Revir’s RTX-117 designated orphan drug for Charcot-Marie-Tooth disease

Oct. 14, 2025

Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.

BioWorld Science Regulatory Neurology/psychiatric FDA Orphan drug

Today's news in brief

BioWorld

BioWorld briefs for Dec. 5, 2025.

Today's news in brief

BioWorld MedTech

BioWorld MedTech briefs for Dec. 5, 2025.

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BioWorld

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Illustration of chromosome unraveling down to the DNA

First phase of synthetic human genome project successfully completes

BioWorld

The first phase of the U.K. synthetic human genome project has successfully completed, realizing key steps in chromosome synthesis. The work has demonstrated a...