The goal of cell and gene therapies (CGTs) is to treat or prevent a disease through genetic modification and is often intended to treat, modify, reverse, or cure a life-threatening disease. This involves modification of genetic information in living cells to address a mutated gene, either by introducing a correct version of the target gene, or by blocking expression of the dysfunctional gene.
As a group, CGTs are referred to as ‘advanced therapeutics’ based on EMA’s advanced therapy medicinal products (ATMP) regulation in 2007, and the FDA’s regenerative medicine advanced therapy (RMAT) pathway in 2016.
While the term CGTs are often referred to interchangeably and the definitions can get murky, in this webinar, we focus on cell vs gene therapies as seen in this figure:
Cell therapies (on the left of the figure above) refer to the genetic modification of cells ex vivo, that is outside of the body. Cells are extracted from a patient, gene transfer happens into the cells in a lab, and the cells are then reintroduced back into the body. This is known as autologous cell therapy. More recent ex vivo approaches have involved allogeneic cells, which are genetically-modified donated cells being cultured and transplanted to the patient.
Gene therapy (on the right of figure above) involves the in vivo delivery of genes directly to cells inside of the body.
How many cell and gene therapies are in development?
At the time of recording this webinar (November 2022), there are over 20 CGTs in the market. But with approximately 427 unique companies developing new candidates, and over an astounding 2,000 CGTs in development, this will become a very crowded space over the next decade.
Ex vivo delivery techniques are being used in roughly 73% of CGTs. In the use of ex vivo techniques, CAR-T cell therapies are in the lead, with over 98% of CAR-T cell therapies in development for cancer indications.
Most of the gene therapy drugs using in vivo delivery techniques also target cancer. Other ‘popular’ target therapy areas include central nervous system disorders, genetic disorders, and ophthalmic diseases.
Navigating the cell and gene therapy market access landscape
The webinar explores the cell and gene therapy landscape, highlighting key market access challenges, and concludes with considerations for optimizing patient access. This includes:
A summary of the evolving treatment landscape in cell and gene therapies
An overview of the key challenges to overcome for achieving optimal access, including: – Demonstrating long-term benefit and managing data collection uncertainties – Managing complexities around small patient pools – Affordability of ‘one-off’ high-cost treatments paid over a short duration – Supporting value differentiation against highly competitive pipelines
Practical considerations on overcoming market access challenges and key barriers for cell and gene therapies.
Supporting your policy, access, value, and evidence needs for cell and gene therapies
The strategic importance of policy, access, value, and evidence for cell and gene therapies is increasing – and the need for an integrated 360 approach is becoming ever clearer. Our dedicated experts can partner with you to explore current trends and challenges and help you understand how you can optimize access for your assets.
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