AM Pharma competitors & alternatives

UltragenyxBiotechJapan

69% matchAI 8/100 ▲Green 5/100 ▲

Biotech

Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.

Similar biotechnology company

Amolyt Pharma is a biotechnology company focused on developing peptide therapeutics for rare endocrine and metabolic diseases. Its lead program, eneboparatide, is being developed for chronic hypoparathyroidism, serving patients with serious hormone-related disorders and the clinicians who treat them.

AMICUS THERAPEUTICS INCBiotechUnited States

67% matchPrivate

Biotech

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.

Alesta TherapeuticsBiotechnologyNetherlands

66% match

Similar biotechnology company

Alesta Therapeutics is a clinical-stage biotechnology company based in Leiden, Netherlands, dedicated to developing novel oral small-molecule therapies targeting GCN2 for underserved rare diseases.

Applied Therapeutics Inc.BiotechnologyUnited States

66% matchPrivate

Similar biotechnology company

Applied Therapeutics, Inc. is a biopharmaceutical company headquartered in New York City, focused on developing novel product candidates targeted at disease targets involving enzyme and metabolic pathways.

Amplio PharmaBiotechnologyNetherlands

65% match

Similar biotechnology company

Amplio Pharma is a pharmaceutical company founded in 2020 that develops proprietary drug products to significantly amplify the efficacy of existing pharmaceutical treatments. The company is headquartered in Leiden, Netherlands.

uniQure N.V.QURE · BiotechAmsterdam, Netherlands

65% matchPublic

Biotech

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.

Amicus Inc. / DEBiotechnologyUnited States

65% match

Similar biotechnology company

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.

Orchard Therapeutics plcBiotechUnited Kingdom

64% matchPrivate

Biotech

Orchard Therapeutics plc is a biopharmaceutical company with operations including a U.S. headquarters located in Boston, Massachusetts.

ProQR Therapeutics N.V.PRQR · BiotechLeiden, Netherlands

64% matchPublic

Biotech

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.

Biotech

Ascendis Pharma A/S is a biopharmaceutical enterprise dedicated to advancing therapeutic solutions for critical medical conditions that currently lack effective treatments. Its commercialized offering is SKYTROFA, a therapy prescribed for individuals suffering from growth hormone deficiency (GHD). The company boasts a robust development pipeline, featuring several TransCon-branded candidates. These include TransCon Growth Hormone (hGH), which is being progressed for pediatric GHD patients in Japan and for adults with GHD. Also under development are TransCon parathyroid hormone for adults with hypoparathyroidism, and TransCon CNP, aimed at treating pediatric achondroplasia. Further expanding its innovative portfolio, Ascendis is also developing a TransCon toll-like receptor (TLR) 7/8 agonist for intratumoral administration, alongside TransCon IL-2 ß/g, designed for systemic delivery. Founded in 2006, this enterprise maintains its corporate headquarters in Hellerup, Denmark.

Biotech

Lixte Biotechnology Holdings, Inc. is a drug discovery company focused on developing new treatments. It employs biomarker technology to identify crucial enzyme targets associated with serious, widespread diseases, then designs innovative compounds to act upon these targets. The company's primary focus lies in protein phosphatase inhibitors, which are being investigated for use both independently and in combination with cytotoxic agents, radiation therapy, and immune checkpoint blockers. Its pipeline includes two main categories of compounds, currently in various stages of pre-clinical and clinical development. The LB-100 series consists of novel chemical entities aimed at treating various cancers, vascular diseases, and metabolic disorders. Meanwhile, the LB-200 series is being developed to address chronic hereditary conditions, such as Gaucher's disease, as well as specific cancers and neurodegenerative ailments. Lixte Biotechnology Holdings, Inc. has forged significant alliances, including a clinical trial research agreement with the Moffitt Cancer Center and Research Institute Hospital Inc. Additionally, it has collaboration agreements for investigator-initiated clinical trials with the Spanish Sarcoma Group, the Netherlands Cancer Institute, and the Oncode Institute. Founded in 2005, the company is headquartered in Pasadena, California.