Remix Therapeutics has disclosed compounds acting as RNA splicing modulators reported to be useful for the treatment of cancer and Huntington’s disease.
Researchers from Murdoch Children’s Research Institute presented data from a study that linked recessive variants in the SART3 gene with a novel neurodevelopmental syndrome.
Several Lario Therapeutics Ltd. patents describe new voltage-dependent R-type calcium channel subunit α-1E (Cav2.3) antagonists reported to be useful for the treatment of neurodegeneration, epilepsy, Parkinson’s disease, cerebral vasospasm, pain, CDKL5 deficiency, endocrine and neurodevelopmental disorders, among others.
Regulus Therapeutics Inc. has provided an update on its preclinical studies evaluating a library of oligonucleotides designed to inhibit miR-155 for the treatment of amyotrophic lateral sclerosis (ALS).
Oryzon Genomics SA has presented preclinical efficacy data on ORY-4001, a selective histone deacetylase 6 (HDAC6) inhibitor that was recently nominated as a clinical development candidate for the treatment of certain neurological diseases such as Charcot-Marie-Tooth (CMT) disease, amyotrophic lateral sclerosis and others.
The University of Sydney has patented P2X7 receptor antagonists reported to be useful for the treatment of stroke, amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer’s disease, Huntington’s disease, atherosclerosis, diabetic retinopathy and myocardial infarction, among others.
Researchers from Children’s Hospital of Philadelphia presented data from a study that linked variants in DNA methyltransferase 1-associated protein 1 (DMAP1) to a novel neurodevelopmental disorder.
Bluerock Therapeutics LP has reported the first in vitro and in vivo results on the company’s new cell therapy DA-02, consisting of human induced pluripotent stem cell (iPSC)-derived dopaminergic neurons.
At the recent International Society for Stem Cell Research meeting, Vita Therapeutics Inc. presented the first preclinical results on VTA-100, a cell therapy consisting of induced pluripotent stem cell (iPSC)-derived satellite cells, for the treatment of limb-girdle muscular dystrophy 2A/R1.
RSS
