RNA Therapeutics Inc. has received a written response from the FDA to its pre-IND application questions regarding the development of its lead asset, RNAT-89 (BLA-761423), an mRNA LNP formulation to express darbepoetin.
A recent study in PLoS One by researchers from Cincinnati Children’s Hospital Medical Center has evaluated the preclinical long-term safety of human A gamma-globin gene-carrying GbGM LV in wild-type mice.
Scientists at the Novartis Institutes for Biomedical Research (NIBR) in Cambridge have discovered a small molecule that could be used as a therapy for sickle cell disease (SCD). The molecular glue oral degraders of the WIZ transcription factor called dWIZ-1 and dWIZ-2, bind to cereblon (CRBN) and WIZ, marking it for degradation and inducing the expression of fetal hemoglobin (HbF).
To be successful, CAR T-cells need a balance between being effective and overkill. Researchers from the University of Pennsylvania and Vittoria Biotherapeutics Inc. have eliminated the CD5 signaling pathway of their CAR Ts to prevent the immunosuppressive brake effect. In return, this improved their proliferation and antitumor activity in T cell lymphomas.
Actinium Pharmaceuticals Inc. has received FDA clearance of an IND application to study Iomab-ACT for targeted conditioning prior to a bone marrow transplant (BMT) in patients with sickle cell disease.
Researchers in Japan have discovered that a phage-derived enzyme called endolysin, which targets highly resistant biofilm-forming bacteria, could help restore the gut microbiota to mitigate acute graft-vs.-host disease. Acute graft-vs.-host disease (aGVHD) is a common complication for patients who undergo allogeneic hematopoietic cell transplantation (allo-HCT). Recent studies have highlighted the significant role of the microbiome in aGVHD, with dysbiosis contributing to its pathogenesis.
Von Willebrand factor (vWF) plays a crucial role in hemostasis, and elevated levels are associated with risk of thrombosis. Gain-of-function mutations in the C-domain of vWF are linked to an increased risk of thrombosis. Previous work has shown that disrupting cysteine-rich C-domain in vWF led to reduced platelet recruitment in a shear-dependent fashion, making it a promising target for pharmacology. Apollo Therapeutics Ltd. has presented data on antibodies targeting the C-domain of vWF, including AP-21 as antithrombotics.
Beijing Gensciences Inc. has developed a FVIIIa-mimetic bispecific antibody named SS-315 for the treatment of hemophilia A. SS-315 was developed by targeting FX with its upper Fab arms and FIXa with its down-side scFv arms, respectively; the hemostatic potential of SS-315 was investigated in vitro and in vivo.
Glycoprotein VI (GPVI) is a platelet collagen receptor involved in platelet activation and an emerging target for treating thrombotic disorders such as ischemic stroke. Data have been presented by University of Würzburg scientists regarding a humanized anti-GPVI Fab antibody, EMA-601, with unprecedented potency in vitro and in vivo.
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