Bioage Labs Inc. is advancing its apelin receptor (APJ) agonist programs for obesity and other indications, and has entered into an option agreement with Jikang Therapeutics to license a novel APJ agonist nanobody.
Regeneron Pharmaceuticals Inc. is adding to its obesity pipeline by in-licensing Hansoh Biomedical Co. Ltd.’s phase III dual glucagon-like peptide 1 (GLP-1)/gastric inhibitory polypeptide (GIP) receptor agonist, HS-20094, for up to $2 billion.
Researchers at Chongqing Pharmaceutical Research Institute Co. Ltd. and Yaopharma Co. Ltd. have identified glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of obesity, type 2 diabetes and nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
Kexing Biopharm Co. Ltd. has announced IND clearances this month by China’s National Medical Products Administration (NMPA) and the U.S. FDA for GB-18, a biologic product for the treatment of cancer cachexia.
Rocket Pharmaceuticals Inc. CEO Gaurav Shah said his firm is investigating how its gene therapy for Danon disease may have created an “unexpected and paradoxical” effect that led to problems for a phase II patient who ultimately died.
Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.
The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.