Fanconi anemia (FA) is a rare genetic disorder characterized by defective DNA repair, leading to hematopoietic stem cell (HSC) dysfunction and bone marrow failure. Replication stress, which compromises HSC maintenance and regeneration, is one of the central underlying mechanisms in FA.
At the recent annual congress of the European Association for Haemophilia and Allied Disorders, researchers from Novalgen Ltd. presented the discovery and preclinical characterization NVG-444, a potentially first-in-class next-generation FVIII mimetic antibody equipped with autoregulation to reduce prothrombotic risks that is being developed for the treatment of hemophilia A.
Researchers at E-Therapeutics plc recently presented efficacy and safety data on ETX-148, a pan-hemophilia agent in murine models of hemophilia A and B.
Immvention Therapeutix Inc. has entered into a collaboration and license agreement with Novo Nordisk A/S to co-develop oral therapies for sickle cell disease and other chronic conditions.
X-linked sideroblastic anemia (XLSA) is a rare genetic disorder caused by mutations in the ALAS2 gene, which plays a critical role in heme biosynthesis within red blood cells.
Yoltech Therapeutics Co. Ltd. has advanced YOLT-204 into the clinic for the treatment of transfusion-dependent β-thalassemia (TDT). If successful, YOLT-204 may provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and hematopoietic stem cell transplantation.
The accelerating pace of U.S. FDA approvals for cell and gene therapies is “great for the field and great news for the patients,” but questions remain over commercialization, with “costs remaining stubbornly high.” That was the glass half-full summary of Tim Hunt, president of the industry group, the Alliance for Regenerative Medicine, reprising progress in 2024, and looking forward to the prospects for further growth and the potential impact of the incoming Trump administration in 2025.
China’s health regulator gave conditional approval to Platinum Life Excellence Biotech Co. Ltd.’s amimestrocel injection (hUC-MSC PLEB-001, Ruibosheng) as the nation’s first human umbilical cord-derived mesenchymal stem cell therapy to treat steroid-refractory acute graft-vs.-host disease on Jan. 2, 2025.
About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
RSS
