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Charcot-Marie Tooth Research Foundation

4062 Peachtree Road NE, Suite A209, Atlanta, GA, 30319, USA

Overview

The CMT Research Foundation is a patient-led non-profit dedicated to developing treatments and cures for Charcot-Marie-Tooth disease.

Total investments
3
Lead investments
0
Investments · 12mo
0
Active investors
2
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Investment portfolio

  • Augustine Therapeutics

    Participated · Series A · Mar 2025

    Augustine Therapeutics develops selective inhibitors of the cytosolic histone deacetylase 6 (HDAC6) enzyme for chronic indications. Its lead program, AGT-100216, is described as the first selective HDAC6 inhibitor designed for long-term treatment of Charcot-Marie-Tooth (CMT) disease. The company highlights a non-hydroxamate, non-hydrazide chemotype intended to provide selectivity and avoid limitations of other chemotypes, and says the approach is built for chronic diseases. Augustine plans to use newly raised capital to advance AGT-100216 through a Phase I/II proof-of-concept clinical trial in CMT. The company targets neuromuscular, neurodegenerative and cardio-metabolic diseases with this selective HDAC6 approach. Augustine is led by CEO Gerhard Koenig and was founded on research by Prof. Ludo Van Den Bosch at VIB-KU Leuven in Belgium. The recent financing strengthens its balance sheet to support clinical development. Augustine Therapeutics is developing novel, potent, subtype-selective small-molecule inhibitors of the cytosolic HDAC6 enzyme targeting neurodegenerative and cardiometabolic diseases. Its lead candidate, AGT100216, is a peripherally restricted selective HDAC6 inhibitor that has shown impressive preclinical efficacy in Charcot‑Marie‑Tooth (CMT) studies and peripheral neuropathies induced by chemotherapy (CIPN). The company’s chemistry is distinct from first-generation hydroxamate-based HDAC6 inhibitors and is designed to safely and selectively reverse pathophysiological changes associated with neuromuscular and neurodegenerative diseases. Augustine plans to advance AGT100216 into a Phase 1/2 first-in-human clinical trial in 2025 and to develop next-generation candidates with peripheral-restricted or brain-penetrant properties. Proceeds from the financing will also support expansion of the executive and R&D teams. Augustine was founded in 2019 as a VIB-KU Leuven spin-off and is based in Leuven, Belgium. Augustine Therapeutics is focused on the discovery and development of innovative therapeutics for Charcot‑Marie‑Tooth disease (CMT) and other neuromuscular disorders. The company is rooted in research from the VIB–KU Leuven labs of Ludo Van Den Bosch and collaborations between the labs of Joris de Wit and Bart De Strooper, which uncovered biological pathways and therapeutic targets in peripheral neuropathies. VIB Discovery Sciences is leading the preclinical development of Augustine’s new therapeutics and will apply industry-trained drug discovery expertise to the early pipeline. Augustine completed a €4.2 million seed financing to support target validation and early-stage development. Day-to-day interim management is being handled by Ward Capoen (V-Bio Ventures) and Jérôme Van Biervliet (VIB) while the company seeks a dedicated management team. The company aims to advance validated targets toward novel treatments that address the limited therapeutic options for CMT patients.

  • ReviR Therapeutics

    Participated · Series A · Jul 2024

    ReviR Therapeutics develops small-molecule RNA-targeting therapies using an AI-driven discovery approach. The company leverages its VoyageR platform to discover and optimize chemical matter against RNA targets, including splice modulation technologies. Its initial therapeutic focus includes Huntington’s disease, Charcot‑Marie‑Tooth disease, amyotrophic lateral sclerosis, and other neurological and rare genetic disorders with limited treatment options. ReviR plans to further augment the VoyageR platform and advance proprietary programs toward clinical development. The company is led by CEO Dr. Peng Yue and was founded in 2021 in Brisbane, CA. Financially, ReviR closed a $30M Series A in the reported round, bringing total capital raised to $54M.

Team

  • Patrick Livney

    Founder & Chair Emeritus CMT Research Foundation

    LinkedIn
  • Susan Ruediger

    Founder, Chief Mission Officer & Board Member

    LinkedIn