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
- 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