Soufflé Therapeutics

Dyne Therapeutics

The company develops oligonucleotide therapeutics to treat patients with genetically driven neuromuscular diseases, including Duchenne muscular dystrophy and myotonic dystrophy type 1.

Stoke Therapeutics

This biotechnology company develops antisense oligonucleotide medicines to treat severe genetic diseases by restoring protein expression for patients with conditions such as Dravet syndrome.

Capricor

This biotechnology company develops cell and exosome-based therapies to treat Duchenne muscular dystrophy and other serious diseases for patients.

Serif Biomedicines

This biotechnology firm develops modified DNA medicines for patients with genetically defined diseases and immune system conditions.

Edgewise Therapeutics

This biopharmaceutical company develops small molecule therapies for patients with serious cardiovascular diseases and musculoskeletal conditions like Duchenne and Becker muscular dystrophy.

MyoPax

This company develops muscle regeneration cell and gene therapies for patients with congenital malformations, trauma, hereditary dystrophies and muscle wasting disorders.

Cellergy Therapeutics

This biotechnology company develops purified mitochondrial transplantation therapies to treat patients with neurodegenerative diseases, muscle degeneration and age-related conditions.

Mahzi Therapeutics

This biotechnology company develops gene and antisense oligonucleotide therapies for patients with rare genetic neurodevelopmental disorders.