Cell and gene therapies (CGTs) have been gaining traction within the medical community as treatment options that are expected to replace lifelong management of chronic diseases with fewer number of doses, which could offer significant benefits for both patients and strained healthcare systems. However, alongside these opportunities, regulatory barriers and cost concerns pose challenges to their widespread adoption, according to GlobalData, a leading data and analytics company.

An analysis of GlobalData’s Drug Database reveals that over the past decade, a total of 14 cell and gene therapies were approved across various therapy areas in the US. Of these, over 70% of them were approved within the last five years (2019-2023). The opportunity for the approval of more CGTs remains high as there are 694 CGTs in development across various therapy areas in the US as of February 2024.

Sravani Meka, Senior Immunology Analyst at GlobalData, comments: “The landscape of CGTs is evolving rapidly, with 694 assets under development, 48 of which are in late-stage development. Previously, CGTs were primarily seen as solutions for rare diseases, but now they are being explored across various therapy areas like cardiology and central nervous system disorders. However, despite the robust pipeline, challenges persist in their adoption due to concerns around clinical trials, regulatory hurdles, and affordability.”

Challenges in CGT development emerge early, especially in clinical stages where patient access and recruitment pose significant hurdles. The rarity of some indications and the perception of CGTs as last-resort treatments hinder participation.

Regulatory frameworks may also struggle to keep up with the pace of advancements, despite efforts like establishing the Office of Therapeutic Products at the US FDA. Moreover, high production costs, particularly for gene therapies, present affordability issues, limiting accessibility within Medicaid systems.

Mekala concludes: “Despite challenges, CGTs offer personalized treatments with potential to revolutionize medicine. They hold promise for addressing difficult diseases and reducing reliance on conventional therapies. Long-term benefits include improved patient outcomes and reduced healthcare expenditures. Ongoing research in the field offers hope for expanded therapeutic options and breakthroughs in conditions like spinal cord injuries and neurodegenerative disorders.”