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20 February 2019

An Important Step in the Advancement of mRNA medicines: Newly Published Clinical Data Show the Early Potential of VEGF-A mRNA as a Regenerative Therapeutic

Tal Zaks
M.D., Ph.D., Chief Medical Officer, Moderna

This week we were very excited to share newly published clinical data showing the potential of mRNA encoding vascular endothelial growth factor-A (VEGF-A) as a regenerative therapeutic in patients. Published in Nature Communications, these findings represent an important milestone as we seek to further understand and establish the safety and efficacy of delivering mRNA to human tissues.

The Phase 1 a/b study of Moderna’s mRNA encoding VEGF-A (AZD8601) achieved three key objectives critical to advancing our joint program into a Phase 2a trial:

  • Tolerability: The study met its primary objective of describing safety and tolerability of AZD8601 injected directly into the skin of patients.

  • Protein Expression : AZD8601 demonstrated dose-dependent VEGF-A protein production.

  • Protein Pharmacology: Increased blood flow was observed at injection sites up to seven days following a single injection of AZD8601.

These results, while early, may hold promise for conditions in which blood flow may be compromised, including heart disease, diabetes and other vascular complications.

Our strategic collaborator AstraZeneca currently is leading a Phase 2a study focused on the therapeutic potential of mRNA for regenerative angiogenesis – or generating new blood vessels and improving blood flow in tissues where it is otherwise restricted – in patients undergoing coronary artery bypass grafting (CABG) surgery. While progress in medicine and surgery has improved outcomes for people with heart failure, only about half the patients remain alive five years from the time they leave the hospital.

This study, which is ongoing in Europe, will help us to better understand how localized injections of mRNA into the heart can support regenerative angiogenesis – and whether this may offer a therapeutic approach to improving cardiac function for patients over time.