ONE RNA EDIT AT A TIME
AIRNA’s powerful and flexible RNA editing platform, RESTORE+™, is designed to restore a patient’s health by creating genetic medicines for rare and common diseases. RESTORE+™ is a systematic advancement of the first published ADAR platform developed by AIRNA’s founders, created to maximize therapeutic properties and address novel targets.
from RNA to protein
RNA contains the genetic code for protein synthesis. RESTORE+™ can specifically target a defined site in the RNA strand, activating changes to the composition and functionality of a therapeutically relevant protein.
Targeting the RNA
AIRNA’s distinctive platform optimizes the chemistry, sequence, and delivery of oligos. Once delivered into the cell, the oligo precisely engages the RNA at the target site.
Site-directed RNA editing by ADAR
After target engagement, the oligo recruits endogenous ADAR to make an adenosine-to-inosine (A-to-I) edit at the target site that is read as guanosine (G).
Change of protein function
The A-to-I edit changes the code in the RNA, enabling the repair of pathogenic point mutations, or inducing therapeutically effective gain or loss-of-function mutations that precisely change protein activity.
One rna Edit at a Time
Precision RNA base editing with engineered and endogenous effectors
Laura S Pfeiffer, Thorsten Stafforst. Nat Biotechnol. 2023 Sep 21. doi: 10.1038/s41587-023-01927-0.
Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides
Merkle T, Merz S, Reautschnig P, Blaha A, Li Q, Vogel P, Wettengel J, Li JB, Stafforst T. Nat Biotechnol 2019, 37:133-138, doi: 10.1038/s41587-019-0013-6, PMID: 30692694.