Last month, Wave Life Sciences announced their first-ever RNA editing therapy, WVE-006, which is set to enter clinical trial development. This program is designed to restore the circulation and production of wild-type alpha 1 antitrypsin (AAT) protein and reduce the concentration of Z-AAT protein, helping to treat AATD, a genetic lung and liver disease associated with AAT protein deficiency.
If approved, the company expects to commence the dosing session of first set of patients in the fourth quarter of 2023 and aims to gather protein restoration data of treated patients by 2024.
The United States based genetic medicine company entered a strategic partnership with GSK in a four-year research term. This collaboration is set to leverage GSK’s insights from human genetics in combination with Wave’s proprietary discovery and drug development platform PRISM. Under the terms of the partnership, Wave received USD 170 million in cash at the start of the partnership.
Wave is also entitled to receive up to USD 225 million for development and launch milestone payments and up to USD 300 million for sales related milestone payments, coupled with double digit royalties as a percentage of net sales for WVE-006 in 2023.
The ongoing clinical development plan includes AATD affected individuals with homozygous PiZZ mutation, along with healthy volunteers. It is designed to provide a competent path to proof-of-mechanism by reinstating the M-AAT protein in serum. It works on correcting the single G-to-A base mutation in mRNA, coded by the SERPINA1 Z allele, which assists the production of functional and wild-type M-AAT protein.
WVE-006 holds paramount significance, as it is a first in class GalNAc-conjugated RNA editing oligonucleotide. It stands out by offering advantages over regular DNA editing. It is much safer and also reversible, offering infrequent subcutaneous dosing. Infrequent subcutaneous dosing offers multiple benefits like reduced side effects, patient convenience and easier compliance with the treatment plans. Moreover, it causes no permanent changes to the genome and shows no evidence bystander editing (unintended changes made to genomic sequences adjacent to the targeted site).
This clinical trial has also garnered huge traction as it addresses alpha-1 antitrypsin deficiency, a common cause of genetic lungs and liver diseases. The prevalence of AATD is reported to be around 1 to 5000 to 1 in 10,000 in United States and Canada, while it spans across the European region affecting around 1 in 5000 individuals.
In the previous month, Wave also hosted a virtual event called ‘Towards the Clinic: Spotlight on RNA Editing for AATD’, highlighting the therapeutic potential of the WVE-006 in front of investors and analysts. The event featured a presentation from D. Kyle Hogarth, MD, FCCP, Professor of Medicine in the Section of Pulmonary and Critical Care Medicine at the University of Chicago, a clinical AATD expert.
At the event, Paul Bolno, MD, MBA, President, and CEO of Wave Life Sciences added, “RNA editing is a promising new therapeutic modality, capable of accessing untapped areas of disease biology. We are incredibly proud to be pioneers leading the way forward in RNA editing, and we plan to share more updates on AATD and other RNA editing programs during our R&D Day later this month.” Looking ahead, the company also plans to take RNA editing beyond the application of hepatocytes.
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