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.
Two days after Monte Rosa Therapeutics Inc. signed a molecular glue degrader deal with Novartis AG, two other companies, Biogen Inc. and Neomorph Inc., are moving forward in the same space in a partnership worth up to $1.45 billion. Cambridge, Mass.-based Biogen and San Diego-based Neomorph will develop molecular glue degraders (MGDs) for priority targets in Alzheimer’s, rare neurological and immunological diseases, using Neomorph’s MGD platform to identify and validate novel small-molecule protein degraders.
Sensorium Therapeutics Inc. has synthesized mesembrine derivatives acting as serotonin transporter (SERT) inhibitors reported to be useful for the treatment of anxiety, depression and stress disorders.
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