Wave Life Sciences (WVE) Corporate presentation summary

Strategic vision and platform innovation

• Focus on unlocking the potential of RNA medicines to transform human health, leveraging a multi-modal PRISM platform for RNA editing, RNAi, splicing, and allele-selective silencing.

• Integration of deep genetic insights and proprietary deep learning models accelerates discovery and clinic timelines.

• In-house GMP manufacturing and strong IP position support pipeline advancement.

• Well-capitalized with a cash runway into 2027.

Pipeline highlights and clinical progress

• Robust pipeline includes first-in-class RNA editing, siRNA, splicing, and allele-selective silencing programs targeting obesity, AATD, DMD, and HD.

• WVE-007 (obesity): Preclinical data show dose-dependent weight loss without muscle loss; synergistic with GLP-1s and potential for infrequent dosing.

• WVE-006 (AATD): Achieved proof-of-mechanism in patients, increasing functional AAT protein; ongoing RestorAATion-2 trial with positive safety and efficacy signals.

• WVE-N531 (DMD): Demonstrated best-in-class dystrophin expression, functional improvements, and reversal of muscle damage; NDA filing planned for 2026.

• WVE-003 (HD): Industry-leading allele-selective mHTT reduction with preservation of wtHTT; Phase 2/3 study preparation underway.

Market opportunity and upcoming milestones

• Pipeline addresses diseases with large patient populations in the US and Europe, including obesity (~175M), AATD (~200K), DMD (~20K new cases/year), and HD (>200K).

• DMD portfolio targets >$2.4B US market, with potential expansion to additional exons and global markets.

• Anticipated milestones in 2025–2026 include clinical data readouts for WVE-007 and WVE-006, NDA submission for WVE-N531, and initiation of new RNA editing programs.

• Regulatory feedback supports accelerated approval pathway for DMD program; multiple CTAs planned for exon skipping franchise.