SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH). Atumelnant is the first and...
treatment News
ZUG, Switzerland and BOSTON, Mass. — CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, taking place October 7-10, 2025....
ZUG, Switzerland and BOSTON, Mass. — CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported new preclinical data from its novel SyNTase™ gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data are featured in an oral presentation...
SAN FRANCISCO, CA — The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to findings of a new study from Gladstone Institutes. Using a novel technique they’ve dubbed “cancer shredding,” the researchers programmed CRISPR to zero-in...
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...
BOSTON, Mass. — Crosswalk Therapeutics, Inc., a biopharmaceutical company focused on developing novel therapies for rare disorders with significant unmet medical need, today announced that it has received a research grant from the National MPS Society to support early development of a next-generation enzyme-based therapeutic approach for the treatment of Mucopolysaccharidosis...
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...
OTTAWA, Ontario – CSL Canada is proud to announce the first Canadian patient has received HEMGENIX® (etranacogene dezaparvovec), a one-time hemophilia B gene therapy, at London Health Sciences Centre (LHSC). This milestone marks a significant advancement in hemophilia B care, reflecting how clinical innovation and health system collaboration are enabling real-world...