Methods for the treatment of Leber congenital amaurosis

US11492621B2 · kind B2 · utility

Assignees

• Institut National de la Sante et de la Recherche Medicale (INSERM) · FR
• CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE · FR
• GENETHON · FR
• UNIVERSITE DE PARIS DESCARTES · FR
• ENSCP—CHIMIE PARISTECH—ECOLE NATIONALE SUPERIEURE DE CHIME DE PARIS · FR
• ASSISTANCE PUBLIQUE—HÔPITAUX DE PARIS (APHP) · FR

Inventors

• Jean-Michel Rozet · Paris, FR
• Antoine Kichler · Molsheim, FR
• Isabelle Perrault · Paris, FR
• Josseline Kaplan · Paris, FR
• Xavier Gerard · Paris, FR
• Daniel Scherman · Paris, FR
• M. Arnold Munnich · Paris, FR

Key dates

• Filing date: Mar 3, 2020
• Grant date: Nov 8, 2022
• Expiry date: Mar 3, 2040

Classification

• Technology area (CPC Y): Emerging Cross-Sectional Technologies
• CPC primary: Y02A50/30
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c. 2991+1655 A>G CEP290 mRNA.