One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier.
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.
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.
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.
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.
Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons.
Sironax has described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Parkinson’s disease, multiple sclerosis, traumatic brain injury, diabetic neuropathy and chemotherapy-induced peripheral neuropathy.
Merck Sharp & Dohme Corp. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis and inflammation.
Diamond Therapeutics Inc. has identified deuterated lisuride analogues acting as 5-HT2A receptor agonists reported to be useful for the treatment of neurological disorders.