Wave Life Sciences Investor Presentation — August 2021

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Wave Life Sciences Reports Second Quarter 2021 Financial Results and Provides Business Update

Dosing underway in FOCUS-C9 clinical trial of WVE-004 in C9-ALS / C9-FTD – first oligonucleotide using PN backbone chemistry modifications in

Recruitment ongoing for clinical trials in HD and DMD

Generating clinical data in multiple trials through 2022 to enable decision making

Leading endogenous ADAR editing capability demonstrated restoration of functional AAT protein in vivo; new data at Wave Research Day September

Wave to host investor conference call and webcast at 8:30 a.m. ET today

CAMBRIDGE, Mass. , Aug. 05, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company

committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the second quarter

ended June 30, 2021 and provided a business update.

“Since the start of the second quarter, Wave has achieved two significant milestones: the intrathecal dosing of patients with an oligonucleotide

containing PN chemistry and preclinical proof-of-concept protein restoration in vivo with ADAR editing for our alpha-1 antitrypsin deficiency program.

Both accomplishments reflect the significant evolution of our PRISM platform since Wave’s founding,” said Paul Bolno , MD, MBA, President and Chief

Executive Officer of Wave Life Sciences . “Amyotrophic lateral sclerosis and frontotemporal dementia are both areas of high unmet need; therefore we

are excited to advance WVE-004 in the FOCUS-C9 clinical trial, which is open to both patient populations as they have the same genetic cause of

disease. Additionally, we continue to progress our clinical trials for Huntington’s disease and Duchenne muscular dystrophy. Our novel PN chemistry,

stereochemical control and years of platform learnings have enabled us to lead the field on RNA editing and develop a best-in-class modality using

endogenous ADAR enzymes, which further expands our genetic medicines toolkit. In June we delivered our first preclinical proof-of-concept data for

our AATD program, and we believe RNA editing is the ideal approach to address this disease. Our upcoming Analyst and Investor Research Day in

September will feature new data on our ADAR editing capability, AATD program, and updates on applications of ADAR editing beyond liver, including

Highlights and upcoming milestones for clinical silencing and exon skipping programs:

WVE-004 for C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD):

WVE-004 is an investigational stereopure antisense oligonucleotide designed to selectively target transcript variants