Geron Corp., a company founded more than three decades ago, is finally celebrating its first U.S. FDA approval. The agency cleared imetelstat, branded Rytelo, for use in transfusion-dependent anemia in adults with low- to intermediate-risk myelodysplastic syndromes, specifically those requiring four or more red blood cell units over eight weeks who have failed or no longer respond to erythropoiesis-stimulating agents (ESA) or who are not eligible for ESA treatment.
The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.
While Servier Pharmaceuticals LLC awaits this summer’s PDUFA date for the IDH-mutant glioma drug vorasidenib, acquired in the buyout of oncology assets from Agios Pharmaceuticals Inc., the latter unveiled positive data from a global phase III study with oral mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.
Interim data from Biohaven Ltd.’s phase I study of BHV-1300, an IgG degrader using an ASGRP-bispecific, produced dose-dependent results in its first four cohorts, though not enough to satisfy investors.
A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.
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.
Researchers from Poseida Therapeutics Inc. presented preclinical data for P-FVIII-101, a novel nonviral gene therapy being developed for the treatment of hemophilia A.
Investigators at Poseida Therapeutics Inc. developed P-KLKB1-101, a nonviral KLKB1 gene editing therapy, being developed for the treatment of hereditary angioedema (HAE).
Kalvista Pharmaceuticals Ltd. has disclosed coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of hereditary angioedema and thrombotic disorder.
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.