PTC Therapeutics, Inc.

AMICUS THERAPEUTICS INCBiotechUnited States

71% matchPrivate

Amicus Therapeutics, Inc. is an American biopharmaceutical company focused on discovering and delivering advanced therapies for rare diseases. The company is publicly traded on Nasdaq and maintains its global corporate headquarters in Princeton, New Jersey.

Protalix BioTherapeutics, Inc., a biopharmaceutical company, engages in the development, production, and commercialization of recombinant therapeutic proteins based on the ProCellEx plant cell-based protein expression system. The company provides Elelyso for the treatment of Gaucher disease; and Elfabrio for the treatment of adult patients with a confirmed diagnosis of Fabry disease. It is also developing PRX-115, a plant cell expressed recombinant PEGylated Uricase, which is in Phase 2 trial for the treatment of gout; and PRX-119, a plant cell expressed PEGylated recombinant human DNase I product candidate for the treatment of neutrophil extracellular traps diseases. The company has agreements and partnerships with Pfizer; Fundação Oswaldo Cruz; and Chiesi Farmaceutici S.p.A. Protalix BioTherapeutics, Inc. has strategic partnership with Secarna Pharmaceuticals GmbH & Co KG. The company is headquartered in Hackensack. New Jersey.

Pliant Therapeutics, Inc.PLRX · BiotechSouth San Francisco, United States

70% matchPublic

Pliant Therapeutics, Inc. is a biopharmaceutical company in the clinical development stage, dedicated to discovering, advancing, and bringing to market innovative therapies for fibrotic and related diseases across the United States. Its flagship investigational product, PLN-74809, is an orally administered, small-molecule, dual-selective inhibitor targeting both avß6 and avß1 integrins, which is currently progressing through three distinct Phase 2a clinical trials. The company's pipeline also includes PLN-1474, a small-molecule selective inhibitor of avß1, which has successfully completed its Phase 1 clinical assessment for treating liver fibrosis associated with nonalcoholic steatohepatitis (NASH). Furthermore, Pliant is nurturing two additional integrin-based initiatives in preclinical development: one focused on oncology, and another involving an allosteric agonistic monoclonal antibody designed to act upon an undisclosed integrin receptor for the treatment of various muscular dystrophies, including Duchenne muscular dystrophy. Founded in 2015, Pliant Therapeutics, Inc. maintains its corporate headquarters in South San Francisco, California.

Voyager Therapeutics, Inc. operates as a gene therapy company, concentrating its efforts on developing innovative treatments and pioneering advanced platform technologies. Its leading clinical asset, VY-AADC, is presently undergoing an open-label Phase 1 clinical trial for the management of Parkinson's disease. The company's preclinical portfolio is extensive, featuring VY-SOD102 for amyotrophic lateral sclerosis (ALS), VY-HTT01 for Huntington's disease, and VY-FXN01 for Friedreich's ataxia. Additionally, Voyager is pursuing a Tau program aimed at various tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, alongside initiatives for spinal muscular atrophy. To advance its gene therapy product pipeline, the company has forged strategic collaboration and licensing agreements with key industry players such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. These partnerships cover the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and maintains its corporate headquarters in Cambridge, Massachusetts.

ProQR Therapeutics N.V.PRQR · BiotechLeiden, Netherlands

69% matchPublic

ProQR Therapeutics N.V. operates as a biopharmaceutical enterprise dedicated to discovering and developing RNA-based therapies designed to address genetic disorders. The company's pipeline includes two primary drug candidates in advanced clinical stages: Sepofarsen is currently in a Phase II/III clinical trial, known as the ILLUMINATE trial, targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also undergoing Phase II/III evaluation for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome. Beyond its clinical programs, ProQR is actively developing its proprietary Axiomer RNA base-editing platform technology. Strategic collaborations are central to ProQR's strategy, evidenced by its licensing agreements with institutions such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Furthermore, a significant license and research partnership exists with Eli Lilly and Company, focused on the discovery, advancement, and commercialization of novel medicines for genetic conditions impacting the liver and nervous system. Established in 2012, ProQR Therapeutics N.V. maintains its corporate headquarters in Leiden, the Netherlands.

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.