Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
Confo Therapeutics NV is banking an up-front payment of $40 million from a licensing deal with Eli Lilly and Co. involving its lead asset, CFTX-1554, an oral inhibitor of the angiotensin II type 2 receptor, which is in phase I development for neuropathic pain. The deal includes up to $590 million more in potential milestones and tiered sales royalties. Ghent, Belgium-based Confo could also secure a further $590 million in additional milestones should Indianapolis-based Lilly elect to take forward an antibody-based inhibitor directed against the same target.
Researchers from Georgetown University presented data from a study that aimed to assess the intrinsic mechanisms by which myeloid cells regulate their activation states during remyelination and to identify new therapeutic targets for multiple sclerosis (MS).
Indole derivative compounds acting as ferroptosis inhibitors have been reported in a Mitoimmune Therapeutics Inc. patent as potentially useful for the treatment of neurodegeneration, diabetes, stroke, myocardial infarction, liver, lung diseases, eye, renal disorders, among others.
Eurofarma Laboratórios SA and Universidade Federal do Rio de Janeiro have patented N-acylhydrazone compounds acting as sodium channel protein type 9 subunit α (SCN9A; Nav1.7) and/or SCN10A (Nav1.8) blockers which are described as being potentially useful for the treatment of pain.
After a long and bumpy road to approval, Reata Pharmaceuticals Inc. is to roll out the first treatment for Friedreich’s ataxia in the U.S. for patients ages 16 and older after the FDA gave its anti-inflammatory drug, Skyclarys (omaveloxolone), the green light.
Tevard Biosciences Inc. has entered into a 4-year global research collaboration with Vertex Pharmaceuticals Inc. aimed at creating new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, with options to expand into additional muscular dystrophies and a second indication.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have prepared and tested cyclopenta-thieno-diazepine derivatives acting as GABA-A receptor subunit α2β2γ1 positive allosteric modulators (PAMs). As such, they are reported to be useful for the treatment of neurological disorders.
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