Juventas' lead product JVS-100 is a non-viral gene therapy which encodes for stromal cell-derived factor-1 (SDF-1).
Stromal cell-Derived Factor-1 (SDF-1) is a naturally-occurring signaling protein that is over-expressed in response to tissue injury. SDF-1 is a strong chemo-attractant of stem cells and progenitor cells that promote tissue preservation and increased and sustained healing of wounded tissue. SDF-1 induction stimulates a number of protective anti-inflammatory pathways, causes the down regulation of pro-inflammatory mediators, prevent cell death, and promote tissue preservation and blood vessel development. Regeneration in adult tissue is generally driven by localized trauma which results in the expression of signaling proteins such as SDF-1. Unfortunately, natural SDF-1 expression lasts for less than a week, and therefore the resultant repair signal quickly fades.
An extensive body of research has demonstrated that increasing transient SDF-1 expression post-tissue injury promotes tissue repair after acute organ injury by preventing cell death and recruiting stem cells to the damaged region. Recent articles from several laboratories have demonstrated SDF-1 therapeutic potential for treatment of cardiovascular disease, stroke, wound management, diabetes, spinal cord repair, and other indications. Juventas has demonstrated that therapeutic interventions which re-establish the ability of SDF-1 to stimulate repair mechanisms may be beneficial for patients that have suffered tissue damage, such as after a heart attack.
JVS-100 has been the subject of several clinical studies and has been administered to more than 200 patients with no significant side effects. JVS-100 is comprised of a naked DNA plasmid which has been engineered to express SDF-1 transiently for approximately three weeks. The vector is non-immunogenic which provides for a strong safety profile and the ability to allow for repeat administrations. JVS-100 benefits from highly scalable manufacturing, a modest cost of goods, and a long shelf life of over two years.
The below slides demonstrate the delivery of JVS-100 to heart failure patients as performed in our Phase II STOP-HF clinical study.