Research at Design Therapeutics Inc. has led to the discovery of conjugates consisting of a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to a protein binding moiety through oligomeric backbone linker. They are transcription modulators reported to be useful for the treatment of Huntington’s disease.
Sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers have been reported in a Humanwell Healthcare (Group) Co. Ltd. patent as potentially useful for the treatment of pain.
Skyhawk Therapeutics Inc. has obtained Australian human research ethic committee (HREC) approval to conduct a phase I trial of SKY-0515 for Huntington’s disease.
Takeda Pharmaceutical Co. Ltd. has identified N-(pyrrolidin-3-yl or piperidin-4-yl)acetamide derivatives acting as somatostatin SST4 receptor agonists and thus reported to be useful for the treatment of Alzheimer’s disease, depression, schizophrenia, autism, epilepsy, pain, bipolar and anxiety disorder.
Epoxysqualene-lanosterol cyclase (lanosterol synthase, OSLC) inhibitors have been reported in a University of Texas System patent as potentially useful for the treatment of amyotrophic lateral sclerosis, glioblastoma, multiple sclerosis, neurodegeneration, Parkinson’s, Alzheimer’s and Huntington’s diseases.
Escient Pharmaceuticals Inc. has patented quinoline derivatives acting as Mas-related G protein-coupled receptor member X2 (MRGPRX2) antagonists and thus reported to be useful for the treatment of pain, psoriasis, inflammatory disorders, atopic dermatitis, eczema, pruritus, urticaria and autoimmune disease, among others.
Royalty monetization is a financing tactic that is becoming increasingly popular during challenging times, and PTC Therapeutics Inc. is the latest firm to leverage a marketed drug to pay off debt and fuel its development pipeline. The South Plainfield, N.J.-based company agreed to sell up to $1.5 billion of its Evrysdi (risdiplam) royalty stream to Royalty Pharma plc, of New York. Evrysdi is a survival motor neuron 2 RNA splicing modifier approved by the U.S. FDA in 2020 to treat spinal muscular atrophy.
Wandercraft SAS has begun commercial operations of its exoskeleton, Atalante X, in the U.S., offering another solution to the millions of people living with mobility impairments. The company also formed a partnership with the Kessler Foundation whose research team will investigate the possible benefits of the Atalante X for neurologically impaired patients, including those recovering from stroke, spinal cord injuries and motor neuron disease.
Alexion Pharmaceuticals Inc. has identified complement C1s subcomponent inhibitors reported to be useful for the treatment of immune thrombocytopenia purpura, lupus nephritis, amyotrophic lateral sclerosis, Guillain Barré syndrome, Huntington’s disease, rheumatoid arthritis, traumatic brain injury, and autoimmune hemolytic anemia, among others.
Researchers have for the first time used human neural stem cells to print 3D brain tissues that mimic the architecture of the brain’s outer layer, the cerebral cortex. This breakthrough technique, developed by a team from the U.K.’s University of Oxford, could one day be used to provide tailored repairs to the millions of people who suffer from severe brain injuries, for which there is no effective treatment.
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