NBSE - NeuBase Therapeutics reports encouraging data for PATrOL-enabled anti-gene in a type of muscular dystrophy

NeuBase Therapeutics (NBSE) announces positive in vitro and in vivo preclinical data for its PATrOL-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1).These new data show that PATrOL-enabled Compound A can rapidly resolve mis-splicing without negatively impacting DMPK protein levels.They also support the potential of NeuBase’s anti-gene approach to treat the underlying cause of DM1 and hypothesized mechanism of action maintaining DMPK. In vivo data highlights in the HSA transgenic mouse model of DM1 that expresses high levels of mutant CUG-repeat-containing mRNA ((HSA)) in skeletal muscle:A single intravenous injection of 29 mg/kg of Compound A traffics to the nucleus and engages HSAmRNA within 24 hours in tibialis anterior ((TA)) skeletal muscle and significantly induces broad correction of global exon inclusion levels of mis-spliced transcripts in HSA TA skeletal muscle at day 13.Statistically significant improvement in global splicing was also seen. NeuBase Therapeutics will hold a conference call today at 8:00 am ET

For further details see:

NeuBase Therapeutics reports encouraging data for PATrOL-enabled anti-gene in a type of muscular dystrophy