Agios Pharmaceuticals Inc. and Alnylam Pharmaceuticals Inc. have entered into an exclusive worldwide license agreement under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).
GC Biopharma Corp., formerly Green Cross Corp., said July 17 that it refiled the BLA for its intravenous immune globulin agent Alyglo (GC-5107B; IVIG-SN 10%) to the U.S. FDA – nearly a year and a half after the regulator’s initial rejection.
Scribe Therapeutics Inc. has announced an expanded collaboration with Sanofi SA, under which Sanofi receives an exclusive license to use Scribe’s CRISPR X-Editing (XE) genome editing technologies for the development of in vivo therapies, including for sickle cell disease.
GC Biopharma Corp., formerly Green Cross Corp., said July 17 that it refiled the BLA for its intravenous immune globulin agent Alyglo (GC-5107B; IVIG-SN 10%) to the U.S. FDA – nearly a year and a half after the regulator’s initial rejection.
Antibodies against β-2-glycoprotein I (β-2-GPI) play a crucial role in thrombus formation in conditions such as antiphospholipid syndrome (APS). Despite the efficacy of recombinant tissue plasminogen activator (rtPA) in patients with APS, this therapy presents significant limitations, including safety concerns.
Fulcrum Therapeutics Inc. has entered into a worldwide, exclusive license agreement with Camp4 Therapeutics Corp. to advance the discovery, development and commercialization of new therapeutic agents against an undisclosed target for the potential treatment of Diamond-Blackfan anemia (DBA).
Research led by St. Jude Children’s Research Hospital and Harvard University shows base-editing approaches could be more effective than CRISPR-Cas9 gene-editing approaches for treating conditions such as sickle cell disease and β-thalassemia. Writing in the July 3, 2023, issue of Nature Genetics, the researchers compared three base-editing approaches with two CRISPR-Cas9 approaches to increasing levels of fetal hemoglobin in CD34+ hematopoietic stem and progenitor cells, and found one of the base-editing approaches was the most potent.
Janssen Pharmaceutica NV has identified bicyclic pyridin-2-one and pyrimidin-4-one derivatives acting as coagulation factor XIa inhibitors and thus reported to be useful for the treatment of thromboembolism, diabetes, diabetic retinopathy, septic shock, hereditary angioedema, arthritis, nephropathy and inflammatory disorders, among others.
Becoming the first gene therapy approved for hemophilia A, Roctavian (valoctocogene roxaparvovec) finally received the U.S. FDA’s blessing on June 29, after developer Biomarin Pharmaceutical Inc. spent nearly three years working to address issues raised in a 2020 complete response letter. The approval came a day prior to the June 30 PDUFA date.
A single low-dose injection with anti-DLL4 in a nonhuman primate model of acute graft-vs.-host disease (aGVHD) dramatically improved post-transplant survival, providing durable protection from otherwise lethal gastrointestinal GVHD, researchers reported in the June 28, 2023, issue of Science Translational Medicine. Blocking DLL4 specifically increased the migration of beneficial regulatory T cells into the intestines, with concomitant reduction in effector T cells, which are the main culprits in aGVHD. Ultimately, these activities effectively provided protection against T-cell-mediated damage in a nonhuman macaque primate model.
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