Daiichi Sankyo Co. Ltd. has disclosed new proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a steroidogenic factor 1 (SF-1)-targeting moiety. They are reported to be potentially useful for the treatment of primary aldosteronism, Cushing syndrome and cancer.
Hangzhou Innogate Pharma Co. Ltd. and Innorace Biopharma Co. Ltd. have synthesized new peroxisome proliferator activated receptor γ (PPARγ) inverse agonists reported to be useful for the treatment of cancer.
Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. has patented new probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) inhibitors reported to be useful for the treatment of cancer.
Neuroinflammation has arisen as a key factor in the pathophysiology of Alzheimer’s disease (AD). Chronic immune activation in the brain leads to the release of pro-inflammatory cytokines and other inflammatory mediators that contribute to neuronal damage, thus impacting cognitive function during the progression of the disease. Transcriptomic and epigenomic analyses were performed to understand the epigenetic mechanisms behind the expression of inflammatory genes in AD brain.
Researchers from Arthex Biotech SL, the University of Valencia (Spain) and collaborators sought to address the lack of optimized microRNA (miRNA)-targeting antisense oligonucleotides (anti-miRs) for upregulating MBNL1 in myotonic dystrophy.
Tessera Therapeutics Inc. has received a grant from the Gates Foundation to support early-stage research exploring multiple genetic approaches aimed at developing a scalable cure for HIV. This research will evaluate several potential strategies leveraging Tessera’s Gene Writing platform to engineer immune cells in vivo.
Medullary thyroid carcinoma (MTC) is a rare, often aggressive, neuroendocrine thyroid tumor with limited targeted treatment options, arising from calcitonin-producing C cells and sometimes associated with RET mutations. Researchers from the Mayo Clinic reported the potential of targeting DLL3 as a therapeutic strategy in MTC.
A new Danish research project focused on Parkinson’s disease has received funding from Innovation Fund Denmark. The DESYNA (Degradation of Extracellular α-SYNuclein Aggregates) project aims to develop a new therapy targeting α-synuclein, the accumulation of which is a key driver of Parkinson’s disease.
The aberrant activation of NACHT, LRR and PYD domains-containing protein 3 (NLRP3) inflammasome is a known crucial driver of neuroinflammation in Alzheimer’s disease (AD) and other related dementias. The inhibition of NLRP3 has shown benefits in preclinical models. Researchers from the University of Texas recently presented data regarding an NLRP3 inhibitor, AMS-17, for the treatment of AD and related dementias.
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