Be Biopharma Inc. has announced the FDA’s clearance of its IND for BE-101, an autologous potentially first-in-class B-cell medicine in development for the treatment of hemophilia B.
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
Geneventiv Therapeutics Inc. has been awarded a Direct to Phase II Small Business Innovation Research (SBIR) grant for approximately $2.5 million from the National Heart Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH) to support development of a universal gene therapy for hemophilia A or B with or without inhibitors.
Centessa Pharmaceuticals plc’s launch of a phase I/IIa trial with LB-101, a PD-L1xCD47 candidate that emerged from the Lockbody platform, highlighted the firm’s efforts against solid tumors. But the diversified company has more in the works, including a registrational study with subcutaneous (subcu) Serpinpc, designed to treat hemophilia (hemo) B. Centessa has a program in narcolepsy, too.
Scientists based at Beijing Institute of Radiation Medicine have reported preclinical evaluation of a novel recombinant activated human factor VII (rFVIIa), GEN-0828, being developed as potential candidate for the treatment of hemophilia and trauma hemorrhage. GEN-0828 is rFVIIa that was mainly produced from Chinese hamster ovary (CHO) cells.
Little more than a month after the U.S. FDA approved the first gene therapy for adults with hemophilia B, Uniqure NV’s Hemgenix, strong phase III data have come from Pfizer Inc. The Pfizer results show fidanacogene elaparvovec, a vector containing an AAV capsid and a high-activity human coagulation factor IX (FIX) gene for treating adult men with moderately severe to severe hemophilia B, hit the primary endpoint in the phase III Benegene-2 study. The one-time therapy is designed to allow those living with hemophilia B to be able to produce FIX instead of receiving regular, ongoing doses of exogenous FIX.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
China’s NMPA has given Belief Biomed Inc. the official go-ahead to start testing its investigational gene therapy, BBM-H901, for the potential treatment of hemophilia B in the country, marking the first time an I.V. gene therapy for a rare disease has been approved in China. The company plans to advance the phase I/II trial for the candidate shortly, it said.
