Wave Preclinical Exon Skipping Research

Wave Life Sciences – Exon Skipping Research

Questions and Answers

What stage is this research?

Wave is applying learnings from our exon 51 clinical program to further our exon skipping research in Duchenne to advance potential therapies targeting other exons. We have multiple research programs in the preclinical phase, meaning they have not advanced to clinical trials involving people yet.

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

This exon skipping research is being conducted and funded by Wave Life Sciences.

What is the goal or purpose of this research?

The goal of our research is to work toward developing medicines for people with Duchenne muscular dystrophy amenable to exon skipping. We have selected and are advancing a development candidate for exon 53 skipping. In addition, we are pursuing preclinical research toward investigational exon skipping therapies for people amenable to exon 44, 45, 52, 54, and 55 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 for any of our preclinical exon skipping programs.

Where would a clinical trial take place?

If our preclinical programs are ready to advance into the clinic, we expect to run global clinical trials and hope to have clinical trial sites in the United States. Due to the preclinical stage of our research, sites have not been selected at this time.

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