Background

Chemotherapy and existing nanoparticle‑based oncology drugs (e.g. Onivyde, Doxil) suffer from dose-limiting toxicities that lead to incomplete remission and relapse in many patients. For example, Ewing sarcoma, an adolescent and young adult cancer, becomes very refractory to existing treatments upon recurrence with over 50% mortality over 5 years. Many other solid tumor types such as liver, pancreas and brain share similar outcomes even with the latest immuno‑oncology treatments. Therefore, a great need exists to improve front line treatment efficacy to prevent relapse from occurring in the first place.

Technology Overview

CHLA in collaboration with NanoValent Pharmaceuticals discovered and patented targeted nanospheres consisting of multiple formulations of hybrid‑polymerized liposomal nanoparticles (HPLN) for oncology and non-oncological applications. HPLN payloads include chemotherapy drugs (doxorubicin, irinotecan), nucleic acids/siRNA, CRISPR‑Cas9 gene editing. Potential for combination payloads being explored. Covalently linked targeting moieties include antibodies to tumor cell surface antigens (CD99, CD19, B7H3) and peptides. Preclinical IND‑enabling studies and GMP process development is underway for the lead candidate NV103, anti‑CD99‑targeted HPLN‑encapsulated irinotecan.

Benefits

• Platform technology with broad applicability in oncology and other therapeutic areas
• Modular design to truly fulfill the promise of personalized medicine
• Improved efficacy and capability of existing agents in existing settings
• Improved applicability of existing drugs to ‘new cancers’
• Improved management of toxicity
• Crosses the Blood‑Brain barrier permitting use in CNS diseases including GBM

Applications

• Oncology, Ewing sarcoma, acute lymphocytic leukemia, liver cancer, gliomas including GBM and DIPG
• Gene editing, knockdown
• Imaging
• Vaccines

Opportunity

CHLA in partnership with NanoValent Pharmaceuticals is looking for investors and development partners to advance our lead candidate NV103 in Ewing sarcoma and other oncology indications where the CD99 cell surface antigen is highly expressed.