Dyne Therapeutics Gains FDA Fast Track for DYNE-101 in Myotonic Dystrophy

Dyne Therapeutics has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its investigational therapy, DYNE-101.

DYNE-101 is an investigational therapy comprising an antisense oligonucleotide (ASO) conjugated to a fragment antibody (Fab). 

This design targets transferrin receptor 1 (TfR1), a protein highly expressed in muscle tissue, to enhance delivery to affected areas. The therapy aims to reduce toxic RNA associated with the DMPK gene mutation, restore normal protein processing, and potentially halt or reverse disease progression. 

DYNE-101 has also been granted orphan drug designation by the FDA and the European Medicines Agency (EMA) for DM1 treatment.

DM1 is a rare, genetic condition characterised by progressive muscle weakness, myotonia, and a range of systemic complications affecting mobility, heart function, breathing, and vision, among others. 

It is caused by an abnormal expansion of CTG repeats in the DMPK gene, leading to toxic RNA accumulation in the nucleus and disrupted protein splicing. 

This condition impacts over 40,000 individuals in the United States and approximately 74,000 in Europe. Currently, no disease-modifying treatments are available for DM1.

The therapy is currently being assessed in the ongoing Phase 1/2 ACHIEVE global clinical trial for the treatment of myotonic dystrophy type 1 (DM1).

The FDA’s Fast Track designation aims to facilitate the development and accelerate the review process for drugs addressing serious conditions with unmet medical needs. 

Benefits include more frequent communications with the FDA, rolling review of regulatory applications, and the potential for earlier patient access through Accelerated Approval or Priority Review pathways, provided specific criteria are met.

Dyne Therapeutics’ ongoing efforts aim to address this significant unmet medical need with innovative therapies such as DYNE-101.

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