Sangamo Therapeutics Inc. has reported data from nonhuman primate (NHP) studies showing the potential of its proprietary AAV capsid variant, STAC-BBB, as a neurotropic capsid when administered intravenously at clinically relevant doses. STAC-BBB outperformed results for other evaluated neurotropic capsid variants.
Advances in understanding the processes underlying brain neurodegeneration have allowed lots of new treatment and prevention strategies to begin to flourish. Several presentations at the 2024 Alzheimer’s & Parkinson’s Diseases Conference recently held in Lisbon reflect that eyes are now on some individuals who, despite showing pathological signs in their brains, stay cognitively healthy across several endogenous mechanisms of resilience.
Agenebio Inc. has identified benzodiazepine derivatives acting as GABA(A) receptor subunits α5β3γ2 (GABRA5) positive allosteric modulators reported to be useful for the treatment of mild cognitive impairment, amyotrophic lateral sclerosis, schizophrenia, post-traumatic stress, autism, substance abuse and dependence, Alzheimer’s disease and Parkinson’s disease, among others.
In a new study, researchers from Harvard Medical School and Regulus Therapeutics Inc. further investigated the role of miR-155 in Alzheimer's disease (AD).
The activation of the NLRP3 inflammasome exacerbates neuronal dysfunction in several neurological diseases such as Alzheimer’s and Parkinson’s diseases, as well as multiple sclerosis. Targeting NLRP3 is an approach to overcome brain inflammation, among others.
In cell and animal models of amyotrophic lateral sclerosis (ALS), the expression of toxic dipeptides in neurons led to changes in the extracellular matrix (ECM) as a protective response. The authors wrote that their findings, which appeared in Nature Neuroscience on Feb. 29, 2024, could suggest new strategies for how to approach ALS.
Researchers at the Japanese Foundation for Cancer Research and RIKEN have disclosed tankyrase 1 (TNKS1; PARP5A) and/or TNKS2 (PARP5B) inhibitors reported to be useful for the treatment of cancer, metabolic disease, cartilage injury, multiple sclerosis, pulmonary fibrosis, amyotrophic lateral sclerosis, herpes simplex virus and Epstein Barr virus infections.
The small-molecule steroid sulfatase (STS) inhibitor STX-64 (ONESTX-1, irosustat) previously showed a good safety and tolerability profile in several phase I and II clinical trials that evaluated the candidate for oncology indications.
Recent findings have suggested GAL-201 from Galimedix Therapeutics Inc. is a robust oral candidate to treat Alzheimer’s disease (AD). GAL-201 binds to the misfolded form of amyloid-β (Aβ) monomers, thus preventing its aggregation and formation of neurotoxic oligomers and protofibrils.
The third day of the AD/PD 2024 conference in Lisbon started with a plenary lecture given by Professor Howard Fillit entitled, “Translating the biology of aging into new therapeutics for Alzheimer’s disease.” Fillit, a recognized neuroscientist and geriatrician, and co-founder of the Alzheimer’s Drug Discovery Foundation (ADDF), pointed to the geroscience hypothesis which postulates that targeting aging processes may result in preventive and therapeutic options for diseases of old age, including Alzheimer’s disease (AD).
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