GSK and Wave Life Sciences have announced they will collaborate on advancing oligonucleotide therapies such as Wave’s preclinical RNA editing program to treat alpha-1 antitrypsin deficiency (AATD).

The announcement, reported via news release, notes that the collaboration has an initial 4-year term and will combine GSK’s human genetics insights and commercial capabilities with Wave’s PRISM drug development platform.

“Oligonucleotide therapeutics are becoming a mainstream modality, and this collaboration will enable us to use our leading position in human genetics and genomics to advance novel oligonucleotide therapies,” Tony Wood, president and chief scientific officer of GSK, said. “Pairing GSK’s genetic expertise with the best-in-class PRISM platform enables us to accelerate drug discovery for newly-identified targets, by matching target to modality.”


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Oligonucleotides are short strands of DNA or RNA that can alter RNA to address a number of genetic diseases, particularly those without current treatments or with limited success with small molecules or biologics, such as AATD.

Read more about AATD therapies

AATD is a rare genetic disorder resulting from a mutation in the SERPINA1 gene, and it leads to lung disease due to the absence of alpha-1 antitrypsin, a protective enzyme in the lungs.

Wave’s PRISM platform offers editing, splicing, and silencing of RNA to optimize the pharmacological activities of oligonucleotides. GSK will receive an exclusive global license for WVE-006, which is Wave’s preclinical AATD program employing their proprietary “AIMer” technology to treat both the liver and lung manifestations of AATD via RNA editing.

The AIMer technology is designed to correct mutations in RNA transcripts by recruiting ADAR enzymes, which are normal proteins in the body that edit DNA bases and can revert mutations that cause disease. The technology redirects a natural system for the treatment of disease and avoids some of the pitfalls of DNA-targeting approaches such as their irreversible nature.

Reference

GSK and Wave Life Sciences announce collaboration to drive discovery and development of oligonucleotide therapeutics focusing on novel genetic targets. News release. GSK plc and Wave Life Sciences Ltd; December 13, 2022.