Promoting α-synuclein (α-Syn) aggregate disassembly is one of the strategies to combat Parkinson’s disease (PD), the second most prevalent neurodegenerative disease after Alzheimer’s disease.
Recent studies have highlighted the potential role of the small GTPase Rac1 as new therapeutic target in SHH-medulloblastoma due to its regulation of Hedgehog signaling via the GLI1 and GLI2 transcription factors.
Anew Medical Inc. has announced plans to advance its Klotho gene therapy program for neurodegenerative disorders. Initial data suggest that maintaining elevated levels of Klotho in the body significantly contributes to longer, healthier life spans, while individuals with depleted or lower than normal levels of Klotho are more susceptible to neurodegenerative disorders.
The efficacy of cannabinoid CB1 receptor antagonists in diet-induced obesity and its related metabolic complications is well established, but they are tied to central nervous system (CNS)-related adverse effects due to brain penetration. There is increasing interest in developing peripherally restricted compounds for use in the clinic, but the molecular mechanisms behind the metabolic benefits from CB1 receptor antagonists is still poorly understood.
A new methodology based on the regulation of genetic enhancers has made it possible to develop a cellular map that reveals new types of helper T cells related to immunological disorders that could be explored for the development of new therapies. “I am very interested in the function of rare T cells, and I am trying to analyze their function by eliminating certain rare T cells with antibodies with ADCC [antibody-dependent cell-mediated cytotoxicity] activity or by disrupting genes that characterize rare T cells in animal models,” senior author Yasuhiro Murakawa told BioWorld.
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.
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.
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.
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.
Recursion Pharmaceuticals Inc. has synthesized molecular glue degraders acting as RBM39 degradation inducers reported to be useful for the treatment of renal cell carcinoma.
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