Wave Life Sciences

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.

• Several companies are advancing potential Huntington's disease treatments, including Wave Life Sciences with WVE-003 and uniQure with AMT-130. • PTC Therapeutics' PTC518 received FDA Fast Track designation after demonstrating reduced mutant huntingtin protein levels in Phase 2 trials. • Prilenia Therapeutics' pridopidine is under EMA review, potentially offering the first treatment to impact Huntington's disease progression. • Despite recent trial failures, the Huntington's disease therapeutic field remains optimistic due to genetic-level targeting advancements.

Wave Life Sciences reported the first-ever RNA editing in humans with WVE-006 for alpha-1 antitrypsin deficiency (AATD), showing increased wild-type AAT protein levels.

• Several Huntington's disease therapies are nearing regulatory approval, potentially becoming the first disease-modifying treatments for the condition. • Prilenia Therapeutics' pridopidine has an application accepted by the EMA, while Wave Life Sciences and uniQure are in discussions with regulators for accelerated approval pathways. • Wave Life Sciences' WVE-003 demonstrated a significant reduction in mutant HTT protein levels and slowed caudate atrophy in clinical trials. • uniQure's AMT-130 gene therapy showed significant slowing of disease progression and reduction in neurofilament light chain levels in clinical studies.