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Mitigating payer-identified risks in cell and gene therapy development

As the case with several therapeutics, developers struggle to balance between clinical and commercial objectives. In a recent webinar, Drs. Emily Woodward and Max Vargas touched on the battle between these two objectives, among other access and reimbursement considerations, in the world of cell and gene therapy (CGT) development. CGT approaches seek to modify genetic … Continued

希少疾患に対する遺伝子治療の期待と落とし穴

遺伝子治療の現状は多くの不確実性に直面しており、最近の規制や臨床上の失敗によって、その将来性に疑問が投げかけられています。希少疾患の遺伝子治療薬としてFDAに認可されたものは、現在までに2種類しかなく、これらの医薬品を開発する道のりはまだ険しいです。This presentation exploreed how the biopharma industry can overcome development challenges in the … Continued

Commercial and Regulatory Success in Accelerated Gene Therapy Development

Thanks to regulatory frameworks and driven by high unmet patient needs, the pace for clinical development of gene therapies (GT) is often greatly accelerated. This presents GT innovators with the opportunity to reach their patients faster, but it also raises the bar for critical decision points such as around trial design, endpoint selection and population … Continued

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