Are we there yet? After 250+ AAV-based clinical trials, do we have a well-paved road toward first-in-human entry?

Gene therapy is a revolutionizing technology that brings hope to millions of patients suffering from rare diseases. Unlike other mature therapeutic modalities, chemistry, manufacturing, and control (CMC) is often on the critical path for gene therapy programs to initiate clinical trials and obtain accelerated approvals. In recent years, health authorities and industry leaders have recognized a need of platform approach to streamline AAV-based gene therapy and initiated programs to address this need. Here we analyze these streamlining efforts in the industry, and emphasize the collective benefit of harmonizing the CMC platform, including manufacturing, analytics, and regulatory filing strategy, for AAV-based gene therapy. This endeavor is particularly impactful for accelerating advancement of gene therapies to the clinic at lower costs, and thereby further incentivizing drug development even for ultra-rare indications. Furthermore, considerations in building a minimum CMC package for initial clinical trial applications and some opportunities for harmonization are highlighted. Finally, potential future breakthroughs to alleviate the current pain points are discussed.

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