WVE-006 is an investigational GalNAc-conjugated RNA-editing oligonucleotide designed for subcutaneous administration and developed using Wave Life Sciences’ proprietary oligonucleotide chemistry platform. The therapy employs adenosine-to-inosine (A-to-I) RNA editing to correct the pathogenic single-nucleotide RNA change responsible for most cases of alpha-1 antitrypsin deficiency associated with the Pi*ZZ genotype. Through this targeted RNA correction, WVE-006 is intended to restore production of functional, wild-type alpha-1 antitrypsin while reducing accumulation of the misfolded mutant protein in the liver, with the goal of addressing both pulmonary and hepatic disease manifestations.
Disease: Alpha-1 Antitrypsin Deficiency, AATD, (NCT06405633)
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Candidate summary:
Disease/Disease category:
- Genetic metabolic diseases
Official title:
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)
Phases:
- Phase 1
- Phase 2
Status:
Active not recruiting
Country:
- United Kingdom
- New Zealand
- Finland
- Canada
- Australia
