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.
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric acid (GABA), contributes significantly to the disorder.
Neurocrine Biosciences Inc. has disclosed vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of neurological and psychiatric disorders, among others.
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
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