DTx-1252
An investigational treatment for Charcot-Marie-Tooth Disease.
The same compound appears under different names depending on the context. Here is how to identify DTx-1252 wherever you encounter it, plus the key facts at a glance.
- Generic name
- DTx-1252
- Development codes
- DTx-1252, DTX-1252
- Drug class
- siRNA (PMP22-targeting, FALCON-conjugated)
- Manufacturer
- Novartis (acquired DTx Pharma in 2023)
- How it's taken
- Designed for subcutaneous (under-the-skin) injection.
An investigational siRNA therapy designed to lower PMP22 expression in CMT1A using DTx Pharma's FALCON conjugation platform. Originally developed by DTx Pharma, acquired by Novartis in 2023 (announced July 17, 2023; completed October 2023) in a deal worth up to $1 billion ($500 million upfront plus $500 million in milestones). DTx-1252 is one of several next-generation PMP22-targeted programs aimed at the most common form of CMT.
Where DTx-1252 fits
Investigational, not approved. One of several PMP22-targeted programs in development for CMT1A, alongside other antisense and gene-therapy approaches at various stages. Only relevant for patients with confirmed CMT1A (PMP22 duplication on genetic testing), so confirming subtype with a broad gene panel is essential.
How DTx-1252 works
CMT1A is caused by an extra copy of the PMP22 gene, which makes nerve cells produce too much of the PMP22 protein. The protein imbalance damages myelin, the insulation around the nerves, and over years to decades produces the foot drop, leg weakness, and hand weakness that define CMT1A.
Small interfering RNAs (siRNAs) are short, lab-made double-stranded RNA molecules designed to find and silence a specific piece of messenger RNA (mRNA) inside cells. When DTx-1252's siRNA binds the PMP22 mRNA, it triggers the cell's natural RNA-interference pathway to break that mRNA down before it can be turned into protein. The result is lower PMP22 protein levels, closer to what the cell would normally produce.
The technical challenge with siRNA therapies is getting them into the right cells. Schwann cells, which make peripheral nerve myelin, are not easy to reach with standard siRNA approaches. DTx Pharma's FALCON (Fatty Acid Ligand Conjugated OligoNucleotide) platform attaches the siRNA to a fatty acid chain that helps the molecule cross into Schwann cells more efficiently. Novartis acquired DTx Pharma in 2023 partly for access to that platform.
Mechanism: Small interfering RNA (siRNA) conjugated to a fatty acid ligand using DTx Pharma's FALCON platform. The siRNA is designed to lower PMP22 mRNA in Schwann cells (the cells that make myelin around peripheral nerves), correcting the gene-dosage imbalance that causes CMT1A. The fatty acid conjugation improves delivery to peripheral nerves.
Side effects and safety
Because DTx-1252 is in early-stage clinical development, the safety profile is still being established. As a class, siRNA-based therapies have been associated with injection-site reactions, transient flu-like symptoms after dosing, and in some products liver enzyme elevations or platelet count changes that require monitoring. The trial team will share complete safety information from the Phase 1 study when results are reported.
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 DTx-1252
Designed for subcutaneous (under-the-skin) injection. Specific dosing and dosing interval are being established through the Phase 1 trial.
Clinical trial results
Novartis has not published trial details under the DTx-1252 name. A Novartis first-in-human Phase 1 study of a drug called EDK060 in adults with CMT1A (NCT07140614) began in September 2025, but Novartis has not publicly confirmed whether EDK060 is DTx-1252. CMT1A patients interested in participating should monitor ClinicalTrials.gov, the Hereditary Neuropathy Foundation, and CMT Research Foundation announcements for the next enrollment phase.
Development history
DTx Pharma was a small biotech developing the FALCON oligonucleotide platform with peripheral nerve targeting as a key application. In October 2023, Novartis acquired DTx Pharma for $500 million upfront plus up to $500 million in milestone payments, primarily to access the FALCON platform and the DTx-1252 program. Since the acquisition, the program has been advanced under Novartis's neuroscience and rare disease group.
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Common questions about DTx-1252
▸What is DTx-1252?
An investigational siRNA therapy designed to lower PMP22 expression in CMT1A using DTx Pharma's FALCON conjugation platform. Originally developed by DTx Pharma, acquired by Novartis in 2023 (announced July 17, 2023; completed October 2023) in a deal worth up to $1 billion ($500 million upfront plus $500 million in milestones). DTx-1252 is one of several next-generation PMP22-targeted programs aimed at the most common form of CMT.
▸How does DTx-1252 work?
CMT1A is caused by an extra copy of the PMP22 gene, which makes nerve cells produce too much of the PMP22 protein. The protein imbalance damages myelin, the insulation around the nerves, and over years to decades produces the foot drop, leg weakness, and hand weakness that define CMT1A.
Small interfering RNAs (siRNAs) are short, lab-made double-stranded RNA molecules designed to find and silence a specific piece of messenger RNA (mRNA) inside cells. When DTx-1252's siRNA binds the PMP22 mRNA, it triggers the cell's natural RNA-interference pathway to break that mRNA down before it can be turned into protein. The result is lower PMP22 protein levels, closer to what the cell would normally produce.
The technical challenge with siRNA therapies is getting them into the right cells. Schwann cells, which make peripheral nerve myelin, are not easy to reach with standard siRNA approaches. DTx Pharma's FALCON (Fatty Acid Ligand Conjugated OligoNucleotide) platform attaches the siRNA to a fatty acid chain that helps the molecule cross into Schwann cells more efficiently. Novartis acquired DTx Pharma in 2023 partly for access to that platform.
▸What are the side effects of DTx-1252?
Because DTx-1252 is in early-stage clinical development, the safety profile is still being established. As a class, siRNA-based therapies have been associated with injection-site reactions, transient flu-like symptoms after dosing, and in some products liver enzyme elevations or platelet count changes that require monitoring. The trial team will share complete safety information from the Phase 1 study when results are reported.
▸How is DTx-1252 taken?
Designed for subcutaneous (under-the-skin) injection. Specific dosing and dosing interval are being established through the Phase 1 trial.
▸Is DTx-1252 FDA approved?
DTx-1252 is currently in phase 1 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.
- Novartis · October 4, 2023. Novartis to acquire DTx Pharma to expand neuroscience pipeline with novel siRNA platform. https://www.novartis.com/news/media-releases/novartis-acquire-dtx-pharma-expand-neuroscience-pipeline-novel-sirna-platform
- CMT Research Foundation. Gene Therapy. https://cmtrf.org/gene-therapy/
- ClinicalTrials.gov. A First-in-human Study of EDK060 in Adults With Charcot-Marie-Tooth Type 1A (CMT1A). https://clinicaltrials.gov/study/NCT07140614