Vyant Bio (NASDAQ:VYNT) Inc senior scientist Matthew Green delivered poster presentations this week at the Loulou Foundation’s CDKL5 forum held in Boston.
CDKL5 (cyclin-dependent kinase-like 5) deficiency disorder (CDD) is a rare neurodevelopmental condition caused by pathogenic mutations in the CDKL5 gene. Its symptoms include early-onset, difficult-to-control seizures and neurodevelopmental impairment that affects cognitive, motor, speech and visual function.
Green presented information on Vyant’s proprietary central nervous system (“CNS”) drug discovery platform, which combines human-derived organoid models of brain disease, scaled biology with machine learning to identify and validate drug targets and therapeutic candidates.
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His presentations emphasized that phenotypic and target-based screening of patient-derived CDKL5 organoids identified several promising drug targets and therapeutic candidates that reversed the abnormal neuronal hyperactivity in a CDD disease-specific manner.
Specifically, Vyant Bio (NASDAQ:VYNT) has developed a CDKL5-patient derived cortical organoid model that provides a robust disease-relevant model of CDKL5 deficiency disorder. In collaboration with Cyclica, a novel receptor was identified that showed a disease-specific deficiency in function in CDD organoids. In silico based screening identified small molecule compounds that were able to restore sensitivity of the receptor in CDD cortical organoids, the company said.
“Our presentations at the Loulou Foundation CDKL5 Forum highlighted the promise and potential of our human first CNS drug discovery platform to drive drug discovery for CNS genetic disorders,” said Robert Fremeau, Vyant's chief scientific officer.
“The key element of our platform is obtaining a deeper understanding of human disease biology at the cellular level through the application of innovative enabling technologies. We are excited by the opportunity to apply our proprietary CNS drug discovery platform to discover breakthrough therapies for patients with complex neurodevelopmental and neurodegenerative disorders.”
New Jersey-based Vyant Bio (NASDAQ:VYNT) has a platform of therapeutics seeking to treat neurodevelopmental and neurodegenerative diseases, with current programs targeting Rett syndrome, CDKL5 deficiency disorders, and Parkinson's disease.
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