Friedreich’s ataxia (FA), the most common form of hereditary ataxia, is an autosomal recessive neurodegenerative disorder affecting multiple organ systems, and causing cardiomyopathy, scoliosis, muscle weakness, speech impairment and other systemic issues.
The farnesoid X receptor (FXR) is a nuclear receptor predominantly expressed in the liver, intestine and kidney. FXR is crucially involved in regulating bile acid homeostasis, controlling inflammatory responses in the liver, and regulating lipid and glucose metabolism. Therefore, FXR plays a role in regulating metabolic dysfunction-associated steatohepatitis (MASH) and has been proposed as a promising target for MASH drug development.
Triple-negative breast cancer (TNBC) cells depend on the transcriptional kinases CDK12 and CDK13 to maintain DNA damage response gene expression and manage replication stress. Due to their functional overlap, inhibition of a single kinase may permit compensatory activity.
At the recent International Stroke Conference, researchers from VST Bio Corp. and Yale University presented preclinical data regarding VB-001, a monoclonal antibody that targets syndecan 2 (SDC2) and is also known as VST-002.
Hidradenitis suppurativa (HS) is a chronic inflammatory skin disease with strong association with psoriasis and inflammatory bowel disease (IBD). While some signaling pathways are well defined in HS, the role of TNF-like ligand 1 (TL1A) is not well understood. A group of researchers has investigated the association of TL1A with HS, as well as its association with other cytokine networks.
Vidac Pharma Holding plc has announced the initiation of an in vivo preclinical program to evaluate the potential of its hexokinase 2 (HK2)/voltage-dependent anion channel (VDAC)-targeting approach in psoriasis.
Dewpoint Therapeutics Inc. has selected a development candidate in its MYC program. The first-in-class small molecule is designed to disrupt MYC-driven oncogenic transcription by modulating the aberrant biomolecular condensates that organize transcriptional control in cancer cells.
Tau is an intrinsically disordered protein that regulates the stability and dynamics of microtubules in physiological conditions. Recent work has revealed the involvement of tau in various neuronal processes. Researchers from the University of California and collaborators aimed to systematically investigate the cellular factors that control the accumulation of tau aggregates in human neurons.
The malfunction of lymphatic drainage that occurs in lymphedema leads to excessive cholesterol accumulation in the affected skin and lymphatic vessels, causing inflammation and fibrosis. However, surgery and the chemical reduction of accumulated cholesterol with cyclodextrin reduce inflammation and regenerate lymphatic vessels. Cholesterol is a potential therapeutic target for treating lymphedema, according to this study published on Feb. 11, 2026, in Nature by Veronique Angeli and her colleagues.
Aquinnah Pharmaceuticals Inc. has disclosed new microtubule-associated protein τ (PHF-τ; MAPT) aggregation inhibitors designed for use in the treatment of Alzheimer’s disease and frontotemporal dementia.
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