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TUCSON, Ariz. — Critical Path Institute (C-Path) is excited to announce the launch of the Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-1) Consortium. The CPA-1 consortium aims to accelerate drug development for alpha-1 antitrypsin deficiency (AATD), a rare disease that affects individuals and families worldwide. This will be achieved by...
CAMBRIDGE, Mass. — Crossbow Therapeutics, Inc., a biotechnology company focused on advancing T-Bolt™ therapies, a novel class of antibody therapeutics that mimic T-cell receptors (TCR-mimetics), today announced the nomination of its first development candidate, CBX-250, a first-in-class, potent, and specific T-cell engager (TCE) for the treatment of myeloid leukemia. Researchers from...
MARBURG, Germany — Global biotechnology leader CSL Behring (ASX: CSL) today announced that two hemophilia B patients were treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France. This milestone achievement makes HEMGENIX® the first gene therapy administered as a treatment in a real-world setting for...
King of Prussia, Pennsylvania – CSL Behring has released promising 3-year results from the pivotal HOPE-B study, confirming the sustained long-term durability and safety of etranacogene dezaparvovec-drlb (Hemgenix®) in individuals with hemophilia B. The data, demonstrating prolonged elevated factor IX activity levels following a single infusion, were recently presented at...
KING OF PRUSSIA, Pa. — CSL Behring, a business unit of global biotechnology leader CSL, today announced the availability of 4- and 5-gram vial sizes for ZEMAIRA® [Alpha1-Proteinase Inhibitor (Human)]. Previously available only in a 1-gram vial, the 4- and 5-gram packaging is significant for the Alpha-1 community as it...
  European Commission converts conditional approval of FILSPARI (sparsentan) into standard marketing authorization for the treatment of IgA Nephropathy (IgAN) Decision follows positive recommendation from Committee for Medicinal Products for Human Use (CHMP) from February 2025 EU approval is based on the complete data set from the phase-III PROTECT study ST. GALLEN, Switzerland and SAN...
ST. GALLEN, Switzerland and SAN DIEGO, Calif. — CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) today announced that the European Commission has granted conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio...