Originally published in BioPharma Reporter on December 21, 2017 (link to article no longer active; summary provided here).
The year 2017 marked a pivotal moment in the cell and gene therapy landscape, characterized by groundbreaking product approvals and significant regulatory advances that transformed the field from experimental medicine to clinical reality.
As Dark Horse Consulting Chairman Anthony Davies noted at the Cell Therapy Manufacturing & Gene Therapy Congress in Amsterdam, 2017 was remarkable for the scope of regulatory changes rather than stagnation. The year began with crucial FDA guidance for the newly introduced Regenerative Advanced Therapy designation, later rebranded as Regenerative Medicine Advanced Therapy (RMAT) to better reflect its purpose.
Under the leadership of FDA Commissioner Scott Gottlieb, who demonstrated "a significant level of interest in cell and gene therapy," regulatory momentum accelerated throughout the year. Gottlieb famously declared that cell and gene therapies were "no longer the stuff of science fiction" while publishing comprehensive industry guidelines for regenerative medicines.
The year's defining moments came through three landmark approvals that validated the potential of advanced therapies:
CAR-T Cell Therapies Break Through
Gene Therapy Milestone
These approvals took on added significance given the safety concerns that had previously shadowed the field. Earlier setbacks, including clinical trial holds for Juno Therapeutics' JCAR015 in 2016 and Cellectis' UCART123 in 2017, had raised questions about whether such complex therapies could successfully translate to clinical practice.
As Paula Salmikangas from NDA Group AB observed, the successful authorizations largely offset these worrying developments, proving that with proper development and regulatory oversight, advanced therapies could deliver on their promise.
The regulatory framework established in 2017, combined with these first-in-class approvals, created a foundation for the continued growth of cell and gene therapies. The year demonstrated that with appropriate regulatory guidance and rigorous development processes, these innovative treatments could move from laboratory concepts to life-changing therapies for patients with previously untreatable conditions.
This summary is based on reporting by Dan Stanton for BioPharma Reporter. Dark Horse Consulting continues to provide regulatory expertise and strategic guidance for companies developing advanced therapies.
