There are unique challenges associated with gene therapy trials especially in those indications which are rare diseases, such as small patient numbers, lack of detailed knowledge of the disease progression, and definition of suitable endpoints.
This white paper explores how the analysis of real-world data can provide insight and help overcome these challenges. It also discusses some of the limitations which reduce their acceptance by the regulatory authorities.
This White Paper will provide you with:
- Key understanding of Gene Therapy Trials
- Examples of how Real-World Data supports Gene Therapy studies
- Knowledge of the Regulatory Requirements for Gene Therapies
- Benefits of CRISPR/CAS9 Technology, and how it has impacted the industry
- The limitations of a Randomized Clinical Trial, including historical control data
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