uniQure N.V. competitors & alternatives

Similar biotech company

Neurogene Inc. is dedicated to engineering transformative genetic treatments for individuals and their families grappling with debilitating neurological conditions. Its current pipeline prominently features two key investigational therapies: NGN-401, an AAV9 gene therapy specifically targeting Rett syndrome, and NGN-101, which is being developed to address neuronal ceroid lipofuscinosis subtype 5 (Batten disease). The company's operations are based in New York, New York.

Amicus Inc. / DEBiotechnologyUnited States

67% match

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.

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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.

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.

Homology Medicines, Inc.BiotechnologyUnited States

65% matchPrivate

Biotechnology

Homology Medicines, Inc. is a clinical-stage genetic medicines company historically focused on transforming the lives of patients with rare diseases through genetic treatments.

Biotechnology

REGENXBIO Inc. is a biotechnology company operating in the clinical stage, dedicated to developing gene therapy candidates. These therapies are designed to introduce genes into cells, aiming either to rectify genetic deficiencies or to stimulate the body's own cells to produce therapeutic proteins or antibodies to combat diseases. Central to its therapeutic development is the proprietary NAV Technology Platform, an adeno-associated virus-based gene delivery system. The company's primary investigational asset, RGX-314, is currently in Phase III clinical trials for treating wet age-related macular degeneration. Its pipeline also includes RGX-121 and RGX-111, both in Phase I/II trials for mucopolysaccharidosis type II and type I, respectively; RGX-181, which is in preclinical development for late-infantile neuronal ceroid lipofuscinosis type II disease; RGX-202, undergoing Phase I/II evaluation for Duchenne muscular dystrophy; and RGX-381, at the preclinical stage for addressing the ocular manifestations of CLN2 disease. In addition to its internal programs, REGENXBIO Inc. licenses its NAV Technology Platform to other biotech and pharmaceutical enterprises. It also maintains a collaborative and licensing agreement with Neurimmune AG for the advancement of innovative gene therapies. Founded in 2008, REGENXBIO Inc. maintains its headquarters in Rockville, Maryland.

CIRCIO HOLDINGCRNA · BiotechNorway

65% matchPublic

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Circio Holding ASA is a biotechnology company that develops novel circular RNA and immunotherapy medicines, utilizing its proprietary circVec platform to enhance gene therapy for genetic diseases. It serves the biopharmaceutical sector and patients with genetic disorders by advancing clinical programs and engaging in research collaborations.

Solid Biosciences Inc.SLDB · BiotechCharlestown, United States

65% matchPublic

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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.

AskBioBiotechnologyUnited States

64% match

Biotechnology

AskBio is a clinical-stage gene therapy company that develops adeno-associated virus (AAV) gene therapies to treat genetic and complex disorders. The company focuses on advancing genetic medicine through its proprietary technology platforms, manufacturing processes, and clinical trials.

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AnGes, Inc., a biopharmaceutical firm headquartered in Ibaraki, Japan, and established in 1999, focuses on pioneering the research and development of gene-based medical solutions. Its primary therapeutic asset is Hepatocyte Growth Factor (HGF) Plasmid, an innovative gene therapy designed to address critical limb ischemia, a severe circulatory condition impacting the legs. The company's diverse pipeline also includes NF-kB Decoy Oligonucleotide, a nucleic acid medicine formulated to suppress inflammation for various ailments, including low back pain. Furthermore, AnGes is actively developing treatments for atopic dermatitis and disc degeneration, as well as specialized medical devices aimed at preventing vascular restenosis. Their development endeavors also extend to therapeutic vaccines for cervical intraepithelial neoplasia, a precursor to cervical cancer, and, through a research partnership with Osaka University, DNA vaccines targeting hypertension. AnGes fosters strategic alliances to expand its global reach and product commercialization. These collaborations include BioMarin Pharmaceutical Inc. for the Japanese market distribution of Naglazyme, a drug for mucopolysaccharidosis VI; and Mitsubishi Tanabe Pharma Corporation, which handles the marketing of HGF Plasmid for peripheral arterial disease in both the United States and Japan. Other key partners are SHIONOGI & CO., LTD. for the commercialization of topical NF-kB decoy oligonucleotide drugs for dermatological conditions, and Vical, Inc. for the development and marketing of Allovectin, a cancer treatment. Additionally, AnGes has joined forces with Osaka University to develop a coronavirus vaccine, and with Brickell Biotech, Inc. for an investigational plasmid DNA vaccine intended to prevent coronavirus across the U.S., South America, and other territories. The company, previously known as AnGes MG, Inc., officially rebranded to AnGes, Inc. in July 2017.

Hunterian Medicine LLCBiotechnologyUnited States

64% matchPrivate

Biotechnology

Hunterian Medicine LLC is a gene-editing company that develops CRISPR-based therapeutics to cure genetic diseases. The company utilizes a patented platform technology with a bidirectional promoter to enable efficient, on-target delivery of CRISPR via a single adeno-associated virus (AAV).

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Stoke Therapeutics, Inc. is an emerging biopharmaceutical company dedicated to developing innovative antisense oligonucleotide (ASO) therapies. Its primary focus is to target and address the fundamental causes of severe genetic disorders, particularly within the United States. The company employs its exclusive Targeted Augmentation of Nuclear Gene Output (TANGO) platform to engineer ASOs that precisely enhance protein expression. Stoke's most advanced candidate, STK-001, is currently progressing through Phase I/IIa clinical trials for Dravet syndrome, a severe and debilitating genetic epilepsy. Additionally, STK-002 is in the preclinical development stage for treating autosomal dominant optic atrophy. The company has also established a licensing and collaborative agreement with Acadia Pharmaceuticals Inc. to identify, develop, and market new RNA-based medicines for critical and rare genetic neurodevelopmental conditions impacting the central nervous system. Formerly known as ASOthera Pharmaceuticals, Inc., the company rebranded to Stoke Therapeutics, Inc. in May 2016. Founded in 2014, Stoke Therapeutics, Inc. maintains its headquarters in Bedford, Massachusetts.