{"content":[{"text":"data for a new oncology program against historically ‘undruggable’ KRAS driver mutations. \n With this tremendous momentum building across our expanding development pipeline, we \n look forward to entering the clinic with our DM1 program in 2022 and continuing to evaluate \n diseases where our PATrOL platform can deliver significant therapeutic value.”","bbox":{"left":0.098039,"top":0.06867,"width":0.804238,"height":0.07090400000000001}},{"text":"DM1 Program Data Highlights \n Experiments conducted in the HSALR transgenic mouse model of DM1, which express high \n levels of mutant CUG-repeat-containing mRNA in skeletal muscle, demonstrated molecular \n rescue in vivo after a single intravenous (IV) dose (29 mg/kg), which resulted in:","bbox":{"left":0.098039,"top":0.155791,"width":0.804467,"height":0.07374499999999998}},{"text":"~75% splice correction of mis-spliced transcripts observed at ~2 weeks (p=<0.0001); \n Complete splice correction of chloride channel (Clcn1) in skeletal muscle, a transcript \n responsible for the myotonia phenotype; \n Restoration of Clcn1 protein in skeletal muscle; \n Reduction of nuclear inclusions at day 21; and \n Good tolerability at pharmacologically active doses.","bbox":{"left":0.147059,"top":0.245753,"width":0.754778,"height":0.10689000000000004}},{"text":"Additional experiments using PATrOL-enabled compounds to treat DM1 showed a 70% \n reduction in myotonia 35 days post first-dose via plantar flexor torque assay (p=<0.05) in \n vivo after subcutaneous (SC) dosing (3 mg/kg dosed weekly x4). This functional rescue via \n SC administration was achieved at a significantly lower dose relative to the IV route.","bbox":{"left":0.098039,"top":0.368859,"width":0.803779,"height":0.07090500000000005}},{"text":"Dr. Stephan stated, “We’ve delivered on several key advancements in our development \n program of PATrOL-enabled compounds to treat myotonic dystrophy type 1. These data \n show that the platform is capable of addressing the underlying genetic causes of DM1 after \n systemic dosing. In addition, these data demonstrate the ability of our candidates to achieve \n these effects using multiple systemic routes, including subcutaneous administration. We are \n continuing to advance our DM1 program and anticipate entering the clinic in calendar year \n 2022.”","bbox":{"left":0.098039,"top":0.455981,"width":0.804478,"height":0.12488099999999996}},{"text":"HD Program Data Highlights \n Experiments conducted in the zQ175 Huntington’s disease mouse model with 190 CAG \n repeats in exon 1 of HTT demonstrated that SC administration of PATrOL-enabled \n compounds:","bbox":{"left":0.098039,"top":0.597079,"width":0.803318,"height":0.07090399999999997}},{"text":"Crossed the blood-brain-barrier and reached target brain regions; and \n Selectively reduced mutant HTT protein (mHTT).","bbox":{"left":0.147059,"top":0.6842,"width":0.604425,"height":0.03491999999999995}},{"text":"These results were achieved with PATrOL-enabled compounds designed to target the \n mutation at either the DNA or RNA level, and the compounds were generally well tolerated.","bbox":{"left":0.098039,"top":0.735337,"width":0.800197,"height":0.03491900000000003}},{"text":"Robert Friedlander, M.D., M.A., Chairman of NeuBase’s Scientific Advisory Board and \n Chairman and Professor of Neurosurgery at the University of Pittsburgh and UPMC, said, \n “NeuBase’s Huntington’s disease development program has made significant progress \n toward our target profile. We have advanced from showing mutant HTT protein knockdown in \n human patient cells to in vivo proof-of-concept after a patient-friendly subcutaneous \n administration. We achieved this reduction in mHTT by targeting the mutation at either the \n RNA or DNA level, which is one of the unique attributes of the PATrOL platform. In addition, \n we are focusing our efforts on systemic dosing routes, which offers the potential for uniform \n brain distribution, while also being a preferable route compared to intrathecal","bbox":{"left":0.098039,"top":0.786473,"width":0.8058080000000001,"height":0.1608670000000001}}],"images_bbox":[],"images_bbox_not_text_overlap":[]}