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The Next Decade In Gene Therapy Innovation â 6 Critical Questions (And Answers)
By Kyle M. OâNeil and Brendan J. Wang, Back Bay Life Science Advisors
As the gene therapy field has tackled delivery vectors as described in “An Analysis Of The Gene Therapy Viral Vector Landscape,” several clinical and commercial questions lowered expectations for gene therapy sales, leading sell-side forecasts to fall more than 50% since 2018.
1 However, more than $6.5 billion in 2026 projected gene therapy sales remain across Duchenne muscular dystrophy (DMD), hemophilia A/B, sickle cell disease, Fabry disease, Huntington’s disease, and Leber congenital amaurosis type 10 (LCA10) alone, putting pressure on industry-leading gene therapy companies to deliver.
An Analysis Of The Gene Therapy Viral Vector Landscape
By Kyle M. O’Neil and Brendan J. Wang, Back Bay Life Science Advisors
Over the last five years, multiple gene therapies have been approved by regulatory agencies and a bolus of late-stage pipeline assets are approaching the market. As the first few gene therapies realize their potential as transformative treatments for the genetic rare diseases, the space is seen as a crucial part of growth for the biopharmaceutical industry. However, as the first gene therapies began to post early wins, several challenges have emerged. With these challenges in mind, leading companies have begun to search for new delivery technologies. Only after addressing delivery will gene therapies be able to achieve their longstanding potential to transform patient outcomes, expand into larger diseases, and ultimately provide returns for investors.