005 C12N 2320/33 C12N 2750/14143
Filing Date
2023-09-29
Application No.
19116221
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