PepGen Inc.

Dyne Therapeutics, Inc. is a U.S.-based biotechnology firm focused on developing advanced treatments for genetically driven muscle disorders. Utilizing its proprietary FORCE platform, which is engineered to deliver disease-modifying therapeutics, the company is actively engaged in various programs. These initiatives target conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, in addition to other rare skeletal, cardiac, and metabolic muscle diseases. The company was founded in 2017 and maintains its corporate headquarters in Waltham, Massachusetts.

Entrada Therapeutics, Inc.TRDA · BiotechnologyBoston, United States

71% matchPublic

Entrada Therapeutics, Inc. operates as a biotechnology enterprise dedicated to pioneering endosomal escape vehicle (EEV) therapeutics, specifically engineered to tackle a range of neuromuscular ailments. The company leverages its proprietary EEV platform to cultivate a rich pipeline of therapeutic programs, integrating oligonucleotide, antibody, and enzyme-based approaches. Its foremost product candidate, ENTR-601-44, is currently undergoing preclinical assessment for its potential in addressing both Duchenne muscular dystrophy and myotonic dystrophy type 1. Furthermore, Entrada is actively developing EEV-PMO-CAG, also targeting myotonic dystrophy type 1. Incorporated in 2016, the firm was formerly known as CycloPorters, Inc., before officially changing its name to Entrada Therapeutics, Inc. in October 2017. The company maintains its corporate headquarters in Boston, Massachusetts.

Design Therapeutics, Inc. is a biopharmaceutical company in the preclinical phase that focuses on creating treatments for genetic conditions stemming from nucleotide repeat expansions. Its current pipeline targets Friedreich's Ataxia (FA), a severe, progressive, monogenic, and autosomal recessive disease. FA primarily impairs mitochondrial function across various organ systems, resulting in neurological, cardiac, and metabolic complications. Another significant area of development is Myotonic Dystrophy Type-1 (DM1). This is a progressive, dominantly inherited monogenic neuromuscular illness that impacts skeletal muscle, the heart, brain, and other bodily systems. Beyond these, the company is advancing its GeneTAC product candidate platform to address an array of other monogenic conditions caused by nucleotide repeat expansions. These include Fragile X syndrome, various spinocerebellar ataxias, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington's disease, and spinobulbar muscular atrophy. Established in 2017, Design Therapeutics, Inc. maintains its headquarters in Carlsbad, California.

Dynacure is a biotechnology company developing antisense oligonucleotide therapies for rare neuromuscular diseases. Its lead program, DYN101, targets DNM2-associated centronuclear myopathy for patients with this serious inherited muscle disorder.

Kelonia Therapeutics, Inc.BiotechnologyUnited States

70% matchPrivate

Kelonia Therapeutics, Inc. is a clinical-stage biotech company headquartered in Boston, Massachusetts, focused on delivering genetic medicine for patients.

ProJenX, Inc.BiotechnologyUnited States

69% matchPrivate

ProJenX, Inc. is a clinical-stage biotechnology company founded in 2021 and headquartered in New York City. The firm focuses on neuroscience, developing novel, brain-penetrant therapies targeting biologically defined motor neuron diseases such as ALS. It advances research programs aimed at addressing severe neurodegenerative conditions.