At AANASTRA we are harnessing the power of uniquely designed RNA along with the targeting capabilities of peptides to develop an entirely novel class of RNA-based therapeutic agents for the treatment of cancer and genetic diseases
ABOUT
AANASTRA (formerly AADIGEN) specializes in the development of in vivo targeted RNA therapeutics in the treatment of cancers and genetic diseases.
The company’s technology is based on proprietary RNA and proprietary peptides complexed together for targeted in vivo delivery of RNA.
Our versatile breakthrough technology tackles the major stumbling block in RNA therapeutics which continues to be the systemic in vivo targeting and cellular-release of RNA-based agents. Our approach provides an effective strategy for a wide range of clinical applications and pathologies.
The company was founded by Dr. Neil Desai (Abraxis, Celgene, Aadi Bioscience) and the technology and its use in the delivery of RNA therapeutics is based on the original research of Dr. Gilles Divita, who pioneered the cell penetrating peptide strategy for oligonucleotide delivery. Aanastra (Los Angeles, CA) works closely on its RNA therapeutics technology with its sister company Divincell SAS (Nimes, France).
SCIENCE & INNOVATION
Advances in RNA Therapeutics and Limitations
Advances in RNA Therapeutics and Limitations
There has been high level of recent interest in gene modulation with RNA based technologies (mRNA, gene editing, etc). Delivery Systems for RNA (typically viral or lipid based) are essential because RNA can cause strong immune/cytokine responses and are highly susceptible to degradation. Most delivery systems accumulate in the liver after IV (systemic) administration, cause toxicity and there are no good strategies for disseminated diseases like cancer.
The current RNA therapeutic applications are therefore limited to vaccines (local IM injection), ex vivo gene manipulation/editing (CAR-T, sickle cell), local delivery (eye) or liver-based diseases. Effective extra-hepatic targeting (e.g., for solid tumors) remains a major stumbling block in the development of RNA therapeutics.
Limitations of Cancer Therapy Today
The root cause of most cancers – i.e., Alterations/mutations in Tumor Suppressor Genes (TSGs) and Oncogenes (OGs) often cannot be directly targeted (only ancillary and secondary targets resulting from these gene alterations may be targetable). Currently available approaches in targeted therapy most often result in build-up of resistance over time due to development of alternate and redundant biological signaling pathways and cause ultimate failure of the treatment.
Aanastra's Approach for the Future of RNA-based Cancer Treatment
Restoring, replacing or removal of the culprit genes as a strategy to treat cancers remains an untapped area due to delivery technology limitations. Aanastra’s approach to targeting TSGs and OGs can provide a major advance in treatment of cancer.
Our focus in cancer is based on a unique two-pronged approach: in vivo TUMOR SUPPRESSOR RESCUE™
(TSG-RESCUE™ , TSG-RESQ™) and in vivo ONCOGENE EDITING™ (ONCOEDIT™). These approaches have been validated by numerous studies in therapeutic cancer models including lung cancer, colorectal cancer, pancreatic cancer and osteosarcoma against targets like P53, BRCA1 and KRAS.
Our focus in other genetic diseases has been validated by studies in therapeutic models including reversal of hemophilia A (Factor VIII) and hypercholesterolemia (PCSK9) using our technology.