Researchers at Suzhou Vigonvita Life Sciences Co. Ltd. have identified N-substituted quinolinones and their prodrugs acting as 5-HT1A and dopamine D3 receptor (DRD3) agonists, 5-HT2A and dopamine D2 receptor antagonists and serotonin transporter (SERT) inhibitors.

Work at Praxis Precision Medicines Inc. has led to the identification of new compounds comprising an azaspiroheptane and acting as T-type calcium channel blockers, particularly voltage-dependent T-type calcium channel subunit α-1G (Cav3.1) blockers. As such, they are reported to be useful for the treatment of essential tremor.

Duchenne muscular dystrophy (DMD) is a disorder characterized by progressive degeneration of muscles, resulting in muscle loss, mobility limitations and lately, mortal risk. DMD is caused by mutations in the dystrophin gene (DMD) and about 80% of these are suitable for exon skipping, obtaining a functional dystrophin protein.

Researchers from Università degli Studi di Foggia presented data from a study that aimed to investigate different circulating microRNAs (miRNAs) as possible biomarkers for the diagnosis and prognosis of multiple sclerosis (MS).

Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.

Sanofi SA has prepared and tested pyrazolopyrazinone compounds acting as cystine/glutamate transporter (solute carrier family 7 member 11; SLC7A11; xCT) inhibitors.

Bloomsbury Genetic Therapies Ltd. has announced it received orphan drug designations from the FDA and the European Commission for BGT-INAD, an investigational gene therapy for the treatment of infantile neuroaxonal dystrophy (INAD).

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.