Researchers from Inovio Pharmaceuticals Inc. described the protective effect exerted by INO-4500, a DNA vaccine produced using the Syncon technology, against lethal Lassa virus in nonhuman primates.
Researchers from State University of New Jersey (Rutgers) presented preclinical data for PW-507, a sigma-1 receptor antagonist being evaluated for the treatment of binge eating disorder.
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
Splicebio S.L. has gained IND clearance from the FDA for its lead program SB-007, a protein splicing gene therapy to address the root genetic cause of Stargardt disease.
Gynecological cancers with amplifications in the CCNE1 gene, which encodes cyclin E1, usually exhibit resistance to standard therapies. Since cyclin-dependent kinase 2 (CDK2) is the primary partner of cyclin E, CDK2 inhibitors represent a potentially effective treatment strategy for these malignancies.
Cyrus Biotechnology Inc. has reported data on its engineered IdeS (IgG-degrading enzyme of S. pyogenes) protease for IgG-driven autoimmune disease, showing potent activity on repeat doses in a rabbit redosing model while eliciting no anti-drug antibodies.
In a presentation at the recent American Society of Hematology meeting in San Diego, Kite Pharma Inc. reported preclinical data for KITE-753, an autologous rapid manufactured anti-CD19/CD20 CAR T-cell therapy being developed for the treatment of B-cell malignancies.
Investigators at the Helmholtz Institute have shown that inceptor, an inhibitor of the insulin signaling pathway, acted by binding insulin and targeting it for degradation. “Insulin was discovered 100 years ago, and the insulin receptor was discovered 50 years ago,” Heiko Lickert told BioWorld. “Now we have a new insulin receptor, which degrades insulin.” Lickert is the senior author of the paper reporting the new insights into how inceptor works, which were published online in Nature Metabolism.
Relation Therapeutics Ltd. has delivered the first commercial validation of its combined wet lab/in silico platform for homing in on disease-related genes in the thick of the genome, signing two collaboration agreements with GSK plc, with a potential value of $200 million per target.
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