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.

Thank you to Pfizer, Inc.,  Bluebird, and Novartis Gene Therapies for your sponsorship of this initiative.

platforms of hop sponsors

Photo: Moebius Syndrome Foundation

Platforms of Hope: RARE Daily

Article #1                                                                      Article #2                                                                 Article #3

January 13, 2021
Study Suggests Genetic Editing Could Correct Rare Disease that Causes Rapid Aging
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February 12, 2021
Patients’ Cells Offer Insight into New Gene Therapy Approach to Rare Eye Disease
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March 3, 2021
Study Shows Potential for Lipid Nanonparticles to Deliver Gene Editing Therapy to Liver
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Article #4                                                                      Article #5                                                                 Article #6

April 20, 2021
The Making of a Gene Therapy for an Ultra-Rare Disease
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May 11, 2021
Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency
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June 15, 2021
Researchers Use Base Editing to Turn Pathogenic Gene Benign in Mice with SCD
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Platforms of Hope: RARECast

RARECast #1                                                        RARECast#2                                                                 RARECast#3

February 5, 2021
Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases
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February 19, 2021
Moving Beyond Viral Vectors for Gene Therapies
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February 26, 2021
The Promise of Gene-Based Therapies for Neurodegenerative Conditions
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RARECast #4                                                        RARECast#5                                                                 RARECast#6

March 12, 2021
Realizing the Potential of CRISPR Gene Editing
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March 26, 2021
A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases
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April 2, 2021
Choosing the Right Viral Vector for a Gene Therapy
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RARECast #7

April 30, 2021
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
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