Raymond James has initiated coverage on WAVE Life Sciences Ltd
with a price target of $9 and an Outperform rating.
The analyst says Wave is the leader to clinic in RNA editing with WVE-006 for Alpha-1 antitrypsin deficiency (AATD) (expected to begin first in human studies early next year), being developed in collaboration with GSK Plc
.AATD is the lack of a protein made by the liver that's released into the bloodstream. AATD is a genetic disease that can affect the liver or lungs. The alpha-1 protein is designed to protect tissues in the body from being attacked by its enzymes.
The discovery collaboration has an initial four-year research term.
The collaboration includes two main components. The first is a discovery collaboration that enables GSK to advance up to eight programs and Wave to advance up to three programs.
RNA editing via endogenous ADARs is a potentially safer alternative to one-and-done CRISPR/gene editing technologies, Raymond James analyst says.
AATD is an optimal proof-of-concept indication given both loss of function (lung manifestation) and gain of function (liver manifestation), which means correcting the causative mutation is the most efficient therapeutic approach in theory (vs. RNAi or gene insertion, which only addresses one or the other separately).
Most recently, Wave discontinued the development of WVE-004 after reporting disappointing data from Phase 1b/2a FOCUS-C9 study for C9orf72-associated amyotrophic lateral sclerosis and frontotemporal dementia.
