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Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

Magenta Therapeutics, Inc., a biotechnology company focus on improving stem cell transplantation. Its product candidates are designed to bring the curative power of stem cell transplant to patients with blood cancers, genetic diseases, and autoimmune diseases. The company's product portfolio includes MGTA-117, an anti-CD117 antibody conjugated to an amanitin payload that targets hematopoietic stem cells (HSCs) and leukemia cells; and MGTA-45, an anti-human CD45 antibody conjugated to a DNA-interacting payload for HSCs, leukemia cells, and immune cells. In addition, it focus on development of ADC-based conditioning program that targets a receptor of T cells; and novel conditioning and translation stem cell sciences, as well as develops cell therapy program, E478, which is a small molecule aryl hydrocarbon receptor antagonist for stem cell-based gene therapy and genome editing. In addition, the company has a research and clinical collaboration agreement with AVROBIO, Inc. for the treatment of lysosomal storage disorders; and Beam Therapeutics, Inc. to evaluate the potential utility of MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia. Magenta Therapeutics, Inc. was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Neptune Wellness Solutions Inc., a health and wellness products company, specializes in the extraction, purification and formulation of cannabis products, as well as other specialty ingredients, such as MaxSimil, a patented ingredient that enhances the absorption of lipid-based nutraceuticals, and various other marine and seed oils. Its custom formulations are available in various delivery forms, such as softgels, liquid solutions, nutritional emulsions, and chewables. Neptune Wellness Solutions Inc. was founded in 1998 and is headquartered in Laval, Canada.

Progenics Pharmaceuticals, Inc., an oncology company, develops, manufactures, and commercializes pharmaceutical products and other technologies to target, diagnose, and treat cancer cancer in the United States and internationally. The company's product candidates include Azedra, a radiotherapeutic product candidate for the treatment of iobenguane scan positive, unresectable, and locally advanced or metastatic pheochromocytoma or paraganglioma for adult and pediatric patients; PyL, a clinical-stage fluorinated prostate specific membrane antigen (PSMA)-targeted PET/CT imaging agent for prostate cancer; and 1095, a PSMA-targeted Iodine-131 labeled small molecule, which is in Phase II clinical trial for the treatment of metastatic castration-resistant prostate cancer. Its product candidates also comprise PSMA TTC, a thorium-227 labeled PSMA-targeted antibody therapeutic that is in Phase I clinical trial for treatment of metastatic prostate cancer; and Leronlimab, a humanized monoclonal antibody, which is in Phase III development for the treatment of HIV infection. The company also offers Relistor-subcutaneous injection for the treatment of opioid-induced constipation (OIC) in adults with chronic non-cancer pain or advanced-illness adult patients; Relistor tablets for the treatment of OIC in adults with chronic non-cancer pain; PSMA AI, an imaging analysis technology that uses artificial intelligence and machine learning to quantify and automate the reading of PSMA targeted imaging; and automated bone scan index, a software that quantifies the hotspots on bone scans and calculates the bone scan index value. It has license agreement with Salix Pharmaceuticals, Inc. for the development and commercialization of Relistor worldwide; and Amgen Fremont, Inc. to use its XenoMouse technology for generating human antibodies to PSMA. Progenics Pharmaceuticals, Inc. was founded in 1986 and is based in New York City, New York.

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 RNA 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 RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; siRNA clinical candidate for the treatment of obesity and other metabolic disorders; WVE-N531, a exon skipping oligonucleotide for the treatment of duchenne muscular dystrophy; and WVE-003, an antisense silencing 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 incorporated in 2012 and is based in Singapore.