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Undoubtedly, the field of gene therapy has matured as there are now 43 FDA-approved gene and cell therapies, with seven approved in 2024 alone. Robust innovation of genetic modification technologies by academic and industry innovators, combined with selection of high unmet need inherited disorders has advanced a record number of programs into clinical development. Consequently, we anticipate a wave of regulatory filings leading to approvals of first-in class therapeutics derived from these novel modalities. Despite the highly publicized clinical successes of gene therapies, concerns remain over the challenging commercial and financial pressures facing biopharmaceutical companies. This has led many firms to abandon funding preclinical and clinical stage ultra-rare disease programs, many with strong proof of concept, simply because they don’t meet the developing firm’s return on investment requirements to remain in their portfolios.
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