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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.

China Biologic Products Holdings, Inc. engages in the research, development, manufacture, and sale of human plasma-based biopharmaceutical products to hospitals and inoculation centers in the People's Republic of China. It offers human albumin for treating shock caused by blood loss trauma or burn; raised intracranial pressure caused by hydrocephalus or trauma; oedema or ascites caused by hepatocirrhosis and nephropathy; and neonatal hyperbilirubinemia, as well as for the prevention and treatment of low-density-lipoproteinemia. The company also provides human immunoglobulin and IVIG for original and secondary immunoglobulin deficiency, auto-immune deficiency, and immunoglobulin G secondary deficiency; human hepatitis B immunoglobulin for the prevention of measles and contagious hepatitis; human rabies immunoglobulin primarily for passive immunity from bites or claws by rabies or other infected animals; and human tetanus immunoglobulin for the prevention and therapy of tetanus. In addition, it offers placenta polypeptide for the treatment of cell immunity deficiency diseases, viral infection, and leucopenia, as well as assists in postoperative healing; factor VIII for treating coagulopathies; human fibrinogen; and human prothrombin complex concentrate for treating congenital and acquired clotting factor II, VII, IX, X deficiency, as well as excessive anticoagulant, vitamin K deficiency, etc. Further, the company is developing Human fibrinogen for the treatment for lack of fibrinogen and increase human fibrinogen concentration; and artificial dura and spinal dura mater products for use in brain and spinal surgeries. The company sells its products directly, as well as through distributors. The company was formerly known as China Biologic Products, Inc. and changed its name to China Biologic Products Holdings, Inc. in July 2017. China Biologic Products Holdings, Inc. is headquartered in Beijing, the People's Republic of China.

Spark Therapeutics, Inc. focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases. Its products include LUXTURNA (voretigene neparvovec) for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells. The company's gene therapy product candidates comprise SPK-8011 and SPK-8016 for hemophilia; SPK-7001 for choroideremia; and SPK-9001 for hemophilia B. It is also developing other liver-directed gene therapies, including SPK-3006 for Pompe disease; and neurodegenerative disease product candidates to address Huntington's disease and others, as well as TPP1 deficiency, which is a form of Batten disease. The company's preclinical programs targets inherited retinal diseases, including Stargardt's disease. The company has collaboration agreement with Pfizer, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B. It also has licensing and commercialization agreement with Novartis to develop and commercialize voretigene neparvovec outside the United States. Spark Therapeutics, Inc. was founded in 2013 and is headquartered in Philadelphia, Pennsylvania.

Repligen Corporation develops and commercializes bioprocessing technologies and systems for use in biological drug manufacturing process in North America, Europe, the Asia Pacific, and internationally. It offers Protein A ligands that are the binding components of Protein A affinity chromatography resins; and cell culture growth factor products. The company's chromatography products include OPUS pre-packed chromatography columns, which are used in the purification of biologics; and OPUS smaller-scale columns that are used in the high throughput process development screening, viral clearance validation studies, and scale down validation of chromatography processes. It also offers ELISA test kits; and chromatography resins under the CaptivA brand. In addition, the company provides filtration products, such as XCell Alternating Tangential Flow systems that are filtration devices used in upstream perfusion and cell culture processing; TangenX flat sheet cassettes, which are used in downstream biologic drug concentration, buffer exchange, and formulation processes; KrosFlo tangential flow filtration and tangential flow depth filtration systems; Spectra/Por laboratory and process dialysis products, and ProConnex TFDF flow paths. Further, it provides process analytics products, such as slope spectroscopy systems under the SoloVPE, FlowVPE, and FlowVPX brands. The company sells its products to life sciences, biopharmaceutical, and diagnostics companies; laboratory researchers; and contract manufacturing organizations. Repligen Corporation has collaboration agreements with Navigo Proteins GmbH to develop multiple affinity ligands. The company was incorporated in 1981 and is headquartered in Waltham, Massachusetts.

Acceleron Pharma, Inc. is a biopharmaceutical company, which engages in the discovery, development and commercialization of therapeutics to treat serious and rare diseases. Its product candidates include Luspatercept, designed to patients with chronic anemia associated within a wide range of blood diseases; ACE-083, designed for the treatment of focal muscle disorders; and Sotatercept, designed to treat pulmonary arterial hypertension. The company was founded by John L. Knopf and Thomas P. Maniatis in June 2003 and is headquartered in Cambridge, MA.