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DYNE-401
- Development stage
- Preclinical
- Lead developer
- Dyne Therapeutics
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Gene Therapies, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
DYNE-401 is an investigational drug being developed by Dyne Therapeutics for the treatment of Pompe disease (Glycogen Storage Disease Type II). It is a next-generation enzyme replacement therapy that utilizes Dyne’s proprietary FORCE platform to deliver acid alpha-glucosidase (GAA) specifically to muscle tissue. The drug is a conjugate targeting both transferrin receptor protein 1 (TfR1) and GAA, aiming to enhance delivery and uptake into muscle cells. Mechanistically, DYNE-401 acts as a transferrin receptor protein 1 (TfR1) antagonist and an acidic alpha-glucosidase inhibitor, designed to address the underlying enzyme deficiency in Pompe disease by improving cellular uptake of GAA. As of March 2025, DYNE-401 remains in preclinical development[1][3].
- Brand names
- FORCE-GAA
- Other names
- FORCE-GAA
02
Targets
GAA (Lysosomal acid alpha-glucosidase)TFRC (Transferrin Receptor)
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