Gene Therapy in 2024: 'Persistent' Approvals, Layoffs, and In Vivo Moving Toward the Clinic
12/18/2024
In 2024 the gene-therapy sector delivered a mix of promising regulatory progress and sobering headwinds. The U.S. Food and Drug Administration (FDA) granted approval to three new gene-therapy products this year and expanded indications for two additional products, all targeting rare diseases. Despite these wins, the field is showing signs of strain: biotech companies are announcing layoffs and tightening their pipelines amid rising costs, investor caution, and slower-than-hoped commercialization.
A major theme is the transition from ex vivo to more scalable in vivo gene therapies. The article highlights how delivery vectors (viral and non-viral) and manufacturing remain major bottlenecks. In particular, the shift to in vivo treatments—where the therapeutic gene is delivered directly to the patient’s body, rather than editing cells outside the body—promises broader reach but carries higher risks: less quality control, potential off-target effects, and more complex regulatory scrutiny.
Another focus is the commercial and operational environment: while rare-disease approvals build momentum, the cost and complexity of making and delivering gene therapies keep margins and growth uncertain. Companies are increasingly forced to refine focus, reduce headcount, and wait for clearer reimbursement pathways before scaling. The article suggests that although gene therapy has entered a new era of meaningful approvals, the industry must now contend with issues of access, affordability, delivery logistics and sustainable business models to realize its full promise.
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