Gene Therapy for Rare Diseases

There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes.
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OTHER ON-DEMAND WEBINARS

High-Dimensional Human Immune Profiling Simplified

Workcast

Immune profiling of PBMC and peripheral whole blood is an essential method for quantifying changes in immune cell populations in autoimmune disease, chronic inflammation, infectious disease and cancer.
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Regulatory Strategies for the Analytical Development of New Biological Entities and Biosimilars

xtalks

The webinar will focus on the relationship between analytics of biotherapeutic products and the interpretation of data to build and support a solid regulatory Chemistry, Manufacturing and Control (CMC) strategy. The importance of data interpretation is illustrated in the development of biosimilars and new biological entities, as well as in regulatory specifications regarding biotherapeutic products.
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Biotechnology Risk Assessment Research Grants FY 2023 RFA Technical Assistance

The Biotechnology Risk Assessment Research Grants (BRAG) program supports the generation of new information that will assist federal regulatory agencies in making science-based decisions about the environmental effects of introducing genetically engineered organisms.
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Key Challenges and Opportunities in the Cell Culture Process Scale Up

Cell therapy products have amazing potential to treat a wide variety of diseases and vast numbers of patients globally. However, establishing a scalable manufacturing technology that can reliably reproduce the yield and quality of derived products generated from small-scale R&D methods at larger scales sufficient for commercial manufacturing can pose many challenges.
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