Genzyme Corporation, now a specialty care division of Sanofi, is a biotechnology company focused on developing treatments for rare diseases, multiple sclerosis, immunology, and oncology. Established in 1981 in Boston, Genzyme has built a strong reputation for its pioneering work in therapies for genetic disorders, particularly lysosomal storage disorders. The company operates through four main business segments: Genetic Diseases, Cardiometabolic and Renal, Biosurgery, and Hematologic Oncology. The Genetic Diseases segment focuses on therapeutic products for genetic conditions, while the Cardiometabolic and Renal segment addresses renal diseases and related endocrine and cardiovascular issues. Biosurgery is dedicated to biotherapeutics and biomaterials, and Hematologic Oncology develops therapies for various cancer types. Since becoming part of Sanofi in 2011, Genzyme has continued to advance its mission of providing innovative solutions for complex medical conditions, enhancing the lives of patients worldwide.
Voyager Therapeutics is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe neurological diseases. The company's lead candidate, VY-AADC, is currently undergoing an open-label Phase 1b clinical trial for Parkinson's disease. In addition to this, Voyager's preclinical pipeline includes therapies targeting amyotrophic lateral sclerosis, Huntington's disease, Friedreich's ataxia, and various tauopathies and synucleinopathies, which encompass conditions like Alzheimer's disease and Lewy Body Dementia. Voyager Therapeutics emphasizes advancing adeno-associated virus (AAV) gene therapy through innovations in vector optimization, dosing techniques, and production processes. The company has established strategic collaborations with the University of Massachusetts and ClearPoint Neuro, along with partnerships with Brammer Bio and Fujifilm Diosynth Biotechnologies to enhance its gene therapy programs. Furthermore, Voyager has a collaboration agreement with Neurocrine Biosciences for the research, development, and commercialization of AAV-based gene therapies. Founded in 2013, Voyager Therapeutics is committed to addressing critical unmet medical needs in the field of central nervous system disorders.
Alnylam Pharmaceuticals is a biopharmaceutical company dedicated to the discovery, development, and commercialization of RNA interference (RNAi) therapeutics. The company focuses on addressing various medical conditions, including genetic diseases, cardio-metabolic disorders, hepatic infectious diseases, and central nervous system and ocular diseases. Its marketed products include ONPATTRO, which treats polyneuropathy associated with hereditary transthyretin-mediated amyloidosis, and GIVLAARI, aimed at adults with acute hepatic porphyria. Alnylam's pipeline includes investigational therapies such as givosiran for acute hepatic porphyria, lumasiran for primary hyperoxaluria type 1, patisiran for transthyretin amyloidosis with cardiomyopathy, and vutrisiran for transthyretin amyloidosis. The company fosters strategic alliances with organizations like Sanofi Genzyme and Ionis Pharmaceuticals, and collaborates with Vir Biotechnology to advance RNAi therapeutics for coronavirus infections. Founded in 2002, Alnylam Pharmaceuticals is headquartered in Cambridge, Massachusetts.
Humanigen is a biopharmaceutical company that uses its biologic capabilities and its proprietary platform technology to develop first-in-class human antibody therapeutics. It focuses on preventing the serious and potentially life-threatening side-effects associated with chimeric antigen receptor T-cell (CAR-T) therapy. Its pipeline includes programs such as Lenzilumab, Ifabotuzumab, and HGen005.
Bioenvision a biopharmaceutical company, engages in the acquisition, development, and marketing of compounds and technologies primarily for the treatment of cancer. Its lead products include Evoltra (clofarabine) which has marketing approval in both the European Union and United States for the treatment of pediatric relapsed or refractory acute lymphoblastic leukemia and Modrenal (trilostane), which has marketing approval in the United Kingdom for the treatment of post-menopausal breast cancer following relapse to initial hormone therapy.
AnorMED
Acquisition in 2006
AnorMED, Inc., a biopharmaceutical company, engages in the discovery, development, and commercialization of therapeutic drugs for the treatment of hematology, human immunodeficiency virus (HIV), and oncology. Its lead drug product candidate include MOZOBIL, which is under phase III clinical trial is used for cancer patients undergoing stem cell transplantation; and AMD070, which is under phase Ib/IIa trial is used as a HIV entry inhibitor.
Peptimmune is a privately held clinical stage biotechnology company focused on utilizing both novel and proven technologies to improve the therapeutic management of chronic autoimmune and metabolic disorders. The company is developing second generation therapeutics that are expected to result in safer and more effective products for obesity, multiple sclerosis, rheumatoid arthritis and pemphigus vulgaris.
BoneCare International
Acquisition in 2005
Bone Care International, Inc., a pharmaceutical company, engages in the discovery, development, and commercialization of therapeutic products to treat the medical needs of patients with debilitating conditions and life-threatening diseases.
Verigen AG, a biotherapy company, provides cell therapy products for cartilage repair. It has operations in Germany, Australia, Denmark, Italy, England, Europe, and the United States.
ILEX Oncology,Inc.
Acquisition in 2004
ILEX is a drug development company focused exclusively on oncology. The company uses its expertise in the identification, development, manufacturing and regulatory approval process of oncology drugs to: (i) develop cancer treatment products, (ii) develop products for the prevention of cancer and (iii) provide contract research services to pharmaceutical and biotechnology companies. ILEX's objective is to be a leading worldwide oncology drug development company. The company has nine cancer treatment compounds under development, including five in Phase II or Phase III clinical trials. In October 1996, ILEX submitted a NDA to the FDA seeking accelerated marketing approval for MGBG as a second-line treatment for patients with AIDS-related non-Hodgkin's lymphoma. Additionally, ILEX has two chemoprevention compounds under development. Consistent with its strategy, the Company has established worldwide development and marketing collaborations with Sanofi, Janssen and MGI Pharma for certain of its compounds. In addition to its proprietary product development programs, the company offers CRO services to both the pharmaceutical and biotechnology industries. ILEX believes it is currently the only full-service provider of contract research services focused exclusively on oncology. ILEX was formed by CTRC in December 1993 for the purpose of conducting certain advanced drug development programs and pursuing commercial opportunities which were not within the scope of CTRC's tax-exempt purpose.
SangStat
Acquisition in 2003
SangStat Medical Corporation, a global biopharmaceutical company, engages in the discovery, development, and marketing of therapeutic products for immunology, transplantation medicine, hematology/oncology, and autoimmune disorders.
Biomatrix
Acquisition in 2000
Genzyme Biosurgery Corporation was engaged in the development, manufacture, marketing, and sale of a series of proprietary viscoelastic products, called hylans that are used in therapeutic medical applications and skin care.
Pharmagenics
Acquisition in 1997
PharmaGenics develops therapeutic products for cancer through the use of genomics technology.
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