A Major Step Forward for MSMDS: VESSEL Project Receives Up to $25.8 Million

There is exciting news for the ACTA2/MSMDS community.

The VESSEL Project, led by Dr. Patricia Musolino and her team at Mass General Brigham, has been awarded up to $25.8 million from the Advanced Research Projects Agency for Health (ARPA-H) to advance gene-editing treatments for rare genetic vascular diseases. MSMDS is specifically included in the project.

VESSEL—Vascular mEdicineS using Scalable Editing pLatform—is developing a gene-editing platform designed to target genetic mutations responsible for serious vascular diseases. The goal is to deliver gene-editing tools directly to the vascular cells affected by these conditions and ultimately correct the underlying genetic cause.

For the ACTA2 and MSMDS community, this is particularly meaningful because there is currently no treatment that corrects the underlying ACTA2 mutation.

The new funding will allow the team to advance this work through important preclinical, manufacturing, safety and regulatory steps needed before a gene-editing treatment can eventually be tested in people.

This does not mean a treatment for MSMDS is available yet, or that a clinical trial is imminent. There is still significant work ahead. But this level of funding represents a major investment in moving gene-editing science toward real treatments for people living with rare vascular diseases.

The ACTA2 Alliance is proud to be involved in this effort through the VESSEL Patient Advocacy Committee, with representatives Warren Anderson, Lydia Anderson, and Angela Cas contributing the patient and family perspective.

For families affected by MSMDS, this is an important moment of hope.

The science is moving forward. And so is the possibility of a future treatment that addresses the cause of MSMDS—not just its complications.