MOLMED is a biotechnology company founded in 1996 in Milan. It originally operated as contract manufacturing organization and other services that to companies developing gene therapy and cell therapy products. In 2000 the company changed its business model and started to develop products of its own. [1]
Biotechnology is the broad area of biology involving living systems and organisms to develop or make products, or "any technological application that uses biological systems, living organisms, or derivatives thereof, to make or modify products or processes for specific use". Depending on the tools and applications, it often overlaps with the (related) fields of molecular biology, bio-engineering, biomedical engineering, biomanufacturing, molecular engineering, etc.
A contract manufacturing organization (CMO), sometimes called a contract development and manufacturing organization (CDMO), is a company that serves other companies in the pharmaceutical industry on a contract basis to provide comprehensive services from drug development through drug manufacturing. This allows major pharmaceutical companies to outsource those aspects of the business, which can help with scalability or can allow the major company to focus on drug discovery and drug marketing instead.
In the medicine field gene therapy is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease. The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.
In 2001 it signed an agreement with San Raffaele Hospital, that gave MolMed an option to license any gene therapy or cell therapy invention made at the hospital in the fields of cancer and HIV. [2]
The San Raffaele Hospital is a university hospital situated in Segrate, the Province of Milan, Italy. It was founded in 1969 by don Luigi Maria Verzé, president of "San Raffaele del Monte Tabor Foundation".
The company was listed on the Milan Stock Exchange in 2008.[ citation needed ]
In early 2015 MolMed partnered with GlaxoSmithKline related to GSK's acquisition of Strimvelis; MolMed had been involved in the early development of the product. [3]
GlaxoSmithKline plc (GSK) is a British multinational pharmaceutical company headquartered in Brentford, London. Established in 2000 by a merger of Glaxo Wellcome and SmithKline Beecham, GSK was the world's sixth largest pharmaceutical company as of 2015, after Pfizer, Novartis, Merck, Hoffmann-La Roche and Sanofi.
Strimvelis is the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID. ADA-SCID is estimated to occur in approximately 15 patients per year in Europe.
Genentech, Inc., is a biotechnology corporation which became a subsidiary of Roche in 2009. Genentech Research and Early Development operates as an independent center within Roche.
Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and delivery of therapies for the treatment of neurodegenerative, hematologic, and autoimmune diseases to patients worldwide.
Metachromatic leukodystrophy is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of sphingolipids. Leukodystrophies affect the growth and/or development of myelin, the fatty covering which acts as an insulator around nerve fibers throughout the central and peripheral nervous systems. MLD involves cerebroside sulfate accumulation. Metachromatic leukodystrophy, like most enzyme deficiencies, has an autosomal recessive inheritance pattern.
David Andrew Sinclair is an Australian biologist and professor of genetics best known for his advocacy for resveratrol as an anti-aging dietary supplement and potential medication.
Tolerx, Inc. was a biopharmaceutical company headquartered in Cambridge, Massachusetts. The company was focused on discovering and developing new therapies designed to treat patients by reprogramming the immune system, allowing for long-term remission of immune-related diseases after a short course of therapy. Targeted diseases include type 1 diabetes, rheumatoid arthritis, Inflammatory bowel disease (IBD), cancer, chronic and viral diseases. In 2008, Tolerx was named one of Fierce Biotech’s Fierce 15. In October 2011, Tolerx was shut down due to an unsuccessful Phase III trial in patients recently diagnosed with Type 1 diabetes.
Christoph Westphal, M.D., Ph.D., is a biomedical entrepreneur.
Michelle Dipp, M.D., Ph.D., is a biotech business executive and venture capitalist. As of 2017 she was co-founder and executive chairwoman of OvaScience, an In vitro fertilisation services and company, and founder and partner of Longwood Fund, a healthcare venture capital firm.
