Gene & Cell Therapies 

The recent, unprecedented FDA approvals of three advanced therapeutics, or gene and cell therapies based on viral DNA transfer platforms, signify that a therapeutic paradigm change is upon us. 

  • Novartis’  Kymriah , an ex-vivo, lentiviral transduced T-Cell for R/R B-Cell Acute Lymphoblastic Leukemia
  • Kite/Gilead’s  Yescarta , an ex-vivo, retrovital transduced T-Cell for R/R Large B-Cell Lymphoma
  • Spark’s  Luxturna , an adenoassociated virus (AAV) based, in vivo therapy for biallelic RPE65 mutation-associated retinal dystrophy

All three of these programs demonstrated dramatic efficacy in previously hopeless clinical situations. Such breakthroughs are based on decades of research and are likely to become a mainstay in treating, if not, curing, many devastating and intractable illnesses. Paving the way, FDA Commissioner Scott Gottlieb recently indicated that the agency is establishing a policy framework for the evaluation and review of a busy queue of advanced therapeutics expecting to seek review over the next year.

In this white paper,   Mike Rice of Defined Health discusses:

  • Investment and deal trends driving the recent advances in gene transfer and cell processing that have enabled the realization of advanced therapies 
  • Implications to biopharma business models, and changes needed to make such curative intent treatments with due respect to such complexities as individual manufacturing, single or short course administration, etc.
  • Market access and risk-sharing strategies for highly value-priced therapies that currently have minimal data from small patient study cohorts with limited longitudinal evidence of durable therapeutic benefit




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WHITE PAPER AUTHOR:

Company team

Mike Rice, MS, MBA, Principal

Defined Health

Mike leads Defined Health’s Gene and Cell Therapeutics and Rare Diseases practices. He also co-heads the oncology practice focusing on hematologic malignancies and genetically defined cancers. Prior to Defined Health, Mike gained over a decade of experience creating new biotech ventures from academic inventions pertaining to nucleic acids and cellular platforms applied across monogenetic diseases and oncology. Industry positions involved translational research, strategic planning, technology transfer, finance and business development.

Mike holds an MBA, with a concentration in Biotechnology from the Alfred Lerner School of Business and Economics, at the University of Delaware, an MS in Molecular Pharmacology from Thomas Jefferson University and a Bachelor of Science degree in Biology from the University of Delaware. Mike studied the molecular basis of cancer at the Kimmel Cancer Institute and is recognized for his extensive intellectual property and publication portfolio pertaining to cancer genetics, recombinational DNA repair, gene therapy, diagnostics, and agricultural trait improvement. 

Mike is an inaugural member of the American Society of Gene and Cell Therapy (ASGCT), as well as a member of the Society for Immunotherapy of Cancer (SITC), the American Society of Clinical Oncology (ASCO), the American Society of Hematology (ASH), the Licensing Executives Society (LES), and the American Heart Association (AHA). Learn More

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