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Beam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. It develops BEAM-101 for the treatment of sickle cell disease or beta-thalassemia; and BEAM-302, a liver-targeting LNP formulation to treat severe alpha-1 antitrypsin deficiency; BEAM-201, an anti-CD7 CAR-T product candidate, which is in Phase 1/2 clinical trials for the treatment of refractory T-cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease 1a. The company has research collaboration agreement with Pfizer Inc., focus on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Apellis Pharmaceuticals to conduct preclinical research on target specific genes within the complement system in various organs, including the eye, liver, and brain; Verve Therapeutics, Inc., for cardiovascular disease treatments; Sana Biotechnology, Inc., to research, develop, and commercialize rights to CRISPR Cas12b for cell therapy programs; and Orbital Therapeutics design RNA for the prevention, treatment or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.

Mesoblast Limited engages in the development of regenerative medicine products in Australia, the United States, Singapore, and Switzerland. The company offers products in the areas of cardiovascular, spine orthopedic disorder, oncology, hematology, and immune-mediated and inflammatory diseases. Its proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage cells. The company offers Remestemcel-L that is in Phase III clinical trials for the treatment of systemic inflammatory diseases, including steroid refractory acute graft versus host disease, acute respiratory distress syndrome, and biologic refractory inflammatory bowel disease; and Remestemcel-L, which is in Phase III clinical trials to treat chronic heart failure and chronic low back pain due to degenerative disc disease. It is also developing MPC-300-IV to treat biologic refractory rheumatoid arthritis diabetic nephropathy; and MPC-25-IC for the treatment or prevention of acute myocardial infarction. It has strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for heart failure and MPC-25-IC for heart attacks in China; JCR Pharmaceuticals Co. Ltd. to treat wound healing in patients with epidermolysis bullosa; and Grünenthal to develops and commercializes cell therapy for the treatment of chronic low back pain. The company was incorporated in 2004 and is headquartered in Melbourne, Australia.

NantKwest, Inc., a clinical-stage immunotherapy company, develops immunotherapeutic treatments for cancer and viral infectious diseases in the United States. The company develops activated natural killer (aNK) cell, a natural killer cell-line and genetically modified derivative capable of killing cancer and virally infected cells; and Nant cancer vaccine, a personalized therapy that utilizes its off-the-shelf natural killer cells. It is also developing high-affinity natural killer (haNK) product candidates primarily for the treatment of various cancers; and tank and t-haNK product candidates to treat solid tumors. NantKwest, Inc. has a co-development agreement with Altor BioScience, LLC to collaborate on the development of therapeutic applications combining the company's proprietary natural killer cells with Altor's N-801 and/or N-803 products; collaboration with Be The Match BioTherapies for the development of cell therapy to prevent COVID-19 deaths; and strategic alliance with Immunitybio. It also has a collaboration with CBR Systems, Inc. to develop a COVID-19 treatment leveraging newborn stem cells. The company was formerly known as Conkwest, Inc. and changed its name to NantKwest, Inc. in July 2015. NantKwest, Inc. was founded in 2002 and is headquartered in San Diego, California.

Vaxcyte, Inc., a clinical-stage biotechnology vaccine company, develops novel protein vaccines to prevent or treat bacterial infectious diseases. Its lead vaccine candidate is VAX-24, a 24-valent investigational pneumococcal conjugate vaccine for the prevention of invasive pneumococcal disease. The company also develops VAX-31 to protect against emerging strains and to help address antibiotic resistance; VAX-A1, a novel conjugate vaccine candidate to prevent disease caused by Group A Streptococcus; VAX-PG, a novel protein vaccine candidate targeting keystone pathogen responsible for periodontitis; and VAX-GI to prevent Shigella, a bacterial illness. The company was formerly known as SutroVax, Inc. and changed its name to Vaxcyte, Inc. in May 2020. Vaxcyte, Inc. was incorporated in 2013 and is headquartered in San Carlos, California.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy. It also develops RGX-121 for the treatment of mucopolysaccharidosis type II that is in Phase III clinical trial; RGX-111 for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II; and RGX-381 to treat the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.