Protalix BioTherapeutics, Inc. (DE) competitors & alternatives

Similar biotech company

Polaryx Therapeutics, Inc. functions as a biotechnology firm dedicated to advancing small-molecule therapies for lysosomal storage and neurodegenerative conditions. Its developmental pipeline includes a trio of promising candidates: PLX-100, which combines PLX-200 with an ancillary supplement, targeting late infantile neuronal ceroid lipofuscinosis (LINCL) and related forms of NCL. PLX-200, a repurposed compound already utilized to address various diseases in both adult and pediatric populations. And PLX-300, derived from an edible plant, which works by activating PPARa to enhance the production of transcription factor EB. The company, established in 2014, maintains its base of operations in Paramus, New Jersey.

AMICUS THERAPEUTICS INCBiotechUnited States

71% matchPrivate

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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.

PTC Therapeutics, Inc.PTCT · BiotechnologyWarren, United States

70% matchPublicAI 0/100Green 0/100

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.

TheranexusBiotechnologyFrance

69% match

Biotechnology

Theranexus (THX Pharma) is a clinical-stage biopharmaceutical company that develops innovative therapeutic solutions for patients suffering from rare neurological diseases such as Batten disease, Niemann-Pick type C, and Gaucher disease. The company utilizes scientific research and antisense oligonucleotides to create targeted drug candidates addressing significant unmet medical needs.

Biotechnology

Rocket Pharmaceuticals, Inc., operating alongside its affiliates, is an innovative biotechnology enterprise dedicated to pioneering gene therapies for rare and severely debilitating diseases. The company's active clinical pipeline features multiple promising programs. It is currently progressing three ex vivo lentiviral vector therapies aimed at specific genetic conditions: Fanconi anemia, characterized by a bone marrow defect that hinders blood cell production; leukocyte adhesion deficiency-I, an inherited disorder resulting in a compromised immune system; and pyruvate kinase deficiency, a rare autosomal recessive red blood cell ailment leading to chronic non-spherocytic hemolytic anemia. Complementing these, Rocket Pharmaceuticals also has an in vivo adeno-associated virus program underway for Danon disease, a serious multi-organ lysosomal storage disorder that tragically often results in early mortality due to heart failure. To facilitate its extensive research and development efforts, the company has established key licensing partnerships with esteemed institutions such as the Fred Hutchinson Cancer Research Center, European research bodies including CIEMAT and its collaborators (Centro de Investigacion Biomedica En Red and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz), UCL Business PLC (in conjunction with CIEMAT), The Regents of the University of California, and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. maintains its primary corporate location in Cranbury, New Jersey.

ProQR Therapeutics N.V.PRQR · BiotechLeiden, Netherlands

67% matchPublic

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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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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.

Protalys TherapeuticsBiotechnologyUnited States

66% match

Biotechnology

Protalys Therapeutics, Inc. is a private drug discovery and development company headquartered in San Diego, California, operating within the therapeutics and drug pipeline sector.

Biotechnology

Can-Fite BioPharma Ltd., an Israeli clinical-stage biopharmaceutical company, specializes in the creation of small molecule therapeutic products. These treatments target a range of conditions including various cancers, inflammatory liver disorders, and erectile dysfunction. Its primary drug candidate, Piclidenoson, has successfully advanced through Phase III clinical trials for psoriasis and completed Phase II trials for COVID-19. Another promising asset, Namodenoson, is currently undergoing Phase III clinical evaluation for hepatocellular carcinoma (a form of liver cancer) and is in a Phase IIb study for non-alcoholic steatohepatitis (NASH). Furthermore, CF602 is in preclinical development, aimed at addressing erectile dysfunction. Beyond its drug pipeline, Can-Fite also commercializes a predictive biomarker blood test kit for A3AR. Strategic partnerships are also key to its operations; a licensing and collaboration agreement with CMS Medical covers the development, manufacturing, and commercialization of both Piclidenoson and Namodenoson. Additionally, the company collaborates with Univo Pharmaceuticals to identify and co-develop cannabis-derived formulations intended for various therapeutic applications, including cancer, inflammatory, autoimmune, and metabolic conditions. Originally incorporated in 1994 as Can-Fite Technologies Ltd., the company adopted its current name, Can-Fite BioPharma Ltd., in January 2001 and is headquartered in Petah Tikva, Israel.

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Pluri Inc., a biotechnology company established in 2001 and headquartered in Haifa, Israel, specializes in developing innovative placenta-derived cell therapies. Its core mission is to tackle a range of inflammatory conditions, muscle injuries, and hematological disorders. The company's therapeutic pipeline centers on placental expanded (PLX) cell technology. A key candidate, PLX-PAD, is in advanced clinical development, with ongoing Phase III trials for muscle recovery after hip fracture surgery, Phase II studies for acute respiratory distress syndrome associated with COVID-19, and Phase I/II trials for steroid-refractory Graft-versus-Host Disease. Additionally, Pluri is advancing PLX-R18, a treatment designed to aid in incomplete hematopoietic recovery following hematopoietic cell transplantation and to address acute radiation syndrome. The company rebranded to Pluri Inc. in July 2022, having previously operated as Pluristem Therapeutics Inc.

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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.

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Denali Therapeutics Inc. is a biopharmaceutical firm, founded in 2013 and based in South San Francisco, California, which focuses on identifying and advancing treatments for neurodegenerative conditions within the United States. The company, initially known as SPR Pharma Inc. until its renaming in March 2015, possesses a robust development pipeline. This includes BIIB122/DNL151, a small molecule inhibitor targeting leucine-rich repeat kinase 2 (LRRK2), currently in Phase 1 and Phase 1b clinical trials for Parkinson's disease. Another candidate, DNL310, is progressing through Phase 1/2 clinical studies for Hunter syndrome. For amyotrophic lateral sclerosis (ALS), DNL343 is in Phase 1 trials, while AR443820/DNL788 has successfully concluded its Phase 1 clinical trial for ALS, multiple sclerosis (MS), and Alzheimer's disease. Furthermore, SAR443122/DNL758 is undergoing Phase 2 clinical trials for cutaneous lupus erythematosus. Denali Therapeutics is engaged in extensive collaborative efforts with a range of partners, including Takeda Pharmaceutical Company, Genentech, Inc., Sanofi, F-star Gamma Limited, F-star Biotechnologische Forschungs-Und Entwicklungsges M.B.H, F-star Biotechnology Limited, SIRION Biotech GmbH, Genzyme Corporation, Harvard University, the Michael J. Fox Foundation, and Centogene. It also maintains a research and option agreement with Secarna Pharmaceuticals GmbH & Co. KG to co-develop antisense therapies specifically for neurodegenerative diseases.