Our proprietary platform consists of dual control achieved at the transcriptional and post-transcriptional level. These ensure therapeutics are released within the tumor. A Tie-2 promoter ensures the transgene transcription occurs in TEMs, while miRNA-126 target sequences inhibit expression of the transgene in HSPCs.
We adapted an autologous ex-vivo gene therapy method to load the patient’s own TEMs precursors, that are hematopoietic stem and progenitor cells (HSPCs), with an immunotherapeutic transgene sequence. By re-introducing gene-modified HSPCs into the patient, we seek to take advantage of the self-renewing and multi-differentiation capability of HSPCs to enable durable and potentially long-term effects following a single treatment.
We have developed proprietary methods for expressing immunotherapeutic payloads in a highly controlled and specific manner within tumors. IFN-α is a well-known therapeutic that was previously administered systemically for treatment of various cancers but it is currently rarely used because of its systemic toxicity. In the tumor microenvironment, IFN-α exerts its anti-tumor activity both directly by promoting cancer cell apoptosis and inhibiting vascularization and indirectly by restoring anti-tumor...