WAVE LIFE SCIENCES – Exon Skipping Investigational Drugs

Frequently Asked Questions (FAQs) about this research:


What stage is this research?

  • This research is pre-clinical, 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 the early discovery research in house (Cambridge, MA) and in collaboration with Dr. Matthew Wood at University of Oxford in the UK.


What is the goal or purpose of this research?

  • The goal of our research is to develop exon-skipping drugs that show increased dystrophin production and enhanced delivery in multiple muscle groups.


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

  • Required IND-enabling and safety preclinical studies will be conducted in order to file our IND. We plan to be in the clinic in the second half of 2017.


Where would a clinical trial take place?

  • The company hopes to have clinical trial sites in the United States and in other countries outside the United States. Sites have not been selected at this time.


Who would be eligible to participate in a clinical trial?

  • Again, it is too early to know what the inclusion criteria would be for a future clinical trial. However, our plan is to include ambulatory and non- ambulatory patients in our trials.


Where can I learn more about this research?