Cell and Gene Therapy:

Unprecedented Opportunities, Unique Challenges

Regenerative medicine and immunotherapies offer unprecedented opportunities, with a remarkable range of potential applications in disorders affecting over 150 million patients in the United States alone. Targets for cell therapies, gene therapies, and tissue-engineered products include cardiovascular, orthopedic, neurologic, and ocular disorders, diabetes and other metabolic disorders, soft tissue injury, autoimmune diseases, and cancer. Market estimates for cell therapy, gene therapy, and tissue-engineered products are expected to reach $25 billion by 2027, with projected annual growth of 40%. In 2019, then-FDA Commissioner Dr. Scott Gottlieb predicted that the FDA will be approving 10 to 20 cell and gene therapy products a year by 2025.

Promising as these novel biologic products are, commercialization presents unique challenges throughout the development pathway. Pharma- and biotechnology-based assumptions and practices do not always translate directly to living biologic products. Specialized expertise is essential, from initial due diligence, to planning preclinical studies, developing scalable, cost-effective manufacturing, and navigating the regulatory pathway.

ADVANCED CELL & GENE THERAPY, LLC is a consulting firm specializing in development and commercialization of cell therapy, gene therapy, and tissue-engineered products. ACGT and its affiliated consultants provide invaluable and actionable guidance informed by over 120 years of combined experience in manufacturing, regulatory, quality, strategic, and device aspects of these novel products.