SEATTLE and SAN RAFAEL, Calif. – The Allen Institute and BioMarin Pharmaceutical Inc. announced a collaboration today that will use technologies developed at the Allen Institute to create new gene therapies aimed at rare genetic diseases of the central nervous system. BioMarin will receive an exclusive license to each program...
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Seattle, WA — With pediatric inflammatory bowel disease (IBD) diagnoses on the rise globally, a clinical trial in Seattle aims to transform treatment approaches through deep molecular profiling. Recruitment has begun for the Seattle STRIDE study, a joint effort by Seattle Children’s Research Institute and the Allen Institute for Immunology....
Chicago, Illinois – Patients with muscle-invasive urothelial cancer and a high risk of recurrence after surgery may have a new treatment option. The Alliance for Clinical Trials in Oncology today announced positive results from the phase III AMBASSADOR (A031501) trial for the adjuvant treatment of patients with localized muscle-invasive urothelial...
SOUTH SAN FRANCISCO, Calif. — Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer and autoimmune diseases, announced that it has received a $15 million grant from the California Institute for Regenerative Medicine (CIRM) to support the clinical...
In a recent study, patients with myelodysplastic syndrome (MDS) who have myelofibrosis (MF) showed a worse prognosis when MF was of greater severity. However, this pattern of severity influencing prognosis appeared negated with allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study findings were reported in the journal Experimental Hematology & Oncology....
SAN FRANCISCO – AllStripes, a healthcare technology company dedicated to accelerating treatment research for patients with rare diseases, today announced a collaboration with the National Center for Advancing Translational Sciences (NCATS), an institute of the NIH established to transform the translational process so that new treatments and cures for disease...
PARIS, France and CAMBRIDGE, MA, USA – Sanofi and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development and commercialization of certain products for the treatment of rare genetic diseases. Specifically: Sanofi will obtain global development and commercialization rights...
CAMBRIDGE, Mass. — Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA® (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to...
MISSISSAUGA, ON – Alnylam Canada ULC is pleased to announce that Health Canada has issued a Notice of Compliance (NOC) authorizing AMVUTTRA® (vutrisiran) for the treatment of cardiomyopathy in adult patients with wild-type or hereditary transthyretin-mediated amyloidosis (wtATTR or hATTR amyloidosis).1 The approval broadens the indication for AMVUTTRA®, which now becomes the first and...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has started a clinical study of a biannual dosing regimen of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The study is being conducted during a randomized treatment extension...