PepGen Inc. competitors & alternatives

Similar biotech company

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

Biotechnology

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.

Similar biotech company

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.

Biotechnology

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

Biotechnology

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

Biotechnology

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.

Biotechnology

Benitec Biopharma Inc., a clinical-stage biotechnology company, focuses on the development of novel genetic medicines. Its Silence and Replace, a DNA-directedRNA interference platform combines RNA interference with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The company is developing BB-301, an AAV-based gene therapy designed to silence the expression of the disease-causing gene for the treatment of oculopharyngeal muscular dystrophy related dysphagia. Benitec Biopharma Inc. is headquartered in Hayward, California.

Similar biotech company

Passage Bio, Inc. is a biopharmaceutical company dedicated to pioneering genetic therapies for conditions affecting the central nervous system. Their advanced pipeline includes PBGM01, designed to treat infantile GM1 gangliosidosis by utilizing a proprietary AAVhu68 capsid to deliver a functional GLB1 gene, which codes for the lysosomal acid beta-galactosidase enzyme, to both the brain and peripheral tissues. Another key program, PBFT02, employs an AAV1 capsid to introduce a functional granulin (GRN) gene, encoding progranulin (PGRN), to the brain as a treatment for FTD-GRN. Additionally, PBKR03 is under development for infantile Krabbe disease, leveraging a proprietary AAVhu68 capsid to deliver a functional GALC gene, responsible for producing the hydrolytic enzyme galactosylceramidase, to the brain and surrounding tissues. The company's portfolio also features PBML04 for metachromatic leukodystrophy, PBAL05 for amyotrophic lateral sclerosis, and PBCM06 for Charcot-Marie-Tooth Type 2A. Passage Bio maintains a significant research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program, alongside a development services and clinical supply agreement with Catalent Maryland, Inc. Established in 2017, the company's headquarters are located in Philadelphia, Pennsylvania.

Neuron23BiotechnologyUnited States

68% match

Biotechnology

Neuron23 is a biotechnology company founded in 2018 that focuses on developing precision therapeutics for genetic diseases. Headquartered in South San Francisco, California, the company relies on a team of scientific experts and entrepreneurs.

Solid Biosciences Inc.SLDB · BiotechCharlestown, United States

68% matchPublic

Similar biotech company

Solid Biosciences Inc. is an American biotechnology firm dedicated to creating therapeutic solutions for Duchenne Muscular Dystrophy (DMD). Its most advanced experimental drug, SGT-001, is a gene transfer treatment currently in Phase I/II clinical trials, designed to encourage the production of functional dystrophin protein in patients' muscles. The company is also progressing with SGT-003, which represents a next-generation gene transfer therapy intended for DMD. Beyond specific drug candidates, Solid Biosciences is actively developing innovative platform technologies, such as "dual gene expression," a technique that allows for multiple therapeutic genes to be bundled into a single viral vector, and researching new capsid designs. The company maintains a strategic alliance through a collaboration and license agreement with Ultragenyx Pharmaceutical Inc., focusing on the joint development and market introduction of novel gene therapies for Duchenne Muscular Dystrophy. Founded in 2013, Solid Biosciences Inc. conducts its operations from its headquarters in Cambridge, Massachusetts.

PrileniaBiotechUnited States

68% match

Similar biotech company

Prilenia Therapeutics is a clinical-stage biotechnology company developing treatments for neurodegenerative and neurodevelopmental disorders. Its lead drug candidate, pridopidine, has been studied in Huntington’s disease and amyotrophic lateral sclerosis (ALS), serving patients with serious neurological conditions.

PTC Therapeutics, Inc.PTCT · BiotechnologyWarren, United States

67% matchPublicAI 0/100Green 0/100

Biotechnology

PTC Therapeutics, Inc. (PTCT) is a biopharmaceutical company committed to the research, development, and commercialization of innovative therapies for patients afflicted with rare genetic disorders. Its robust pipeline encompasses both commercialized therapies and a variety of experimental drug candidates, spanning all stages of development—from early research and preclinical studies to clinical trials—with a primary focus on addressing various rare disease indications. Among its commercialized products, PTC Therapeutics provides Translarna and Emflaza, offering therapeutic options for Duchenne muscular dystrophy patients in the European Economic Area and the United States. Translarna also addresses nonsense mutation Duchenne muscular dystrophy in Brazil and Russia. The company additionally commercializes Tegsedi and Waylivra for various rare conditions throughout Latin America and the Caribbean. Furthermore, in Brazil, PTC Therapeutics distributes Evrysdi as a treatment for spinal muscular atrophy (SMA) in patients aged two months and older. Leveraging its proprietary splicing platform, the company is also actively developing PTC518, a promising therapeutic candidate aimed at treating Huntington's disease. To bolster its research and development capabilities, PTC Therapeutics has established strategic collaborations, including partnerships with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation, focusing on advancing drug discovery and development research, particularly in regenerative medicine. Additionally, it collaborates with Akcea Therapeutics, Inc. for the commercialization of Tegsedi and Waylivra in the Latin America and Caribbean region. Established in 1998, PTC Therapeutics, Inc. maintains its corporate headquarters in South Plainfield, New Jersey.