join our team
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 sanath@gpx4.org 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
Medical Director
Responsible for providing clinical guidance on proposed mechanisms of intervention, charting the path to the clinic, and liaison with physicians and clinical partners.
Volunteer only. Approx. 2 - 4hrs/week
Director of Drug Development
Responsible for individual basic science, translational science and drug development activities including planning, drafting the statement of work, working with research partners, managing timelines and communications.
Volunteer or Part-time. Approx. 5 - 10hrs/week
Various positions available. You will be responsible for diving deep into the science when needed including actively conducting research, writing papers/articles, drafting statements of work, analyzing results, proposing new research projects etc.
Volunteer only. Approx 2 - 4 hrs/week