Wave Life Sciences Ltd., an RNA medicines company, has announced its first clinical trial application for WVE-006, a revolutionary RNA editing therapeutic candidate aimed at treating alpha-1 antitrypsin deficiency (AATD). AATD is a genetic disorder causing lung and liver diseases, affecting roughly 200,000 patients in the U.S. and Europe. Current treatments for lung disease involve weekly intravenous infusions, while liver disease has no specific treatment other than transplantation. WVE-006, however, offers a potential breakthrough. Engineered to correct the genetic mutation causing AATD, preclinical data shows that it not only restores the alpha-1 antitrypsin protein but also improves markers of liver disease.

The clinical development plan for WVE-006 will include both healthy volunteers and AATD patients with the PiZZ mutation. Trials are expected to begin in Q4 2023, with the company aiming to establish proof-of-mechanism by 2024. Success could pave the way for broader applications of RNA editing therapies.

This development is part of Wave’s strategic collaboration with pharmaceutical company GSK. Wave received $170 million upfront and could earn up to $3.3 billion in milestone payments and royalties. Specifically for WVE-006, the company could gain up to $225 million in development and launch milestone payments, and up to $300 million in sales-related milestones. The company expects its existing cash reserves to fund operations until 2025, not accounting for any future milestone payments.

WVE-006 employs a specialized RNA editing technique called A-to-I(G) RNA editing oligonucleotides (“AIMers”). Unlike DNA-targeting methods that make irreversible changes to the genome, AIMers recruit existing bodily enzymes to edit specific RNA sites, thereby providing a reversible and more targeted approach. With these features, WVE-006 presents a potentially game-changing treatment modality not only for AATD but also for a host of other genetic disorders.

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