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HYOGO, Japan--(BUSINESS WIRE)--JCR announced that is has achieved a key research milestone in one of its collaborative programs with Alexion, AstraZeneca Rare Disease....

MINNEAPOLIS--(BUSINESS WIRE)--A study by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) found that a donor search prognosis-based strategy helps physicians move patients with blood cancers and other blood diseases to hematopoietic cell transplant (HCT) faster, resulting in similar transplant success regardless of likelihood of finding a matched unrelated donor (MUD). Investigators from the consortium of U.S. transplant centers, CIBMTR® (Center for International Blood and Marr...

KYOTO & FUJISAWA, Japan--(BUSINESS WIRE)--xFOREST Therapeutics Co., Ltd. (“xFOREST”) and Axcelead Drug Discovery Partners, Inc. (“Axcelead DDP”) today announced the launch of a joint research collaboration aimed at developing RNA structure-targeted small molecule therapeutics for multiple diseases. xFOREST has developed a proprietary drug discovery platform, “FOREST Technologies,” which integrates a custom-designed RNA structure library and high-throughput screening technology. The platform spe...

REDWOOD CITY, Calif.--(BUSINESS WIRE)--The Siebel Scholars Foundation today announced the recipients of the 2026 Siebel Scholars award. Now in its 25th year, the Siebel Scholars program annually recognizes nearly 80 exceptional students from the world’s leading graduate schools of business, computer science, and bioengineering. The 78 distinguished students of the Class of 2026 join past Siebel Scholars classes to form an unmatched professional and personal network of more than 2,000 scholars,...

FRAMINGHAM, Mass. & SALISBURY, England--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the European Commission (EC) and Swiss Agency for Therapeutic Products, Swissmedic, have approved EKTERLY® (sebetralstat), a novel, oral plasma kallikrein inhibitor, for the symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. EKTERLY is the first and only oral on-demand treatment for HAE in the European U...

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that TRYNGOLZA® (olezarsen) has been approved in the European Union (EU) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The approval follows the positive opinion of the Committee for Medicinal Products for Human Use. The approval is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA 80 mg demon...

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel). “Today is a turning point for eligible people in Italy living with transfusion-dependent beta thalassemia and sickle cell disease, two life-shortening diseases with...

ALAMEDA, Calif.--(BUSINESS WIRE)--Infinimmune Announces Research Collaboration with Immunome...

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. today announced it has rebranded as Genetix Biotherapeutics Inc., returning to its foundational roots. Genetix, now strengthened both financially and operationally, is significantly better positioned to harness its decades of scientific innovation and extensive clinical data from hundreds of treated patients to more effectively deliver life-changing genetic therapies. The recently appointed Genetix management team, led by CEO David Meek, is...

GAITHERSBURG, Md.--(BUSINESS WIRE)--GeneDx Appoints Visionary AI and Precision Medicine Leader Dr. Thomas Fuchs to Board of Directors...