We are embarking on an exciting mission to find treatment for SSMD disease and tackle other diseases where GPX4 plays a significant protective role. We are assembling a team of proven drug development experts to build a treatment for SSMD and lay the foundations for the future. This is an exciting opportunity to make a direct and meaningful impact to lives of patients living with this devastating disease.

2020 ACHIEVEMENTS

• Raised over $250,000 for research
• Built knock-in mouse model; Tested RT001 on knock-out mice; Created and stored patient-derived fibroblasts and iPSCs in biobank; Created cell-based screening assay; Understood the biochemical mechanism of action of GPX4 mutations; Treated one patient with RT001; In the process of starting Tecfidera to one patient; Started building gene therapy (currently in in-vitro verification).
• Submitted two papers: A roadmap paper explaining our research strategy and progress (In Review with Orphanet); A paper elucidating the biochemical mechanism of GPX4 mutation (In Review with Cell)

2021 GOALS

Short-term (3 - 6 months): Identify two additional FDA approved drugs to repurpose for SSMD

Long-term (12 - 18 months): Identify the most potent drug for this disease using any therapeutic tech (gene therapy, LnP, CRISPR etc).

Strategy: Treatment to SSMD today will be the stepping stone for treatments to larger indications such as Stroke, Ischemia-Reperfusion injury, or other skeletal dysplasia.

Proposed Investment: $500k

Advisors & Research Partners: curegpx4.org/team

OPEN POSITIONS

Email [email protected] if you are interested

CHIEF SCIENTIFIC OFFICER

Responsible for the scientific strategy of the organization including identifying opportunities to reach the short and long-term goals, creating strategic partnerships, providing directions for research programs, and evangelizing our work with the science and clinical community.

Volunteer only. Approx. 2 - 5hrs/week