Insight Series Briefing: Gene and Cell Therapies

Presenter: Mike Rice, MS, MBA, Principal, Defined Health

The recent, unprecedented FDA approvals of three advanced therapeutics, in the form of 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 Defined Health’s upcoming presentation, Mike Rice of Defined Health will discuss:

• 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