MARCH 2025LIFE SCIENCES REVIEW8s a new modality, Gene therapy offers great promise to develop effective treatments or potential cures for many serious diseases that previously had no meaningful treatment. While bringing new promises and opportunities, the field of gene therapy also faces a unique set of difficult challenges. During the past few years, regulatory agencies around the world have been taking very cautious approaches regarding gene therapy approval, demanding additional preclinical work, safety assessments and durability data. As a result, the gene therapy field encountered some regulatory headwinds, with programs put on clinical hold and regulatory filings significantly delayed or denied. There was no more gene therapy in the US approvals for three years after the first two approvals in 2017 and 2019.However, 2022 turned out to be a banner year in gene therapy regulatory successes. Four gene therapy products, Roctavian, Upstaza, Skysona, and Zynteglo, received regulatory approvals in the US and/or EU during the past few months. The fresh momentum came after many important changes made on the industry side in response to global regulatory guidance. There is reason to hope that this momentum will be here to stay.From the pipeline perspective, the field of gene therapy has been enjoying a rich and diverse collection of preclinical assets, in the thousands by recent count. Compared with small molecules, it's relatively easy to construct promising gene therapy assets at preclinical stages. However, according to the newly published ASGCT Q2 2022 report, merely a small percentage of preclinical gene therapy assets have moved into clinical development stages. Among those clinical-stage gene therapy assets, most are genetically modified cell therapies for oncology indications, and only a minority are gene therapies in other major therapeutic areas (e.g., neurological, sensory, alimentary/metabolic). The scarcity of clinical-stage gene therapy assets is not just a temporary CHALLENGES AND OPPORTUNITIES IN GENE THERAPY CLINICAL DEVELOPMENTBy Haichen Yang, MD, MA, MBA, Vice President, Clinical Research, Amicus Therapeutics IncHaichen YangIN MY OPINIONa
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