Drug intelligence / Profile preview

BBB-002

Development stage
Preclinical
Lead developer
ELVESCA Biotherapeutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody-Based Therapeutics, Nanoparticles → Drug Delivery Systems
01

Overview

BBB-002 is a preclinical-stage siRNA-based therapeutic being developed by ELVESCA Biotherapeutics for the treatment of Ewing Sarcoma. The drug utilizes the proprietary ELART technology platform, which consists of an siRNA payload encapsulated within a nanocarrier system functionalized with an anti-IGF1R antibody. This antibody acts as a targeting selector by binding to the insulin-like growth factor 1 receptor (IGF1R), which is highly expressed on Ewing Sarcoma cells, ensuring the selective delivery of the siRNA to tumor tissue. Inside the target cells, the siRNA silences the expression of the EWS-FLI1 fusion oncogene, the pathognomonic driver of Ewing Sarcoma. This precision medicine approach aims to provide a targeted therapy for pediatric and young adult patients suffering from this aggressive cancer.

02

Targets

EWS-FLI1 (Ewing sarcoma breakpoint region 1–Friend leukemia virus integration 1 fusion protein)

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