From BioMarin’s hemophilia therapy offload to Sarepta’s FDA standoff, explore how top companies are recalibrating their gene therapy strategies in this Trendline.
Gene therapy is once again at the forefront of biomedical research, catalyzed by advances in safer delivery of genes to cells. Science may also move quickly past gene replacement therapy to gene editing via CRISPR and other methods, an approach in which the unknowns are even greater and clinical results are just beginning to emerge.
included in this trendline
- FDA unveils new regulatory roadmap for bespoke drug therapies
- A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases
- Sarepta, the FDA and a Duchenne gene therapy crisis
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