Gene Therapies For All: Getting Life-Changing Treatments To Everyone In Need

By Krishna Kalyan A, Malik Kaman, Pranav Srivastava, and Pedro Ornelas

After decades of research in gene therapies—as well as successes and setbacks—approval of therapies for rare diseases are offering patients hope. Recent years have seen the approval of treatments such as Zolgensma for spinal muscular atrophy (SMA) and Luxturna for Leber’s congenital amaurosis (LCA) and retinitis pigmentosa (RP). This means approximately 2,000 U.S.-based patients are eligible for gene therapies today and can now hope for transformative outcomes.

While the science of gene therapies and their potential effects on health outcomes are impressive, adoption has not been as high as expected. So far in the U.S., only an estimated 300 patients have received commercial gene therapies, or approximately 15% of the eligible population.

There are several explanations for this slow uptake, ranging from barriers around the logistics of deploying these treatments, to a general lack of awareness of them by patients and physicians—not to mention hurdles tied to reimbursement and commercialization. We explore the role gene therapy manufacturers play in addressing these challenges to help pave the way for a future gene therapy landscape.