The Broad Institute and MIT scientists who first harnessed CRISPR for mammalian genome editing have engineered a new molecular system for efficiently editing RNA in human cells. RNA editing, which can alter gene products without making changes to the genome, has profound potential as a tool for both research and disease treatment. In a paper […]

Kite Pharma, acquired by Gilead Sciences in October 2016 for $11.9 billion, has announced that the FDA has granted approval to Yescarta™ (axicabtagene ciloleucel), a CAR T (chimeric antigen receptor T cell) cancer therapy. CAR T therapy is a breakthrough in hematologic cancer treatment in which a patient’s own T cells are engineered to seek […]

Leading RNAi therapeutics company, Alnylam Pharmaceuticals, and Sanofi Genzyme have announced that the Phase 3 study of patisiran met its primary efficacy endpoint and all secondary endpoints. Patisiran is an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy. “We are very proud to report the first ever positive Phase 3 […]

On Wednesday, September 27th at 5:15 pm, the Women’s Leadership Forum & Dinner will take place at Biotech Week Boston at Hynes Convention Center, presented by Knect365. This will be an inspiring program focused on how we can meaningfully impact gender diversity within life sciences. A panel discussion will kick-off the evening with actionable strategies and […]

Alexion Pharmaceuticals has announced an operational plan to “re-align the global organization with its refocused corporate strategy.” The plan is expected to save approximately $270 million by 2019 and will reduce the Alexion’s global workforce by approximately 20 percent. The restructuring includes the closing of multiple Alexion sites, including the Alexion Rhode Island manufacturing facility. […]

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, […]