Myotonic dystrophy drug development pipeline

Last modified: April 8, 2026

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Active pipeline

Development Phase	Program	Modality	DM Subtype	Drug Target	Community Updates
PRE P1 P2 P3 P4 Phase 3 Clinical trial	Tideglusib	Small Molecule	DM1, CDM	Glycogen synthase kinase 3 beta (GSK3ß)	2024 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 3 Clinical trial	AOC 1001 del-desiran	Antibody Conjugated Oligonucleotide	DM1	DMPK	2025 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 3 Clinical trial	Mexiletine	Small Molecule	DM1, DM2	Sodium Channels	2025 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 3 Clinical trial	Dyne 101 z-basivarsen	Antibody Fragment Conjugated Antisense Oligonucleotide	DM1	DMPK	2025 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 2 Clinical trial	ATX-01	Antisense microRNA Oligonucleotide	DM1	MBNL via miR-23b	2025 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 2 Clinical trial	Pitolisant	Small Molecule	DM1	Histamine-3 receptor (H3R)	2023 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 2 Clinical trial	PGN-EDODM1	Peptide Conjugated Antisense Oligonucleotide	DM1	DMPK	2025 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 2 Clinical trial	JUV-161	Stem Cell-Secreted Proteins	DM1	-	2025 Update Meet the DM Drug Developers
PRE P1 P2 P3 P4 Phase 2 Clinical trial	VX-670	Peptide Conjugated Oligonucleotide	DM1	DMPK	2025 Update
PRE P1 P2 P3 P4 Phase 1 Clinical trial	-	miRNA Technology in Adeno-Associated Virus		DMPK	2025 Update
PRE P1 P2 P3 P4 Phase 1 Clinical trial	SRP-1003	"Investigational RNA interference (RNAi) Therapeutic"	DM1	r(CUG) of DMPK
PRE P1 P2 P3 P4 Pre-clinical	ATX-01	Antisense microRNA Oligonucleotide	DM2, CDM	MBNL via miR-23b	2025 Update Meet the DM Drug Developers

Past efforts

Company	Company Status	DM Program	Notes
	Closed	Unknown	Company closed in 2022; regenerative medicine focus included DM in early-stage research.
	Active	Sold / Outlicensed	DM program acquired by Sarepta in 2025.
	Active	Unknown	Gene therapy research for DM ended; company acquired by Bayer in 2020. Company operates as subsidiary of Bayer.
	Acquired	Active	Acquired by Astellas in 2020; DM programs carried forward into Astellas pipeline.
	Active	Unknown	Ionis partnership for antisense therapy ended after failed Phase I/II trial in 2015; published study.
	Active	Unknown	Acquired Prosensa in 2014 with myotonic dystrophy program; no activity.
	Acquired	Unknown	Acquired by Bristol-Myers Squibb in 2019; historical DM relevance from investment in RNA-targeting companies.
	Active	Active	Investigational in vivo gene-editing therapy for DM1 in collaboration with Vertex.
Cydan Development Inc.	Closed	Unknown	Company closed in 2021; rare disease accelerator; incubated companies with neuromuscular programs.
	Active	Unknown	Ended DM program; initially pursued neuromuscular CNS-targeting therapeutics.
	Active	Unknown	Ended Pfizer-collaboration on DM program targeting toxic RNA condensates.
	Acquired	Active	Acquired by Novartis in 2023; DM program ongoing within Novartis Gene Therapy.

(PRE) Pre-clinical

Pre-clinical research is the stage before a treatment is tested in humans, where scientists study its basic safety and potential effectiveness.

(P1) Phase 1 — Initial Safety & Dosage

First studies in humans.
Focus: safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD).
Helps determine safe dose range and side effects.

(P2) Phase 2 — Preliminary Effectiveness

Conducted in people with the disease.
Focus: preliminary evidence of effectiveness + continued safety evaluation.
Refines dose and regimen.

(P3) Phase 3 — Confirmatory Evidence

Large, well-controlled studies.
Focus: confirm effectiveness, monitor adverse reactions, compare to standard care.
Provides the primary evidence for FDA approval.

(P4) Phase 4 — Postmarketing Studies

Post-approval.
Focus: additional information on risks, benefits, and optimal use.
Includes real-world safety (pharmacovigilance) and effectiveness.

Key FDA nuances

Phases are not strictly linear (can overlap or combine, e.g., Phase 1/2).
FDA focuses on evidence standards, not just phase labels.
For serious/rare diseases, flexibility is common (smaller trials, adaptive designs).

About the myotonic dystrophy drug development pipeline

This tool helps you explore therapies being developed for myotonic dystrophy (DM), from early research through approved treatments.

The main pipeline shows current and ongoing programs, including:

Company developing the therapy
Drug name and approach (e.g., gene therapy, small molecule)
Development phase
Target disease subtype

How to read the pipeline

Think of the pipeline as a journey:

Discovery → Pre-clinical → Phase 1 → Phase 2 → Phase 3 → Approval → Phase 4

Not all therapies make it through every stage
Many programs are being explored at once across different approaches (e.g., gene therapy, repurposed drugs)

1. Start with a search (optional)

Use “Search by company name” if you’re looking for a specific organization or therapy.

Type part or all of a company name
Results will update automatically

2. Filter by development phase

Use this filter to understand how far along a therapy is in development.

Pre-clinical – Early research in labs or animals (not yet tested in humans)
Phase 1 – First human trials focused on safety and dosage
Phase 2 – Tests effectiveness and continues safety evaluation
Phase 3 – Large trials to confirm effectiveness and compare to existing treatments
Phase 4 – Approved treatments monitored for long-term safety

3. Filter by DM Subtype

Narrow results based on the type of myotonic dystrophy.

Review Past Efforts

Scroll to the table below to see previous or inactive programs.

Use filters to sort by:

Company name
Status:
- Active – Still progressing
- Acquired – Taken over by another company
- Closed – No longer in development

Myotonic dystrophy drug development pipeline

Active pipeline

Phase 2

Phase 2

Phase 2

Phase 2

Phase 2

Phase 1

Phase 1

Pre-clinical

Past efforts

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