Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Cardiovascular disease

Heart failure

SMT3 suppressor of mif two 3 homolog 1
(SUMO1); ATPase Ca++ transporting cardiac muscle slow twitch 2 (ATP2A2; SERCA2A)

Pig studies suggest SUMO1 gene therapy could help treat heart failure. Previous work in mouse models of heart failure showed that an adeno-associated viral (AAV) vector expressing mouse Sumo1 increased cardiac function compared with an AAV vector expressing GFP. In pig models of heart failure, an AAV serotype 1 (AAV1) vector expressing human SUMO1, human SERCA2A or both all led to similar increases in cardiac function compared with saline control. Ongoing work includes selecting which AAV vector and promoter to advance to IND-enabling toxicology studies.
Celladon Corp.'s Mydicar, a recombinant AAV vector bearing SERCA2A, is in Phase II testing to treat heart failure and preclinical testing to treat hypertension.

SciBX 6(48); doi:10.1038/scibx.2013.1387
Published online Dec. 19, 2013

Patented by the Icahn School of Medicine at Mount Sinai; unlicensed

Tilemann, L. et al. Sci. Transl. Med.; published online Nov. 13, 2013;
doi:10.1126/scitranslmed.3006487
Contact: Roger J. Hajjar, Icahn School of Medicine at Mount Sinai, New York, N.Y.
e-mail:
roger.hajjar@mssm.edu
Contact: Kiyotake Ishikawa,
same affiliation as above
e-mail:
kiyotake.ishikawa@mssm.edu
Contact: Ahyoung Lee, same affiliation as above
e-mail:
ahyoung.lee@mssm.edu
Contact: Lisa Tilemann, same affiliation as above
e-mail:
lisa.tilemann@googlemail.com