Lysoway Therapeutics Inc. has synthesized mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of age-related macular degeneration, aging, Alzheimer’s disease, cancer, Charcot-Marie-Tooth disease, cystic fibrosis, muscular dystrophy and type C Niemann-Pick disease.
As an array of developers push their Friedreich’s ataxia (FA) drugs along, PTC Therapeutics Inc.’s recent update on its program piqued thirst for the 15-lipoxygenase inhibitor vatiquinone. Warren, N.J.-based PTC said the prespecified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.
Belief Biomed Inc.’s gene therapy drug BBM-D101 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD).
Reducing microglial activity in the presence of apolipoprotein E4 (APOE4) has uncovered a mechanism associated with the deposition of misfolded amyloid and tau in a novel mouse model of Alzheimer’s disease. By transplanting human neurons into the mouse brain and eliminating the mouse microglia, scientists at the Gladstone Institutes in San Francisco observed that amyloid and tau deposition was reduced. These results support therapeutic strategies that target APOE4 and microglia.
Neushen Therapeutics Inc. has synthesized 5-HT2A receptor agonists reported to be useful for the treatment of depression, schizophrenia, anxiety and post-traumatic stress disorder.
Data from two Alzheimer’s tests being developed by Roche Holding AG showed high accuracy in detecting the disease in patients being tested for the condition. The tests measure biomarkers in the blood linked to Alzheimer’s and will help provide desperately needed information by letting patients know whether they have the disease or not, Margherita Carboni, Neurology Indication Lead at Roche Diagnostics told BioWorld.
Damona Pharmaceuticals Inc. has obtained IND clearance from the FDA for DPX-101 for the treatment of cognitive deficits in brain disorders, including major depressive disorder.
Mitochondria In Motion Inc. has identified new N-(trans-4-hydroxycyclohexyl)-6-phenylhexanamide derivatives acting as mitofusin activators and reported to be useful for the treatment of Alzheimer’s, Parkinson’s and Huntington’s disease, cancer, amyotrophic lateral sclerosis, stroke, mitochondrial myopathy and Charcot-Marie-Tooth disease, among others.
Regeneron Pharmaceuticals Inc. has patented rapamycin analogues acting as peptidyl-prolyl cis-trans isomerase FKBP1A (FKBP12; Rotamase) ligands reported to be useful for the treatment of Alzheimer’s disease, graft-vs.-host disease, cancer, sarcopenia and more.
Inbrain Neuroelectronics SL closed a $50 million series B financing round to advance clinical trials for its graphene-based brain-computer interface therapeutics platform. The company also secured additional funding from Merck KGaA GmbH which will go towards developing the technology for application across both central and peripheral nervous systems.
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