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BioCryst Pharmaceuticals, Inc., a biotechnology company, develops oral small-molecule and protein therapeutics to treat rare diseases. The company markets peramivir injection, an intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza under the RAPIVAB, RAPIACTA, and PERAMIFLU names; and ORLADEYO, an oral serine protease inhibitor to treat hereditary angioedema. It is also developing BCX10013, an oral factor D inhibitor for complement-mediated diseases. The company has collaborations and in-license relationships with the Torii Pharmaceutical Co., Ltd.; Seqirus UK Limited; Shionogi & Co., Ltd.; Green Cross Corporation; National Institute of Allergy and Infectious Diseases; Biomedical Advanced Research and Development Authority; the U.S. Department of Health and Human Services; and The University of Alabama at Birmingham, as well as Albert Einstein College of Medicine of Yeshiva University and Industrial Research, Ltd. BioCryst Pharmaceuticals, Inc. was founded in 1986 and is headquartered in Durham, North Carolina.

bluebird bio, Inc., a biotechnology company, researches, develops, and commercializes gene therapies for severe genetic diseases. Its product candidates for severe genetic diseases include ZYNTEGLO (betibeglogene autotemcel) for the treatment of transfusion-dependent ß-thalassemia; lovotibeglogene autotemcel for the treatment of sickle cell disease (SCD); and SKYSONA (elivaldogene autotemcel) to treat cerebral adrenoleukodystrophy. The company's clinical development programs include HGB-205, HGB-206, and HGB-210 to evaluate the safety and efficacy of lovo-cel in the treatment of patients with SCD; and HGB-204, HGB-205, HGB-207, and HGB-212 to evaluate the safety and efficacy of beti-cel in the treatment of patients with ß-thalassemia. It has license agreement with Orchard Therapeutics Limited. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was incorporated in 1992 and is headquartered in Somerville, Massachusetts.

Kymera Therapeutics, Inc., a biopharmaceutical company, focuses on discovering and developing novel small molecule therapeutics that selectively degrade disease-causing proteins by harnessing the body's own natural protein degradation system. It engages in developing IRAK4 program, which is in Phase II clinical trial for the treatment of immunology-inflammation diseases, including hidradenitis suppurativa, atopic dermatitis; STAT3 program for the treatment of hematologic malignancies and solid tumors, as well as autoimmune diseases and fibrosis; and MDM2 program to treat hematological malignancies and solid tumors. The company develops STAT6, a Type 2 inflammation in allergic diseases; and TYK2, a treatment for inflammatory bowel disease, psoriasis, psoriatic arthritis, and lupus. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.

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.

Rubius Therapeutics, Inc., a biopharmaceutical company, focuses on the development of red cell therapeutics for the treatment of cancer and autoimmune diseases. The company was formerly known as VL26, Inc and as changed its name to Rubius Therapeutics, Inc. in 2015. Rubius Therapeutics, Inc. was incorporated in 2013 and is based in Foxborough, Massachusetts.