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The Neurofibromatosis Therapeutic Acceleration Program (NTAP) is now accepting applications for the Francis S. Collins Scholars Program in Neurofibromatosis Clinical and Translational Research. The Collins Scholars program brings together a community of exceptionally well-trained clinician scientists who will be leaders in the field of NF1 research.
WORKSHOP RECAP: The Growing Promise of Gene Therapy Approaches to Rare Diseases
On Aug 20-21, 2018 the National Center for Advancing Translational Sciences (NCATS) and FDA’s Center for Biologics Evaluation and Research (CBER) hosted a workshop on gene therapy in Bethesda, MD. Titled ‘The Growing Promise of Gene Therapy Approaches to Rare Diseases,’ the workshop was attended by experts from academia and industry, NIH and FDA staff, representatives from several rare disease foundations, and other stakeholders. The two-day event was filled with presentations and panel discussions on advancements and challenges in preclinical and clinical development of gene therapy products, vector manufacturing processes, public-private partnerships including with research foundations, business models, patient access especially in very low prevalence diseases, and reimbursement models.
The Pershing Square Sohn Cancer Research Alliance (PSSCRA) will open for applications for its 2019 Pershing Square Sohn Prize for Young Investigators in Cancer Research on October 1st, 2018. At least six New York City-area-based scientists will each be awarded $200,000/year – for up to three years – to empower them to pursue groundbreaking research.
The Children’s Tumor Foundation (CTF) is pleased to announce the initiation of a research program in gene-based therapeutic approaches for the treatment of neurofibromatosis type 1 (NF1). This initiative begins through this request for applications (RFA), with the first goal to fund two proof-of-principle studies in NF1 gene editing. Funding for each study will be $240,000 total (inclusive of 10% indirect costs) for a duration of two years.