Regulatory Strategy for Gene Therapy: Challenges and Opportunities

Description: The rapidly changing field of gene therapy promises several innovative treatments for rare diseases. Given that approximately 80 percent of the 7,000 known rare diseases are caused by a single-gene defect, there has been increased research in the development of cell and gene therapies to treat rare diseases. However, a number of challenges hinder these efforts, including the high cost of bringing these drugs to market, unique manufacturing and supply chain challenges, pricing and reimbursement, and our current limited understanding of disease pathology and progression. While these challenges may seem common across other drug markets, in the case of rare diseases, these complexities are exacerbated by limited patient populations.

This presentation, will address the following questions and overcome those hurdles by developing the best suited regulatory roadmap to product commercialization.

- What are the key challenges faced by companies developing gene therapy products for rare diseases

- What would it take and how long would it take to bring the product from research to market

- What are the regulatory pathways that can help expedite the drug development processes

- How should we develop and organize various teams (e.g., regulatory, clinical, quality, CMC etc.) to be more collaborative and efficient