Abzielung auf Opsinexpression mit Mrna-Therapie zur Myopiekontrolle und Myopiebehandlung

Details

Veröffentlichungs-Nr.

EP4806453

Anmeldetag

14. März 2025

Veröffentlichung

16. September 2026

Rechtsraum

EP

Abstract

A synthetic mRNA molecule is provided comprising a sequence encoding an opsin protein for use in a therapeutic method for preventing and/or treating myopia. It was surprisingly found that a synthetic mRNA molecule is suitable for the treatment of myopia. Further, the synthetic mRNA molecule is provided for use in a therapeutic method to increase intracellular opsin expression. Further, a use of the synthetic mRNA molecule is provided for the manufacture of a pharmaceutical composition for treatment and/or prophylaxis of development of myopia. Further, a pharmaceutical formulation is provided containing the synthetic mRNA molecule. Moreover, a method is provided for manufacturing a pharmaceutical formulation comprising a mRNA molecule carrying the coding information for an opsin protein, the method comprising the steps of: - S1: Selecting a target opsin protein whole expression needs to be increased, - S2: Providing DNA encoding the amino sequence of the target opsin protein within an expression vector, - S3: Producing mRNA by in vitro transcription, - S4: Mixing the synthesized mRNA with at least one delivery agent, - S5: Formulating the mixture of the synthesized mRNA and the delivery agent into a pharmaceutical composition.

Anmelder

Firma

Carl Zeiss Vision International GmbH

Land

🇩🇪 Deutschland