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ProQR Therapeutics N.V. competitors & alternatives
The 12 closest competitors and alternatives to ProQR Therapeutics N.V. among biotech companies — ranked by similarity to what ProQR Therapeutics N.V. actually does, not by market-cap band.
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About ProQR Therapeutics N.V.
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.
ProQR Therapeutics N.V. at a glance
ProQR Therapeutics N.V. is a biotech company headquartered in Leiden, Netherlands. It is publicly listed (PRQR) with a market capitalisation of $223.7M. Xcout tracks 26 named people at ProQR Therapeutics N.V., including Dennis Hom (Chief Financial Officer).
ProQR Therapeutics N.V. leadership
- Dennis HomChief Financial Officer
- Christopher HartChief Data & AI Officer
- Cristina Lopez LopezChief Medical Officer
- Dr. Cristina Lopez Lopez, MD, PhDChief Medical Officer
- Sheila SponseleeChief People & Operations Officer
- Daniel Anton de BoerFounder, CEO & Executive Director
20 more named people on record for ProQR Therapeutics N.V., with roles, board committees and tenure — see the full roster →
Top 12 ProQR Therapeutics N.V. competitors
Ranked by semantic similarity — how close each company is to ProQR Therapeutics N.V. by what it does, using Xcout's live company graph. Click any company for its full profile, or its competitor set.
Similar biotech company
Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.
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.
Biotechnology
InteRNA Technologies is a venture-backed biotech and life sciences company founded in 2008 and headquartered in Utrecht, Netherlands. The company specializes in the development of microRNA (miRNA)-focused drug pipelines and therapeutics. It operates within the biopharmaceutical sector, supported by various institutional backers and investors.
Similar biotech company
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
Amylyx Pharmaceuticals, Inc. operates as a clinical-stage biopharmaceutical firm, primarily focused on creating therapies for amyotrophic lateral sclerosis (ALS) and various other neurodegenerative conditions. A key asset in its developmental pipeline is AMX0035, a proprietary dual UPR-Bax apoptosis inhibitor. This compound, which integrates sodium phenylbutyrate and taurursodiol, is under investigation for treating ALS, and its application is also being explored for other neurodegenerative diseases. The company was established in 2013 and is based in Cambridge, Massachusetts.
Biotechnology
4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company that engineers therapeutic agents by leveraging its proprietary adeno-associated virus (AAV) vector technology. The company's developmental efforts are concentrated across a trio of therapeutic fields: ophthalmology (eye conditions), cardiology (heart ailments), and pulmonology (respiratory disorders). Within its active pipeline, three specific product candidates have advanced into clinical trial phases, all currently undergoing Phase 1/2 clinical assessment: 4D-125 is being evaluated for the treatment of X-linked retinitis pigmentosa. 4D-110 targets choroideremia. 4D-310 is focused on addressing Fabry disease. Beyond these, two Investigational New Drug (IND) candidates are in development: 4D-150, aimed at combating wet age-related macular degeneration, and 4D-710, designed to tackle cystic fibrosis lung disease. To advance its mission, 4D Molecular Therapeutics, Inc. actively engages in research and development partnerships with entities such as uniQure, CRF, Roche, and CFF. The firm commenced operations in 2013 and maintains its headquarters in Emeryville, California.
Similar biotech company
LianBio is a biopharmaceutical firm dedicated to advancing and marketing therapeutic agents across various critical health sectors, including cardiovascular disorders, oncology, ophthalmological conditions, and inflammatory ailments. Its operations are primarily concentrated in China and the broader Asian region. The company's pipeline features several promising drug candidates targeting a range of severe illnesses. These include mavacamten, aimed at both obstructive and non-obstructive hypertrophic cardiomyopathy, as well as heart failure with preserved ejection fraction. TP-03 is being developed for Demodex blepharitis and meibomian gland dysfunction. For cancer, LianBio is advancing NBTXR3 for head and neck squamous cell carcinoma and other solid tumors, alongside Infigratinib for cholangiocarcinoma (both first-line and second-line settings) and gastric cancers, and BBP-398 also for solid tumors. Furthermore, Omilancor and NX-13 are in development for inflammatory bowel diseases like ulcerative colitis and Crohn's disease. Other programs include LYR-210 for chronic rhinosinusitis and Sisunatovir to combat respiratory syncytial virus. LianBio has established key collaborations, partnering with Tarsus Pharmaceuticals, Inc. on the development and market introduction of TP-03 specifically for Demodex blepharitis. Additionally, it maintains a strategic alliance with Nanobiotix S.A. for NBTXR3, a novel radioenhancer intended for direct intratumoral injection before conventional radiation therapy. Established in 2019, the company's corporate headquarters are located in Princeton, New Jersey.
Similar biotech company
Prothena Corporation plc, a late-stage clinical biotechnology company, focuses on discovery and development of novel therapies to treat diseases caused by protein dysregulation. Its product pipeline includes Prasinezumab, an investigational humanized monoclonal antibody which is in Phase 3 clinical trial for the treatment of parkinson's disease and other synucleinopathies. The company also develops Coramitug, an investigational antibody, which is in Phase 3 clinical trial for the treatment of transthyretin amyloidosis; BMS-986446, an anti-tau antibody which is in Phase 2 clinical trial to treat Alzheimer's disease; PRX019, an investigational antibody which is in Phase 1 clinical trial for the treatment of neurodegenerative diseases; and PRX123, a Dual Aß-Tau Vaccine, which is in preclinical trial for treating Alzheimer's disease. In addition, it develops discovery- and late-preclinical-stage programs include TDP-43 CYTOPE for the treatment of amyotrophic lateral sclerosis; PRX012, a next-generation anti-Aß antibody which is in phase 1 clinical trial to treat Alzheimer's disease; and PRX012-TfR, a preclinical program for the treatment of Alzheimer's disease. It has a license, development, and commercialization agreement with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. to develop and commercialize antibodies that target a-synuclein, including prasinezumab; and master collaboration agreement with Bristol Myers Squibb to develop and commercialize antibodies targeting tau, TDP-43. Prothena Corporation plc was incorporated in 2012 and is based in Dublin, Ireland.
Biotechnology
Phlox Therapeutics is a private biotechnology company pioneering RNA-based gene therapies to alleviate and cure severe genetic heart diseases and cardiomyopathies. Headquartered in Naarden, Netherlands, the company focuses on innovative cardiovascular treatments.
Similar biotech company
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.
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
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.
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