Sanofi Genzyme is the specialty care global business unit of Sanofi, focusing on the treatment of rare diseases, multiple sclerosis, immunology, and oncology. Established in 1981 as Genzyme and headquartered in Cambridge, Massachusetts, the division is recognized for its pioneering work in developing therapies for rare genetic disorders and its innovative contributions to medical science and biomanufacturing. Sanofi Genzyme's portfolio includes therapeutic products that address a range of conditions, including lysosomal storage disorders, renal diseases, and various cancers. The organization is dedicated to advancing new therapies to provide hope to patients and their families facing complex and often difficult-to-diagnose health challenges. Since becoming part of Sanofi in 2011, Sanofi Genzyme has expanded its focus to include multiple sclerosis and has established itself as a leader in these therapeutic areas through close collaboration with physician and patient communities.
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 specializing in the discovery, development, and commercialization of RNA interference (RNAi) therapeutics. Its innovative pipeline targets a range of serious conditions, including genetic disorders, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system and ocular diseases. The company has successfully brought several RNAi therapeutics to market, including ONPATTRO for hereditary transthyretin-mediated amyloidosis and GIVLAARI for acute hepatic porphyria. Additionally, Alnylam is advancing other investigational therapies, such as givosiran for acute hepatic porphyria and lumasiran for primary hyperoxaluria type 1. The company collaborates with various partners, including Sanofi Genzyme and Vir Biotechnology, to enhance its research and development efforts. Founded in 2002 and headquartered in Cambridge, Massachusetts, Alnylam Pharmaceuticals aims to transform treatment approaches for patients with rare and debilitating diseases through its RNAi technology.
Humanigen, Inc. is a clinical-stage biopharmaceutical company based in Burlingame, California, that specializes in the development of proprietary monoclonal antibodies for immunotherapy and oncology. The company's lead product candidate, Lenzilumab, targets granulocyte-macrophage colony-stimulating factor and is currently undergoing Phase Ib/II clinical trials for chronic myelomonocytic leukemia and juvenile myelomonocytic leukemia. Additionally, Humanigen is developing Ifabotuzumab, which targets the EphA3 receptor and has completed the Phase I dose escalation in multiple hematologic malignancies, as well as HGEN005, an anti-EMR1 monoclonal antibody in pre-clinical stages for eosinophilic diseases. The company collaborates with Kite Pharma to conduct a multi-center Phase Ib/II study of Lenzilumab in patients with relapsed or refractory diffuse large B-cell lymphoma and has partnered with the Department of Defense to further develop Lenzilumab for potential emergency use in COVID-19. Founded in 2000, Humanigen was formerly known as KaloBios Pharmaceuticals and rebranded in August 2017.
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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