Researchers from the University of Texas Medical Branch have developed a novel tau immunotherapy delivered via intranasal route, able to enter the brain, and recognize and successfully clear tau aggregates in mouse models of tauopathy. Aberrant tau aggregates cause neurodegenerative symptoms in Alzheimer’s disease (AD), progressive supranuclear palsy (PSP) and dementia with Lewy bodies (DLB). Although these conditions present phenotypic differences, the fact of sharing tau deposits as a major hallmark tags them as tauopathies. Read More

Therapeutics for peripheral neuropathies, such as Charcot-Marie-Tooth (CMT) disease, are still quite limited and focus on managing symptoms. Augustine Therapeutics NV has presented an orally available histone deacetylase 6 (HDAC6) inhibitor, AGT-100216, for the treatment of CMT. AGT-100216 was designed to work through improving axonal regeneration and nerve functional restoration. Read More

Researchers from Oricell Therapeutics (Shanghai) Co. Ltd. presented the discovery and preclinical characterization of ORIC-613, a dual-targeting CAR T-cell therapy being developed for the treatment of pancreatic and gastric cancer. Read More

Beijing DP Technology Co. Ltd. has nominated DPT-0415, a novel small molecule targeting lipoprotein‐associated phospholipase A2 (Lp-PLA2), as a preclinical candidate for the treatment of diabetic retinopathy (DR) and diabetic macular edema (DME). Read More

Immvention Therapeutix Inc. has disclosed benzimidazole-containing compounds acting as inflammasome inhibitors reported to be useful for the treatment of inflammatory disorders. Read More

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. Read More

Targeting human glutaminyl-peptide cyclotransferase (QPCT) has emerged as a potential strategy in Alzheimer’s disease (AD). As the main substrates of this enzyme are involved in biological processes associated with neurodegenerative diseases, Parkinson’s disease (PD) and others may be other options to explore as well. Read More

Recursion Pharmaceuticals Inc. has synthesized molecular glue degraders acting as RBM39 degradation inducers reported to be useful for the treatment of renal cell carcinoma. Read More

UCB SA has identified imidazotriazine derivatives acting as interleukin-17 (IL-17) modulators reported to be useful for the treatment of inflammation and autoimmune diseases. Read More

Myricx Bio (Myricx Pharma Ltd.), a spin out from Imperial College London and the Francis Crick Institute, has closed a series A financing raising £90 million ($114 million) to support its work focused on the discovery and development of a novel class of payloads for antibody-drug conjugates (ADCs). Read More

Denali Therapeutics Inc. has divulged NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of glaucoma, spinal cord Injury, multiple sclerosis, Niemann-Pick disease, stroke, Alzheimer’s disease, amyotrophic lateral sclerosis and diabetic neuropathy, among others. Read More

Hansoh Bio LLC has described MAP kinase-activated protein kinase 2 (MAPKAPK2; MK2) inhibitors reported to be useful for the treatment of cancer, infections, neurodegeneration, autoimmune disease, immunological disorders, inflammatory conditions and cardiovascular disorders. Read More

Large-scale human genetic studies have revealed that loss-of-function INHBE variants are associated with a lower abdominal to gluteofemoral fat ratio, improved metabolic profile, and reduced fasting glucose levels. Read More