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PTC Therapeutics, Inc. competitors & alternatives
The 12 closest competitors and alternatives to PTC Therapeutics, Inc. among biotech companies — ranked by similarity to what PTC Therapeutics, Inc. actually does, not by market-cap band.
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About PTC Therapeutics, Inc.
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.
PTC Therapeutics, Inc. at a glance
PTC Therapeutics, Inc. is a biotech company headquartered in Warren, United States, founded in 1998. It is publicly listed (PTCT) with a market capitalisation of $5.4B. Xcout tracks 32 named people at PTC Therapeutics, Inc., including Matthew Klein (Chief Executive Officer & Director). Xcout recorded 64 job postings from PTC Therapeutics, Inc. in the last 90 days.
PTC Therapeutics, Inc. leadership
- Matthew KleinChief Executive Officer & Director
- John BairdChief of Staff to the CEO
- Matthew B. KleinChief Executive Officer
- Pierre GravierChief Financial Officer
- Eric PauwelsChief Business Officer
- Lee GoldenExecutive Vice President & Chief Medical Officer
26 more named people on record for PTC Therapeutics, Inc., with roles, board committees and tenure — see the full roster →
PTC Therapeutics, Inc. funding rounds on record
Every round on record:
| Date | Round | Amount | Investors |
|---|---|---|---|
| — | $26.6M | — |
Some filings are not shown as funding rounds.
Who invested in each round, lead investors, valuations and the co-investor graph — open the full funding history →
Hiring at PTC Therapeutics, Inc.
Xcout recorded 64 job postings from PTC Therapeutics, Inc. in the last 90 days, from a hiring record Xcout has kept since June 2026.
The weekly hiring trend, the roles and locations behind it — see PTC Therapeutics, Inc.'s hiring signals →
Top 12 PTC Therapeutics, Inc. competitors
Ranked by semantic similarity — how close each company is to PTC Therapeutics, Inc. by what it does, using Xcout's live company graph. Click any company for its full profile, or its competitor set.
Similar biotech company
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.
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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.
Biotechnology
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.
Similar biotech company
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.
Similar biotech company
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.
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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.
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.
Similar biotech company
Wave Life Sciences Ltd., a clinical-stage biotechnology company, designs, develops, and commercializes ribonucleic acid (RNA) medicines through PRISM, a discovery and drug development platform. The company’s medicines platform, PRISM combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. It is developing WVE-006, a GalNAc-conjugated RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; WVE-007, a GalNAc-conjugated small interfering RNA designed to silence INHBE mRNA targeting obesity; WVE-008, a GalNAc-conjugated RNA editing oligonucleotide for the treatment of liver disease; WVE-N531, an exon splicing oligonucleotide for the treatment of Duchenne muscular dystrophy; and WVE-003, an allele-selective oligonucleotide for the treatment of Huntington’s disease (HD). The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was founded in 2012 and is based in Singapore.
Biotechnology
Amicus Therapeutics, a subsidiary of BioMarin Pharmaceutical Inc., is a biotechnology company that researches and delivers medicines for individuals living with genetically defined rare diseases. The company provides patient advocacy and support services alongside its clinical development pipeline.
Biotechnology
United Therapeutics Corporation, a biotechnology firm, is dedicated to discovering, developing, and bringing to market medical solutions for individuals suffering from chronic and severe, often life-threatening, illnesses. Its operations span both the United States and international markets. The company's current commercial portfolio features several key therapeutics: Remodulin, prescribed for pulmonary arterial hypertension (PAH) to lessen symptoms experienced during physical activity. Tyvaso, an inhaled form of the prostacyclin analogue treprostinil, designed to enhance exercise capacity in patients with PAH and pulmonary hypertension linked to interstitial lung disease (PH-ILD). Orenitram, a treprostinil tablet formulation, also aims to boost the exercise capabilities of PAH patients. Unituxin, a monoclonal antibody utilized in the treatment of high-risk neuroblastoma. Adcirca, an oral PDE-5 inhibitor, which helps improve exercise performance in those with PAH. United Therapeutics also maintains a robust development pipeline, including: Tyvaso DPI, a dry powder inhaler version of Tyvaso. The Remunity Pump, a compact, lightweight, and durable infusion system for treprostinil, accompanied by a separate controller. RemoPro and Ralinepag, both investigational treatments for PAH. Aurora-GT, a gene therapy product focused on regenerating blood vessels within the lungs. Clinical trials such as Tyvaso PERFECT and TETON, which are evaluating Tyvaso's efficacy in patients with World Health Organization (WHO) Group 3 pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD). The company has forged strategic alliances and licensing agreements with various entities, such as DEKA Research & Development Corp. for a semi-disposable subcutaneous treprostinil delivery system; MannKind Corporation for the development and licensing of treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. for the development of Ralinepag. Established in 1996, United Therapeutics Corporation is headquartered in Silver Spring, Maryland.
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uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in phase I/II clinical study for the treatment of Huntington’s disease. The company also develops AMT-260, which is in phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Biotechnology
Falcon Therapeutics, Inc., in association with Skyhawk Therapeutics, conducts global clinical trials for Huntington's disease. The company serves patients with Huntington's disease by evaluating the small molecule splicing modifier SKY-0515 to lower mutant huntingtin protein levels and slow disease progression.
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