CMT Research Foundation
Patient-led non-profit organization focused on accelerating development of treatments and cures for Charcot‑Marie‑Tooth (CMT) disease by funding translational research, convening stakeholders, and connecting academic and industry partners.
Industries
N/A
Nr. of Employees
small (1-50)
CMT Research Foundation
4062 Peachtree Road, Suite A209, Atlanta, GA 30319
Services
Provides targeted funding, milestone management, and scientific oversight for preclinical and translational projects focused on CMT.
Annual multi‑day meeting that convenes scientists, clinicians, industry, investors and patients to share data and stimulate collaborations.
Volunteer network that supports fundraising, awareness, and local outreach for research initiatives.
Provides newly‑diagnosed information packages, clinical trial listings and guidance for patients seeking research participation.
Provides targeted funding, milestone management, and scientific oversight for preclinical and translational projects focused on CMT.
Annual multi‑day meeting that convenes scientists, clinicians, industry, investors and patients to share data and stimulate collaborations.
Volunteer network that supports fundraising, awareness, and local outreach for research initiatives.
Provides newly‑diagnosed information packages, clinical trial listings and guidance for patients seeking research participation.
Expertise Areas
- Translational research funding and portfolio management
- Gene therapy development for peripheral neuropathies
- Oligonucleotide and RNA therapeutic delivery to peripheral nerves
- Patient‑derived cellular models and high‑throughput screening
Key Technologies
- Lipid nanoparticle delivery
- Viral vector gene therapy
- Antisense oligonucleotides (ASOs)
- Oligonucleotide conjugation and receptor‑mediated delivery
News & Updates
Summary of INSPIRE Phase 2/3 results reporting sustained reductions in blood sorbitol, correlations between sorbitol reduction and functional and patient‑reported outcomes, exploration of MRI muscle‑fat fraction as a potential biomarker, and overall safety and tolerability through 24 months.
Announcement that the developer will meet with the U.S. Food and Drug Administration to discuss a potential New Drug Application for a small‑molecule therapy for CMT‑SORD; also notes launch of a free urine sorbitol assay program to support identification of affected patients.
Announcement of funding to support manufacturing launch for a gene therapy candidate targeting a CMT subtype.
Investment in a program to develop viral gene therapy candidate for CMT2D.
Progress report on a funded project exploring transferrin receptor antibodies to enhance oligonucleotide delivery to peripheral nerves.
Report describing industry licensing of a CMTRF‑funded asset and downstream commercialization activity.
Summary of INSPIRE Phase 2/3 results reporting sustained reductions in blood sorbitol, correlations between sorbitol reduction and functional and patient‑reported outcomes, exploration of MRI muscle‑fat fraction as a potential biomarker, and overall safety and tolerability through 24 months.
Announcement that the developer will meet with the U.S. Food and Drug Administration to discuss a potential New Drug Application for a small‑molecule therapy for CMT‑SORD; also notes launch of a free urine sorbitol assay program to support identification of affected patients.
Announcement of funding to support manufacturing launch for a gene therapy candidate targeting a CMT subtype.
Investment in a program to develop viral gene therapy candidate for CMT2D.
Progress report on a funded project exploring transferrin receptor antibodies to enhance oligonucleotide delivery to peripheral nerves.
Report describing industry licensing of a CMTRF‑funded asset and downstream commercialization activity.