H. Lundbeck A/S and Vernalis (R&D) Ltd. have jointly patented leucine-rich repeat kinase 2 (LRRK2; Dardarin) and/or LRRK2 mutant inhibitors reported to be useful for the treatment of Lewy body dementia, multiple system atrophy and Parkinson’s disease.
A Voronoi Bio Inc. and Voronoi Inc. patent describes platelet-derived growth factor receptor (PDGFR), mast/stem cell growth factor receptor kit (KIT; c-Kit; CD117) and their mutant inhibitors reported to be useful for the treatment of cancer, fibrosis, neurological diseases, atherosclerosis, and pulmonary hypertension.
Research at Sensorium Therapeutics Inc. has led to the identification of deuterated alkaloids acting as serotonin transporter (SERT) inhibitors reported to be useful for the treatment of asthma, chronic obstructive pulmonary disease, depression, rheumatoid arthritis, stress and anxiety disorders.
Polydeuterated analogues of ambroxol and bromhexine have been reported in a Zywie LLC patent as potentially useful for the treatment of aging, Parkinson’s and Alzheimer’s diseases, dementia with Lewy bodies and frontotemporal dementia.
Researchers from City of Hope discussed preclinical data for CD33-targeted chimeric antigen receptor (CAR) T cells being developed for the treatment of acute myeloid leukemia (AML).
Friedreich’s ataxia (FA) is an autosomal recessive disorder caused by mutations in the FXN gene and characterized by cardiomyopathy, gait ataxia and sensory loss, with cardiac complications the main cause of death among patients with FA.
Homology Medicines Inc. has reported data on an adeno-associated viral (AAV) vector-based therapy, HMI-104, an AAV treatment intended to induce hepatic expression of a complement C5 monoclonal antibody, named as C5mAb, for the potential treatment of paroxysmal nocturnal hemoglobinuria (PNH) as well as other complement-driven pathologies. C5mAb is thought to bind to C5 and inhibit the C5-mediated hemolysis observed in PNH.
Arrhythmogenic cardiomyopathy (ACM) is a devastating inherited disorder characterized by massive cardiomyocyte loss, fibrofatty infiltration and ventricular arrhythmias, among others. Most known genetic causes of ACM involve the gene PKP2, which encodes plakophilin-2. An unmet medical need exists regarding therapies that correct this PKP2 deficiency.
Trigeminal neuralgia (TN) is a chronic disorder caused by the hyperactive functioning of a damaged trigeminal nerve that provokes severe facial pain coming from the trigeminal nerve.
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