CMT Research Foundation Funds Project with Alloy Therapeutics’ 82VS to Test Novel RNA Therapeutics for CMT Disease

ATLANTA, GA, UNITED STATES, November 13, 2024 /EINPresswire.com/ -- The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)* 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.”

82VS is the venture studio of Alloy Therapeutics, empowering exceptional scientist-entrepreneurs to build the most capital-efficient drug companies in the world. Leveraging Alloy’s next-generation platforms and R&D services, along with seed capital, 82VS supports teams from ideation through to company creation, drug discovery, financing, and scaling of their businesses. Aligned with the collaborative ecosystem ethos at Alloy, 82VS partners with academics, entrepreneurs, patient advocacy groups, biotechnology companies, and other venture capital groups from the earliest stages of company creation to develop innovative medicines for patients.

CMT Research Foundation (CMTRF) is a patient-led, non-profit focused on delivering treatments and cures for CMT. The foundation identifies significant obstacles or deficiencies impeding progress toward a cure and seeks collaborators to address these issues. To date, CMTRF has funded 24 projects, of which 8 are completed. Of those 8 completed projects, 5 have clinical candidates. CMTRF's mission to invest in promising science with high potential leading to treatments and cures was proven effective and ground-breaking when DTx Pharma - with a CMTRF- backed program as its lead candidate -was acquired by Novartis for $1 billion. Founded by two patients who are driven to expedite drug delivery to people who live with CMT, the 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.

*Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms, causing walking, running and balance problems as well as abnormal functioning of hands and feet. CMT affects one in 2,500 people (about the same prevalence as cystic fibrosis), including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT.

George H Simpson
CMT Research Foundation
+1 203-521-0352
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