Santaris Pharma A/S is a biopharmaceutical company founded in 2003 in Copenhagen, Denmark with a small branch in San Diego, California that opened in 2009. Created by a merger between Cureon and Pantheco, Santaris Pharma A/S has become a leading clinical-stage biopharmaceutical company that develops RNA-targeted medicines using a Locked Nucleic Acid (LNA) Drug Platform and Drug Development Engine.
Atlas Venture is an early-stage venture capital firm that invests in life sciences startup companies in the U.S.. Atlas is headquartered in Cambridge, Massachusetts where the majority of its investments are located. Atlas rolled out its eleventh biotech fund totaling $350 million in June 2017, after closing its tenth fund in April 2015 with $280 million in commitments.
Kite Pharma, a subsidiary of Gilead Sciences, develops cancer immunotherapy products with a primary focus on genetically engineered autologous T cell therapy with chimeric antigen receptors (CAR-T).
Amicus Therapeutics is a public American biopharmaceutical company based in Cranbury, New Jersey. The company went public in 2007 under the NASDAQ trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates.
Immutep Ltd is a biotechnology company working primarily in the field of cancer immunotherapy using the LAG3 immune control mechanism. The company was originally built on CVac, a therapeutic cancer vaccine. In late 2014 the privately held French immunotherapy company Immutep SA was purchased by Prima Biotech.
GSK2831781 is a monoclonal antibody being developed by GlaxoSmithKline (GSK) for autoimmune diseases. The antibody targets the T cell activation marker LAG-3, which is mainly expressed in inflamed tissues. In GSK's March 2015 Product development pipeline document the antibody is listed under 'Immuno-inflammation' candidates. GSK2831781 entered a Phase I clinical trial in psoriasis early in 2015.
GVAX is a cancer vaccine composed of whole tumor cells genetically modified to secrete the immune stimulatory cytokine, granulocyte-macrophage colony-stimulating factor (GM-CSF), and then irradiated to prevent further cell division. The product exists as both autologous and allogeneic therapy.
Axovant Sciences Ltd. is a clinical-stage pharmaceutical company that develops, in association with other pharmaceutical companies, small molecule drugs and gene therapies to treat neurological and neuromuscular disorders. The company is legally incorporated in Bermuda, but has headquarters in London, along with secondary offices in Basel, Switzerland and New York City. The company was founded by former hedge fund analyst Vivek Ramaswamy in 2014 as a wholly owned subsidiary of Roivant Sciences.
Editas Medicine is a discovery-phase pharmaceutical company based in Cambridge, Massachusetts which aims to develop therapies based on CRISPR–Cas9 gene editing technology. It was founded in 2013 with funding from Third Rock Ventures, Polaris Partners and Flagship Ventures and licensed CRISPR patents from the Broad Institute's Feng Zhang, patents from Harvard's David Liu and George Church and patents from Partners Healthcare-MGH's J. Keith Joung. These four were co-founders and scientific advisory board members along with Jennifer Doudna.
Craig M. Crews, Ph.D. is an American scientist at Yale University. As the Lewis B. Cullman Professor of Molecular, Cellular and Developmental Biology, Crews also holds joint appointments in the departments of Chemistry and Pharmacology. He currently serves as an Editor of Cell Chemical Biology and is the Executive Director of the Yale Center for Molecular Discovery. His research interests focus on Chemical Biology, particularly on controlled proteostasis, which has led to the development of the FDA approved anti-cancer drug Carfilzomib (Kyprolis®).
Roivant Sciences Ltd. is a pharmaceutical company that builds subsidiary biotech and health technology companies. Roivant's initial strategy of in-licensing late-stage drug candidates through subsidiaries was later expanded to develop earlier stage drug candidates and other healthcare technologies through those same subsidiaries.
Intellia Therapeutics is a biotechnology company developing biopharmaceuticals using a CRISPR gene editing system invented by Jennifer Doudna and colleagues at University of California. The company has partnerships with Novartis and Regeneron.