Wave Life Sciences

Korro Bio's lead RNA editing therapy KRRO-110 for alpha-1 antitrypsin deficiency failed to produce functional protein at expected levels in early clinical testing.

Wave Life Sciences announced dose-dependent Activin E reductions of up to 85% one month after a single dose of WVE-007 in the INLIGHT clinical trial for obesity treatment.

Wave Life Sciences' experimental RNA editing medicine WVE-006 successfully helped patients with alpha-1 antitrypsin deficiency produce therapeutically relevant levels of a critical protein their bodies cannot make.

XtalPi has signed a Letter of Intent with DoveTree LLC, founded by Harvard Professor Gregory Verdine, for a collaboration worth $100 million upfront plus potential milestone payments exceeding $10 billion.

Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production.

Wave Life Sciences achieves first-ever RNA editing results in humans for alpha-1 antitrypsin deficiency, demonstrating therapeutic protein level increases with a single dose treatment.

Wave Life Sciences demonstrates groundbreaking success with WVE-006, the first RNA-editing therapy tested in humans for alpha-1 antitrypsin deficiency, showing 60% increase in functional enzyme levels.

Wave Life Sciences is set to initiate dosing for WVE-007 in its Phase 1 INLIGHT trial for obesity in Q1 2025, with proof-of-concept data expected later in the year.

• Ionis Pharmaceuticals, a pioneer in antisense oligonucleotide (ASO) drugs, priced a $500 million IPO to fund clinical programs and commercial launches. • Isarna Therapeutics' lead candidate, ISTH0036, targeting TGF-β, shows promise in Phase 2 trials for wet AMD and DME, major ophthalmic conditions. • Regulus Therapeutics is advancing RGLS8429, an ASO targeting microRNA for autosomal dominant polycystic kidney disease (ADPKD), with positive Phase 1b data. • Sarepta Therapeutics, with three approved PPMO therapies for Duchenne muscular dystrophy (DMD), continues to expand its RNA-based therapeutic pipeline.

• Korro Bio has received approval to begin a Phase I/IIa trial in Australia for KRRO-110, an RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). • The REWRITE trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of KRRO-110 in healthy adults and AATD patients with the PiZZ genotype. • KRRO-110 leverages Korro's OPERA platform to correct the disease-causing mutation in the SERPINA1 gene, potentially restoring normal AAT protein secretion. • Interim data from the trial is expected in the second half of 2025, with study completion anticipated in 2026.