A Bold Goal to Cure Sickle Cell Disease by 2030

By IGT Scientific, Academic & Medical Advisory Council Chair Donna M. Christensen


Last week, during the Congressional Black Caucus Foundation’s 51st Annual Legislative Conference, I had the opportunity to join policymakers, patients, and medical experts for the 2nd Annual Sickle Cell Disease Issue Forum. I was inspired by the dedication each of the panelists brings to their role in improving the lives of patients with sickle cell disease (SCD).


SCD affects approximately 100,000 people in the U.S. alone. It is an inherited red blood cell disorder that predominantly affects African Americans. The condition is severe and life-threatening, involving significant pain, organ damage and infection when misshapen blood cells block blood flow. Patient advocate and founder of the Ruby Ball Foundation, Ijeoma Azubuko, explained that living with SCD goes beyond the pain. SCD patients worry about symptoms and progression throughout their life.


We were honored that Department of Health and Human Services Secretary Xavier Becerra joined the session and addressed the panelists and attendees. Noting the seriousness of SCD and the historic racism underlying research and treatment of the disease, he put forth a bold, but achievable, goal: Cure sickle cell disease by 2030.


With leaders in innovation, like the Institute for Gene Therapies’ (IGT) Corporate Advisory Council member and Forum sponsor Vertex, plus others who are developing transformative therapies, the community has reason to be hopeful. Recent progress in gene therapy studies and clinical trials demonstrate great promise for the treatment of SCD. Gene therapies are fundamentally different from traditional drugs in that they treat diseases at the DNA level getting at their root cause for lasting effect.


The panel was hosted by two Members of Congress, Rep. Barbara Lee (D-CA) and Rep. Danny Davis (D-IL), who expressed their dedication to the fight. Congresswoman Lee explained the importance of “continued partnership to build awareness and beat this disease.”


As the Chair of IGT’s Scientific, Academic & Medical Advisory Council, I couldn’t agree with her more. IGT is committed to continued collaboration with the SCD community, so patients can access safe, effective gene therapies. The promise of transformative gene therapies is incredible for patients with genetic diseases like SCD. These often-one-time treatments target the source of the disease, rather than mitigating signs and symptoms, giving us an opportunity to change the future of care.


However, as Tennessee State Senator Raumesh Akbari noted, our government must keep pace with the science, “…we have evolving technologies, but governments are the slowest to catch up. People with sickle cell disease who are on government assistance programs should have opportunities to benefit.”

Access to and affordability of potentially curative treatments are essential. To achieve both we must define value holistically and equitably starting with what matters to patients. We must address the barriers and inequities that exist in all aspects of our healthcare system, including the misconceptions and stereotypes surrounding SCD, barriers to screening and diagnostic tools, and outdated reimbursement models.


IGT is focused on innovative ways of paying for gene therapies so that all patients who stand to benefit, will have access. We are working with our patient, academic, scientific, and corporate stakeholders to better define and educate policymakers and the public about the unique value that gene therapies offer. And we’re helping to remove barriers to these transformative treatments for patients with SCD and others. If we all commit to the goal that Secretary Becerra set forth – curing SCD by 2030 – we will achieve it.