NOD2 deficiency is involved in the pathogenesis of Crohn’s disease (CD) due to failure of gut innate immunity and loss of intestinal tissue homeostasis. Orchard Therapeutics Ltd. has presented preclinical data on OTL-104, human hematopoietic stem cell (HSC) gene therapy for the treatment of NOD2-deficient CD.
Researchers at Indian Institute of Technology (BHU) Varanasi and collaborators have designed inhibitors of cholinesterase based on benzothiazole-phenylpiperizine as a potential treatment for Alzheimer’s disease.
Amphista Therapeutics Ltd. has nominated AMX-883, a selective and orally bioavailable degrader of BRD9, as its first clinical development candidate. AMX-883 is being advanced for the treatment of acute myeloid leukemia (AML), with an IND application planned for early next year.
A recent study published in PLOS Genetics has identified several novel genetic variants associated with the risk of orofacial clefts, a common and complex congenital disability. Orofacial clefts are caused by a combination of genetic and environmental factors and can occur in isolation or simultaneously with other birth defects.
Tumors suffer metabolic stress, such as oxygen and nutrient deficiency; as a result, altered metabolism is a common feature of tumors. Cancer cells enhance the production of energy and the synthesis of macromolecules to grow at pathologically increased rates. It is crucial to identify genes that modulate cellular fitness under these stressful scenarios.
Littdd Medicines Ltd. has described mTOR complex 1 (mTORC1) and/or mTOR complex 2 (mTORC2) inhibitors reported to be useful for the treatment of cancer, idiopathic pulmonary fibrosis, transplant rejection, autoimmune and metabolic diseases, Huntington’s disease, Parkinson’s disease and Alzheimer’s disease, among others.
Researchers at Shanghai Jingxin Biopharmaceutical Co. Ltd. and Zhejiang Jingxin Pharmaceutical Co. Ltd. have divulged compounds acting as leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
Aurigene Oncology Ltd. has identified proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase protein binding moiety covalently linked to probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) and/or transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β) binding moieties through a linker reported to be useful for the treatment of cancer.
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