Defined Health Insight Series Briefing: Gene And Cell Therapies – It’s Show Time!

Presenter:

  • Michael C. Rice, MS, MBA, Principal, Defined Health

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 webinar, 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 suchcomplexities 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