Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy

Next generation exon skipping therapies for Duchenne Muscular Dystrophy (DMD) represent a promising area of research aimed at addressing the underlying genetic mutations that cause the disease.

DMD is caused by mutations in the dystrophin gene, which results in the absence or dysfunction of dystrophin, a protein essential for muscle function. Exon skipping aims to bypass these mutations by promoting the inclusion or exclusion of specific exons in the mRNA, enabling the production of a shorter but functional version of the dystrophin protein. [Learn More: What is exon skipping and how does it work?]

AOC 1044 (Avidity Biosciences)

AOC 1044 is developed to precisely skip exon 44 of the dystrophin gene in order to facilitate dystrophin synthesis in those who are living with DMD44-related conditions.

Web Page: EXPLORE44

Clinic Trial: NCT05670730

BMN 351 (Biomarin)

For patients who are able to undergo exon 51 skipping, BMN 351 is a therapy that involves skipping exons.

Web Page: BioMarine

Clinic Trial: NCT06280209

Dyne-251 (Dyne Therapeutics)

A therapy known as Dyne-251 is an exon skipping therapy that is coupled to an antigen-binding fragment (Fab) in order to facilitate the medication’s delivery to muscle cells that are capable of being skipped by exon 51.

Web Page: Dyne 251

Clinic Trial: NCT05524883

ENTR-601-44 (Entrada Therapeutics)

The potential of ENTR-601-44, an experimental treatment for Duchenne patients who are amenable to exon 44 skipping, to restore the mRNA reading frame and enable the translation of dystrophin protein that is marginally shortened but still functional is being assessed.

Web Page: Entrada

Clinic Trial: Phase 1

NS-050/​NCNP-03 (NS Pharma)

A therapy known as NS-050/NCNP-03 is an exon skipping therapy that is intended for patients who are able to skip exon 50.

Web Page: Nippon Shinyaku

Clinic Trial: NCT06053814

NS-089/NCNP-02 (NS Pharma)

An antisense oligonucleotide (AON) that is shown to be effective in treating Duchenne muscular dystrophy and can skip exon 44. It is now recruiting patients with DMD in a phase 2 trial that is being conducted for the purpose of researching this medication. [Read More: What is Brogidirsen?]

Web Page: Nippon Shinyaku

Clinic Trial: NCT05996003

PGN-EDO51 (PepGen)

An antisense oligonucleotide (AON) that is suggested for the treatment of Duchenne muscular dystrophy that is capable of bypassing exon 51. Currently, patients with DMD are being recruited for participation in a phase 2 clinical research that is being conducted to investigate this medication.

Web Page: PepGen

Clinic Trial: NCT06079736

SQY51 (Sqy Therapeutics)

An antisense oligonucleotide (AON) that is approved for the treatment of Duchenne muscular dystrophy that is bypassing exon 51. In France, patients with Duchenne muscular dystrophy are now being recruited for a phase 1/2a (AVANCE1) trial that is being conducted to investigate this therapy.

Web Page: Sqy

Clinic Trial: NCT05753462

WVE-N531 (Wave Life Sciences)

An antisense oligonucleotide (AON) that is shown to be effective in treating Duchenne muscular dystrophy that is capable of bypassing exon 53. This treatment is now being investigated in a clinical trial that is in the phase 1b/2a stage and is currently active, but it is not recruiting patients.

Web Page: Wave Life Sciences

Clinic Trial: NCT04906460

In conclusion, next generation exon skipping therapies represent a breakthrough in the treatment of Duchenne muscular dystrophy, offering the potential to slow disease progression, improve muscle function, and enhance the quality of life for affected individuals. With continued advancements and clinical validation, this approach holds great promise for managing DMD more effectively in the future.

Learn More: Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy