Cell Therapies For CMT

Cell therapies involve the transplantation of human cells into an individual in order to slow or halt progression of disease. A number of different cell types can be used — including various types of stem cells. If the therapeutic cells are taken from and returned to the same individual, they are considered autologous. If they instead originate from a donor, they are allogeneic.
In some cases, a hybrid approach is used that combines elements of both cell and gene therapy. Cells may be removed from a patient, genetically modified to include a new gene, and then returned to the patient from which they were taken.
The goal in cell therapy is for the transplanted cells to counteract the effects of missing or dysfunctional proteins. With the restoration of normal protein function, it may be possible to preserve, maintain or restore physical function.
CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.
Related News & Research
Dr. Grace Pavlath Joins CMT Research Foundation’s Research Advisory Committee
ATLANTA (November 14, 2023) The CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced that Dr. Grace Pavlath has joined its newly formed Research Advisory Committee...
Update from the Novartis CMT1A Development Team
As you know, the CMT Research Foundation is proud to have introduced DTx leadership to Charcot-Marie-Tooth disease in 2019. We funded their first work in CMT to demonstrate the FALCON platform's potential to deliver a drug to the Schwann cells. And from there, the...
ARM-101: A Potential “Precision Drug Therapy” For CMT1A
The CMT Research Foundation has invested in a project at Armatus Bio, Inc. that will advance a novel miRNA gene therapy candidate for CMT type 1A. Principal Investigator Dr. Brian Price and his colleagues aim to harness the potential of microRNAs (miRNAs), snippets of...
CMT Research Foundation Invests in Armatus Bio, Inc. to Advance Potential Therapeutic for CMT1A
ATLANTA (October 24, 2023) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a project at Armatus Bio that will advance a novel miRNA gene therapy candidate for CMT type...
CMT Research Foundation Launches Research Advisory Committee of Renowned Scientific Experts
ATLANTA (October 18, 2023) The CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced the formation of a Research Advisory Committee (RAC) consisting of: Dr. William...
Arthur Suckow, former Co-founder and Chief Executive Officer of DTx Pharma, Joins CMT Research Foundation Board of Directors
ATLANTA (October 3, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced that Arthur Suckow, Ph.D. former Co-founder and Chief Executive Officer of DTx...
Another Asset with Potential to Treat Charcot-Marie-Tooth has been Acquired by Pharma
An asset that is being tested in CMT4B1 underwritten by the CMT Research Foundation has been licensed by AcuraStem to Takeda for approximately $580 million. The exclusive, worldwide license will enable Takeda to develop and commercialize AcuraStem's PIKFYVE targeted...
Endgame Campaign for CMT1A Tops $6.6 Million
Recently, Peter J. deSilva, CMTRF Board member and Chair of the ENDGAME Capital Campaign to end CMT1A and Susan Ruediger, CMT patient, co-founder, and Chief Mission Officer of the CMTRF, hosted a private call with donors to update them on the progress being made in...
The CMT Research Foundation Launches Annual CMT Action Month with Special September of Events
ATLANTA, Sept 1, 2023 — To mark CMT Action Month this September, the CMT Research Foundation (CMTRF) today launched a month-long lineup of events, bringing together artists, advocates, and the CMT community from across the U.S. The series of events aim to raise...
W. David Arnold, MD Joins CMT Research Foundation Scientific Advisory Board
ATLANTA (August 14, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced that Dr. W. David (Dave) Arnold has joined the Foundation’s Scientific Advisory...

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