The Future of Indiv. Medicine: Funding Models & Regulatory Considerations for Rare Genetic Therapies скачать в хорошем качестве

The Future of Indiv. Medicine: Funding Models & Regulatory Considerations for Rare Genetic Therapies

Individualized medicine has emerged as an approach offering bespoke therapeutics to patients with rare genetic conditions. We will begin with a discussion of the current regulatory pathways available to offer this investigational treatment. In spite of this advancement substantial challenges remain, including funding models supporting such endeavors. In this discussion we will explore historically successful funding models, as well as considerations related to financing such treatments. The meeting began with Erin welcoming attendees and announcing an upcoming N1C annual meeting, followed by Ashley providing a regulatory overview of individualized medicines and N of 1 INDs in the U.S. Roger then presented on funding individualized therapies, discussing the challenges and resources required for these treatments and sharing findings from a survey conducted with the InnovOne Collaborative. The discussion concluded with participants addressing implementation aspects of NF1 therapeutic development, regulatory challenges, and ongoing trials, while agreeing to connect at upcoming events. See our upcoming webinars: https://www.oligotherapeutics.org/web...