Unmet Need / Invention Novelty: Current strategies to provide personalized treatment based on molecular profiles for patients suffering from MB lack efficiency and are accompanied by severe side effects and high mortality rates. There is an unmet need to understand the molecular mechanisms of group 3 MBs to drive precision medicine.
Technical Details: Researchers at Johns Hopkins and UCF have identified a novel long non-coding RNA (lncRNA) as a potential molecular marker and therapeutic target for MBs. The lncRNA, identified through interrogation of RNA-seq data from group 3 MB patients, is highly expressed in group 3 MB cell lines, patient-derived xenografts and primary MBs. Targeted depletion of the lncRNA reduces MB proliferation, induces apoptosis and results in smaller tumors both in vitro and in vivo. Administration of anti-sense oligonucleotides (ASOs) against the lncRNA result in increased survival in a group 3 MB in vivo model and presents a promising new strategy to prevent and cure the disease.
Value Proposition:
· Identification of a clinically relevant and biologically functional lncRNA as a biomarker for group 3 MBs
· Modulation via ASO administration suitable as a therapeutic strategy for MB
· Broadly useful as a novel diagnostic of curative strategy
Looking for Partners to: Develop & commercialize as a diagnostic and therapeutic target for MBs.
Stage of Development: Pre-Clinical
Data Availability: in vitro & in vivo
Inventors: Ranjan Perera & Sudipta Seal
Patent Status: PCT application pending.
Publication(s)/Related Technology: Katsushima K, Lee B, Kunhiraman H, et al. The long noncoding RNA lnc-HLX-2-7 is oncogenic in Group 3 medulloblastomas. Neuro Oncol. 2021;23(4):572-585. doi:10.1093/neuonc/noaa235
