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.
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.
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.
NRG Therapeutics Ltd., has nominated NRG-5051 as its first development candidate, and secured a $5 million grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support its preclinical development of as a disease-modifying treatment for Parkinson’s disease.
The FDA has granted orphan drug designation to Capsida Biotherapeutics Inc.’s CAP-002, an investigational gene therapy for the treatment of developmental and epileptic encephalopathy due to syntaxin-binding protein 1 (STXBP1) mutations.
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