Since launching their collaboration with CMT Research Foundation in November 2024, researchers at 82VS, the venture studio of Alloy Therapeutics, have made rapid progress and are ahead of schedule testing their novel technology focused on improving RNA therapeutic delivery to peripheral nerve cells affected by Charcot-Marie-Tooth disease.

A key challenge in RNA-based treatments, such as oligonucleotides, for CMT is delivering them effectively to the target tissues and cells. To address this, CMTRF funded 82VS to explore Alloy Therapeutics’ library of transferrin receptor (TfR1) antibodies as a potential solution. TfR1 antibodies have recently been shown to enhance delivery of oligonucleotide therapeutics to muscle, brain and peripheral nerves.

The team has successfully identified promising TfR1 antibodies and is now combining them with an oligonucleotide-based compound. Next, they will test these combined compounds in animal models to evaluate whether TfR1 antibodies enhance delivery to CMT-affected tissues and cell types.

Dr. Carlos Loya, the principal investigator of this project, presented some of these exciting findings at the Alloy Therapeutics 2025 R&D Day in March 2025.

CMTRF is encouraged by the progress so far and eagerly anticipates the results of the upcoming animal study.

For more information on this CMTRF-funded project, click here.