Is there a piece of your work —a program, a campaign, a breakthrough, a report, a paper — that you’d like more people to know about? Tell us about it and share a link if there is one.
A breakthrough that I would like to share that is extremely important to me is the recent approval of the first gene-therapy for Sickle Cell Disease using CRISPR. This approval and delivery of treatment to market was a very important moment in my career because of the impact and meaning it will have for the Sickle Cell community and the Rare Disease movement as a whole. Advancing treatments in the rare disease space has many challenges but wins such as this one helps everyone receive the proper care that they deserve. There is a wonderful publication that describes this breakthrough in the American Journal for Cell & Gene Therapy.
We recently held a very powerful congressional briefing to a bipartisan standing room only audience. The panel was comprised of patients, providers, community leaders and Pharma. The event sparked many conversations and helped to elevate the awareness of a newly reformed caucus dedicated to Sickle Cell Disease and to SCD awareness as a whole within the policy and legislative space which is essential for new treatments to come to market. A win for one rare is a win for all rare which will improve the lives of many!!!
