As gene therapies and editing technologies rapidly advance, providing updates to the rare disease community on how these technologies can be used across multiple diseases is an important part of making these therapies accessible. Platforms of Hope: Advances in Gene Therapy and Gene Editing, is a multimedia approach to ensuring this information is communicated in a digestible and shareable way. Featuring thought leaders from the National Center for Advancing Translational Sciences (NCATS), the NIH Common Fund’s Somatic Cell Genome Editing (SCGE) program and other leading voices in these fields, this series hopes to empower patients, advocates, and other rare disease stakeholders to turn these discoveries into beneficial medicines.
In addition to ongoing coverage through videos, RARE Daily publications, and RARECast podcasts, you can check out a special report at the end of 2021 on gene therapy and gene editing innovation! This addresses a wide range of topics, with a focus on efforts to accelerate the translation of discoveries into genetic medicines that benefit patients with rare diseases. If you’re interested in learning more about this initiative, check out our Press Release for more information.
Photo: Moebius Syndrome Foundation