Researchers from Sapienza University of Rome...

Sickle cell disease (SCD) is autosomal recessive disorder caused by mutations in the β-globin gene, and induction of fetal γ-globin is considered an established therapeutic strategy for the treatment of this disease. A research team led by scientists at Kyorin Pharmaceutical Co. Ltd. has discovered RK-701, a small-molecule inhibitor of G9a and G9a-like protein (GLP) as a potential therapeutic agent for SCD.

Evidence has suggested free fatty acid receptor 1 (FFAR1), also known as GPR40, as a promising therapeutic target in the treatment of type 2 diabetes, since its activation in pancreatic β cells promotes insulin secretion in a glucose concentration-dependent manner.

Researchers from Taipei Medical University presented the discovery of novel store-operated calcium channel (SOC) inhibitors as potential anticancer candidates. Synthesis and optimization of a series of difluorobenzamide derivatives led to the discovery of MPT-0M004 as the lead candidate with promising SOC inhibitory activity. In vitro, both migration and invasion of colorectal cancer cells were significantly suppressed after 48 hours of treatment with MPT-0M004, with the growth inhibitory effect of the compound being similar to that seen for the reference SOC inhibitor.

A research team based at Hangzhou Healzen Therapeutics Co. Ltd. presented preclinical data for the novel STAT3 selective degrader HZ-R061.

Researchers from Hangzhou Healzen Therapeutics Co. Ltd. and Biopolar Hongye (Nantong) Pharmaceutical Co. Ltd. presented the discovery and preclinical characterization of a next-generation Bcl-2 inhibitor, HZ-L105, being developed for the treatment of hematological cancers.

To date, there are no current orally available compounds that promote bone formation for treating osteoporosis; most treatments act by inhibiting osteoclastic bone resorption, leading to increased bone mineral density and reduced hip fracture rate in a modest way. Analogues of parathyroid hormone (PTH) are the most frequently used bone anabolic agents, which even though effective, they require daily injections.

Type I JAK2 inhibitors improve symptoms and outcomes of patients with myeloproliferative neoplasms (MPNs), but mutant allele JAK2 VF remains unchanged with this therapy. Type II JAK2 inhibitors bind the inactive conformation of the kinase domain and reduce the fraction of JAK2 VF mutant allele in vivo, suggesting an improved approach for JAK2 inhibition. Ajax Pharmaceuticals Inc. presented preclinical data on the type II JAK2 inhibitor AJ1-10502 for the treatment of MPNs.