RESEARCH TRIANGLE PARK, N.C. — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Spanish Ministry for Health (Ministerio de Sanidad) has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older. “We have an...
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NEW HAVEN, Conn. and LEUVEN, Belgium – Biohaven Therapeutics Ltd., a subsidiary of Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), along with CD3 and LICR at KU Leuven, announced that they have entered into an exclusive global license and research agreement to develop and commercialize first-in-class TRPM3 antagonists to address...
New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function Data from the largest retrospective, multi-year observational study of more than 600 children and adults with hypochondroplasia found significantly higher rates of...
Tosse, France – After decades of limited progress—owing to the difficulty of treating the disease and resultant market risk—glioblastoma research is entering a new phase spurred by smarter trials, targeted funding and renewed interest from companies like Merck and Jazz Pharmaceuticals. Glioblastoma, a deadly brain cancer, is notoriously difficult to...
Santa Cruz, Calif. – A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications. They’re rare, they’re confounding, and R&D for therapies to address them is painfully underfunded. Rare...
CAMBRIDGE, Mass. – bluebird bio, Inc. (NASDAQ: BLUE) today announced the company has completed the tax-free spin-off of its oncology programs and portfolio into 2seventy bio, Inc., an independent, publicly-traded company. bluebird bio will continue its work focused on severe genetic disease, with three near-term opportunities to bring transformative gene...
CAMBRIDGE, Mass. – bluebird bio, Inc. (NASDAQ:BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2020 and shared recent operational progress. “For over ten years, bluebird bio has been pioneering gene therapies for patients with rare diseases and cancer, and our...
NEW YORK – Quest Diagnostics subsidiary Blueprint Genetics said on Thursday that it has partnered with the Helsinki Biobank and Helsinki University Hospital for a research initiative called Rare3k that aims to develop algorithms to accelerate the identification of patients who could benefit from genetic testing for certain rare diseases....
Cambridge, Mass. — Blueprint Medicines Corporation (Nasdaq: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, announced that, effective April 1, 2023, the Compensation Committee of Blueprint Medicines’ Board of Directors granted non-qualified stock options to purchase an aggregate of 5,933 shares of its...
Hamburg, Germany – Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) today announced that Bristol Myers Squibb has decided to exercise their option to extend its partnership with Evotec in the field of targeted protein degradation. Evotec and Bristol Myers Squibb (the successor in interest to Celgene) initiated this...