ALISO VIEJO, Calif.--(BUSINESS WIRE)--As gene therapies and editing technologies rapidly advance, it is more urgent than ever to provide updates and information to the rare disease community on how these technologies can be applied across multiple diseases. Global Genes, a leading rare disease patient advocacy organization, is pleased to announce they will be publishing a multimedia series, titled Platforms of Hope: Advances in Gene Therapy and Gene Editing, throughout 2021 regarding upcoming data announcements and information on gene therapy and editing technology advances with 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.
In addition to ongoing coverage through Global Genes’ videos, online publication RARE Daily and RARECast podcast, the organization will be publishing a special report at the end of 2021 on gene therapy and gene editing innovation. This will address 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.
“With more than 7,000 rare diseases, there is an urgent need to keep the rare disease community abreast of developments in the rapidly changing fields of gene therapy and genome editing,” said P.J. Brooks, program director at the Office of Rare Diseases Research at the NCATS. “In this collaboration, NIH will help Global Genes identify the ideas, technologies and advances that have broad implications for many patients and families affected by rare diseases and provide information that could positively impact their lives and care in the future.”
“This collaboration will bring visibility into cutting-edge science at the frontier of genetic medicines and provide the rare disease community with insights into emerging technologies and therapies in development for rare diseases,” said Christian Rubio, vice president, strategic advancement at Global Genes. “It’s critically important to educate the rare disease community on these rapidly evolving events.”
For more information, visit www.globalgenes.org/media-hub.
About Global Genes®
Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf -- helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease, or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.