Patient Advocacy Group–Led Preclinical Strategy to Assess PURA Dosage Sensitivity in Support of Gene Therapy Development
Presented May 12 at the ASGCT 2026 Annual Meeting
Download a PDF version here.
About Jack’s Tomorrow
Jack's Tomorrow is a patient advocacy organization whose mission is to fund research toward a treatment—and ultimately a cure — for PURA Syndrome.
About JAX
JAX is an independent, nonprofit biomedical research institution with nearly 3,000 employees in locations across the United States, Japan and China. Its mission is to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health. The Rare Disease Translational Center serves that mission by empowering rare disease solutions through partnership, innovation, and scaled pre-clinical pipelines to deliver targeted therapies from lab to clinic swiftly and effectively. The Center aims to provide patients with an efficient path from diagnosis to therapy, allowing them to live longer, healthier lives. For more information, please visit www.jax.org/rare.
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