EN001 (mesenchymal stem cell therapy)
An investigational treatment for Charcot-Marie-Tooth Disease.
The same compound appears under different names depending on the context. Here is how to identify EN001 (mesenchymal stem cell therapy) wherever you encounter it, plus the key facts at a glance.
- Generic name
- EN001 (mesenchymal stem cell therapy)
- Development codes
- EN001, EN-001
- Drug class
- Mesenchymal stem cell therapy (intravenous)
- Manufacturer
- ENCell (Republic of Korea)
- How it's taken
- Given as an intravenous (IV) infusion in a clinic or infusion center.
An investigational mesenchymal stem cell therapy in development by ENCell (a Korean biotech) for CMT1A. A Phase 1 trial in 9 adults with genetically confirmed CMT1A was completed in 2024 and showed the therapy was well tolerated, with measurable improvements in CMT Neuropathy Score, walking tests, and nerve conduction. Phase 1b/2a is enrolling. The FDA granted EN001 Orphan Drug Designation for CMT in March 2025.
Where EN001 (mesenchymal stem cell therapy) fits
Investigational, not approved. The most clinically advanced cell-therapy program for CMT1A. Different in approach from gene therapies and PMP22-modulating drugs because it works through MSC paracrine effects rather than addressing the genetic defect. Only relevant for patients with confirmed CMT1A (PMP22 duplication on genetic testing).
How EN001 (mesenchymal stem cell therapy) works
Mesenchymal stem cells (MSCs) are a type of adult stem cell that can be grown in laboratories from sources like bone marrow or umbilical cord. They have been studied in many different diseases for their ability to send anti-inflammatory and tissue-supportive signals (so-called paracrine effects) to nearby cells. They generally do not work by transforming into the damaged cell type and replacing it.
In CMT1A, the peripheral nerves and the Schwann cells that produce myelin around them are progressively damaged because of an extra copy of the PMP22 gene. EN001 is a specific MSC-based therapy that ENCell developed in the lab and tested in animal models of CMT1A, where it was shown to help support myelin and improve some measures of nerve function.
In the Phase 1 trial, EN001 was given as a single intravenous infusion at one of two doses. Patients showed improvements in standard CMT measures (CMT Neuropathy Score, 10-Meter Walking Test, nerve conduction) at 16 weeks, with the high-dose group benefiting more. Whether these improvements are durable and whether they can be sustained with repeat dosing are questions the Phase 1b/2a trial is designed to answer.
Mechanism: Investigational mesenchymal stem cell therapy delivered by intravenous infusion. The mesenchymal stem cells are believed to support nerve and Schwann cell health through paracrine effects (signaling molecules they release after infusion) rather than by replacing damaged cells directly.
Side effects and safety
In the Phase 1 trial in 9 patients, EN001 was well tolerated, with no dose-limiting or serious adverse events and no infusion-related reactions. Mesenchymal stem cell therapies as a class are generally considered low-risk for systemic toxicity, though longer-term and larger-scale data are still being collected for EN001 specifically.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking EN001 (mesenchymal stem cell therapy)
Given as an intravenous (IV) infusion in a clinic or infusion center. Phase 1 used a single infusion at one of two doses (0.5 million or 2.5 million stem cells). Phase 1b/2a dosing protocols are being defined.
Clinical trial results
Phase 1 enrolled 9 adults with genetically confirmed CMT1A, randomized to a single IV infusion of EN001 at 0.5 million or 2.5 million cells. Results were presented at the Peripheral Nerve Society annual meeting in Montreal in 2024. The CMT Neuropathy Score version 2 dropped by an average of 2.89 points across all patients at 16 weeks (3.5 points in the high-dose group), with improvements also reported on the 10-Meter Walking Test, the Functional Disability Scale, and nerve conduction studies. The Phase 1b/2a trial (NCT06328712) began in May 2024 and plans about 27 patients in total, including a 21-patient Phase 2a part that compares 2 doses with placebo on the CMT Neuropathy Score at 24 weeks.
