The CMT Research Foundation has invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate (AOC) therapeutics for CMT.
Oligonucleotide therapeutics offer promise for treating genetic disorders but have faced challenges as potential drugs for CMT due to limited delivery to the peripheral nerves and affected cell types. Transferrin receptor (TfR1) antibodies have recently been shown to enhance oligonucleotide delivery to muscle, brain, and peripheral nerves. This project by 82VS aims to combine Alloy Therapeutics’ novel TfR1 antibodies with oligonucleotides to improve delivery and efficacy in CMT-relevant tissues. If successful, this approach could enable treatments for all types of CMT.
“Combining next generation TfR1 antibody and oligonucleotide technologies offers a promising solution to a major obstacle that has hindered the advancement of RNA therapies for CMT, potentially leading to powerful and effective treatments” says Dr. Riann Egusquiza, CMTRF’s Director of Research. “Additionally, this project with 82VS will offer valuable insights into using TfR1 antibodies to target affected cell types in CMT, which could optimize delivery methods for various other CMT therapies.”
Alloy Therapeutics and 82VS actively partner with patient advocacy groups to tackle challenging diseases, recognizing that collaboration is key to advancing effective treatments. By combining scientific expertise with the insights and support of advocacy organizations, Alloy Therapeutics aims to accelerate the development of therapies that address unmet needs and improve people’s lives.
“This partnership between the CMT Research Foundation and 82VS is an innovative approach to tackling Charcot-Marie-Tooth disease,” says Mike Schmidt, General Partner of 82VS and CSO of Alloy Therapeutics. “Alloy’s combined expertise in antibody discovery and oligonucleotide development make us uniquely suited to develop these next-generation therapeutic approaches. We’re excited to partner with the patient advocacy expertise of CMTRF to pursue new medicines for CMT patients.”
