Asteria, Targeted gene therapy platform: tackling
the root of blood cancer.
Antigonie Biosciences has engineered a targeted gene therapy platform to create next-generation anti-cancer RNA therapeutics for blood cancers. We are moving hemato-oncology from lifelong management to potentially curative strategies through the creation of therapeutics that can eliminate the root of blood cancers: malignant HSPCs.
In contrast to systemic chemotherapy regimens that treat the consequence of blood cancer - an overproduction of mature blood cells - but not the cause, our RNA therapeutics deliver to malignant HSPCs in the bone marrow.
The platform delivers mutation-specific RNAi designed with high specificity to silence the mutant genes (e.g., JAK2V617F) that cause hematologic malignancies in HSPCs. Our therapeutics “shoot the messenger” by eliminating mutant messenger RNA (mRNA). Their specificity means they completely spare the healthy gene mRNA, minimizing off-target effects in healthy cells.
By successfully delivering a selective killing agent to the root disease-initiating cells (HSPCs), our platform triggers mutation-specific cell death. Our therapeutics induce the functional depletion and eradication of the malignant HSPC clone.
Our lead candidate, W-0301, targeting JAK2V617F Myeloproliferative Neoplasms (MPNs), is successful proof-of-concept for our platform technology.
Core validation data
Single IV injection achieved cargo delivery to up to 20% LTHSCs/MPPs in the bone marrow.
In vivo, treatment led to a dramatic reduction in disease-driving erythroid progenitor cells, reversing the MPN phenotype.
Demonstrated allele-specific apoptosis of JAK2V617F cells in patient-derived samples.
W-0301's strong preclinical profile, recently highlighted in a major scientific abstract, validates our ability to safely and effectively deliver genetic payloads to the challenging bone marrow environment. This success accelerates the development of our entire pipeline.
The validated LNP platform allows us to rapidly target other prevalent somatic mutations and oncogenic drivers across the spectrum of hematologic malignancies.
Status: Preclinical/IND-enabling
Status: Preclinical/IND-enabling
Status: Lead optimization
