Human gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use. Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms: Replacing a […]

The FDA issued a historic action on August 30, making the first gene therapy available in the United States. With its approval of Novartis’ Kymriah therapy for a form of acute lymphoblastic leukemia (ALL), the FDA ushered in a new approach to the treatment of cancer and other serious and life-threatening diseases. “We’re entering a […]

Cambridge-based Ironwood Pharmaceuticals has announced Duzallo was approved by the FDA as a treatment for hyperuricemia, or elevated serum uric acid levels (sUA) in the blood, associated with gout. Duzallo is the first drug that combines the current standard of care for the treatment of hyperuricemia associated with gout, the xanthine oxidase inhibitor (XOI) allopurinol, […]

The innate immune system is the body’s first line of immunological response and the master regulator of subsequent immune activity. In cancer, the innate immune system mediates presentation of tumor antigens, priming and activation of tumor-specific immune cells that enable optimal development of adaptive anti-tumor immunity. Existing immunotherapies are mostly focused on harnessing the adaptive […]

Bristol-Myers Squibb (BMS) has agreed to pay $300 million to acquire Cambridge-based IFM Therapeutics, a venture-backed startup founded in 2015 focused on innate immunity and the role it plays in regulating the immune system. IFM’s team has discovered and developed small molecules that modulate novel targets in the innate immune system as next-generation therapies for […]

Avecia, a pharmaceutical manufacturer in Milford, says it is building what will become the largest manufacturing site in the world for a specialized drug ingredient called oligonucleotide. The expansion, which will add 20,000 square feet of space, will more than double the Milford site’s total capacity, the company said in a recent announcement. Oligonucleotide, a […]

The MassBioEd Foundation released its 2017 Annual Job Trends Forecast at Life Sciences Workforce 2017, its second annual conference on life sciences workforce trends.  The event was held at Northeastern University and featured twenty-seven industry and higher education speakers, as well as Lt. Governor Karyn Polito, who provided keynote remarks. The Forecast included the following […]

Novartis has announced that the FDA Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of CTL019 (tisagenlecleucel), an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of children and young adults with B-cell acute lymphoblastic leukemia (ALL) that has relapsed or resisted treatment (r/r). A Biologics License Application (BLA) for this […]

Alnylam and Sanofi Genzyme have announced new positive results from the ongoing Phase 2 study with fitusiran in patients with hemophilia A and B, with or without inhibitors. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B, that is designed to lower levels of AT […]