Development history
ENCell developed EN001 specifically for CMT1A, building on preclinical work showing mesenchymal stem cells could support nerve and Schwann cell function in animal models. After completing Phase 1 in Korea, ENCell received Orphan Drug Designation from the U.S. FDA for EN001 in CMT in March 2025. The Phase 1b/2a trial is enrolling under approval from the Korea Food and Drug Administration.
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Common questions about EN001 (mesenchymal stem cell therapy)
▸What is EN001 (mesenchymal stem cell therapy)?
An investigational mesenchymal stem cell therapy in development by ENCell (a Korean biotech) for CMT1A. A Phase 1 trial in 9 adults with genetically confirmed CMT1A was completed in 2024 and showed the therapy was well tolerated, with measurable improvements in CMT Neuropathy Score, walking tests, and nerve conduction. Phase 1b/2a is enrolling. The FDA granted EN001 Orphan Drug Designation for CMT in March 2025.
▸How does EN001 (mesenchymal stem cell therapy) work?
Mesenchymal stem cells (MSCs) are a type of adult stem cell that can be grown in laboratories from sources like bone marrow or umbilical cord. They have been studied in many different diseases for their ability to send anti-inflammatory and tissue-supportive signals (so-called paracrine effects) to nearby cells. They generally do not work by transforming into the damaged cell type and replacing it.
In CMT1A, the peripheral nerves and the Schwann cells that produce myelin around them are progressively damaged because of an extra copy of the PMP22 gene. EN001 is a specific MSC-based therapy that ENCell developed in the lab and tested in animal models of CMT1A, where it was shown to help support myelin and improve some measures of nerve function.
In the Phase 1 trial, EN001 was given as a single intravenous infusion at one of two doses. Patients showed improvements in standard CMT measures (CMT Neuropathy Score, 10-Meter Walking Test, nerve conduction) at 16 weeks, with the high-dose group benefiting more. Whether these improvements are durable and whether they can be sustained with repeat dosing are questions the Phase 1b/2a trial is designed to answer.
▸What are the side effects of EN001 (mesenchymal stem cell therapy)?
In the Phase 1 trial in 9 patients, EN001 was well tolerated, with no dose-limiting or serious adverse events and no infusion-related reactions. Mesenchymal stem cell therapies as a class are generally considered low-risk for systemic toxicity, though longer-term and larger-scale data are still being collected for EN001 specifically.
▸How is EN001 (mesenchymal stem cell therapy) taken?
Given as an intravenous (IV) infusion in a clinic or infusion center. Phase 1 used a single infusion at one of two doses (0.5 million or 2.5 million stem cells). Phase 1b/2a dosing protocols are being defined.
▸Is EN001 (mesenchymal stem cell therapy) FDA approved?
EN001 (mesenchymal stem cell therapy) is currently in phase 2 clinical trials for Charcot-Marie-Tooth Disease. It has not yet received FDA approval.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- ENCell (Business Wire) · March 6, 2025. ENCell's EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease. https://www.businesswire.com/news/home/20250306675394/en/ENCells-EN001-Receives-Orphan-Drug-Designation-from-the-U.S.-FDA-for-Charcot-Marie-Tooth-Disease
- ENCell (Business Wire) · June 24, 2024. ENCell Presents Phase 1 Clinical Trial Results of Mesenchymal Stem Cell Therapy EN001 for Charcot-Marie-Tooth Disease at PNS Conference. https://www.businesswire.com/news/home/20240624485930/en/ENCell-Presents-the-Phase-1-Clinical-Trial-Results-of-their-Mesenchymal-Stem-Cell-Therapy-EN001-for-Charcot-Marie-Tooth-Disease-at-the-PNS-conference
- Charcot-Marie-Tooth Association. ENCell Announces Phase 1b/2a Stem Cell Trial for CMT1A. https://cmtausa.org/simply-cmt/encell-phase-1b-2a-trial/
- ClinicalTrials.gov. A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients With CMT1A. https://clinicaltrials.gov/study/NCT06328712