Questions and Answers

What stage is this research?

This research is in the preclinical phase, meaning it has not advanced to clinical trials involving people yet.

Where is this research being done and who is funding this research?

Wave Life Sciences is conducting discovery research in Cambridge, MA, and in collaboration with Dr. Matthew Wood at the University of Oxford in the UK. This research is being funded by Wave Life Sciences.

What is the goal or purpose of this research?

The goal of this early research is to work toward developing a medicine for people with Duchenne muscular dystrophy amenable to exon 53 skipping.

What steps need to be completed before moving into a clinical trial?

Required research studies must be completed and regulatory applications filed and approved prior to initiating a clinical trial.

Where would a clinical trial take place?

The company expects to run a global clinical trial and hopes to have clinical trial sites in the United States. Sites have not been selected at this time.

Where can I learn more about this research?

Please visit www.wavelifesciences.com or email medicalinformation@wavelifesci.com for additional information.