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The trial demonstrated that C3G glomerulopathy patients treated with the oral factor B inhibitor saw a significant reduction in protein in their urine. The results come days after iptacopan won the FDA’s green light for a rare blood disease.
The Indiana-based pharma shed shares after a study published Monday in JAMA showed “substantial” weight gain after patients discontinued treatment with the GLP-1 receptor agonist.
The company’s fiscal report for 2023 details revenue losses of $539 million and layoffs of 1,100 employees but notes that future GLP-1 manufacturing revenues could help stabilize its finances.
A CAR-T cell therapy for autoimmune diseases generated significant attention at the 2023 American Society of Hematology (ASH) annual meeting and Arcellx and Seagen posted promising data.
The New York-based pharma reported at ASH that odronextamab had an 80% objective response rate, with 73% of patients experiencing a complete response.
Patients treated with the investigational sickle cell therapy saw an increase in hemoglobin levels and improved red blood cell parameters.
The investigational allogeneic CAR-T therapy P-BCMA-ALLO1 appears to be more effective following strong immunosuppressive preconditioning.
These factors, along with bluebird bio’s failure to receive a priority review voucher, all hurt the company’s stock price following Lyfgenia’s approval.
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
Along with CRISPR/Cas9-based Casgevy—developed by Vertex Pharmaceuticals and CRISPR Therapeutics—the regulator on Friday approved bluebird bio’s Lyfgenia, a second gene therapy for sickle cell disease.
Another Flagship-founded company is shutting down its operations, according to an SEC filing. Axcella Health, which was focused on the development of a long COVID therapeutic, is no more.
Plexxikon, which was acquired by Daiichi Sankyo, and Novartis have agreed to settle a patent case involving the cancer drug Tafinlar and its sale in the U.S.
PRESS RELEASES
Phio’s Presentation to take place on Wednesday January 28, 2026 at 9 AM ET
Australia’s Department of Health has issued import permits for Optimi’s MDMA and psilocybin capsules for use under the Authorised Prescriber Scheme
Execution layers are required to turn AI recommendations into governed action in regulated workflows.
