Tim Mustill, Principal at Astrocyte Consulting, outlines the challenges of gene and cell therapy development and clinical use – and how medcomms professionals may be able to help.

Recorded 4 October 2017 at a MedComms Networking event in Oxford. Produced by NetworkPharma.tv

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ABSTRACT:

The advent of gene and cell therapies has been an exciting development, representing a genuine paradigm shift in patient care. These therapies have huge potential for treating and curing those with hitherto intractable conditions. However, they also carry big risks both clinically and commercially.

Gene therapy is based on a simple concept: that genetically determined or mediated dysfunction in cells can be cured by the insertion of a functioning gene. The main strategies for achieving this are gene replacement, gene addition, knockdown of mutant genes and gene editing. The process is far from simple, involving at least seven steps, which start with identifying the gene defect, creating a replacement sequence and finding a vector or delivery method. Once in the body, the gene must be inserted into the genome of the target cells and its expression facilitated. Each step has many options and creates many challenges. There have have been pitfalls and setbacks in clinical trials. The technical and commercial challenges of upscaling are also considerable. The risk of off-target effects is high, for example; patients must attend dedicated centres that are few and far between, and the manufacture of lentiviral vectors is time-consuming and expensive. On the positive side, while cancer and rare diseases are still dominant, multiple indications are now being addressed in gene therapy clinical trials across diverse therapy areas. Many opportunities exist for medcomms professionals to support the development of these products throughout their lifecycles.

Further reading: ICER Gene Therapy White Paper at https://icer-review.org/wp-content/uploads/2017/03/ICER-Gene-Therapy-White-Paper-030317.pdf

Written by Penny Gray, Freelance Medical Writer

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We are building a library of free webcasts, like this one, for the global MedComms Community and others at http://www.networkpharma.tv and we’d welcome your suggestions for new topics and speakers.

Full details of this MedComms Networking event are at http://medcommsnetworking.com/event_041017.html

Tim’s presentation (PDF format) is at http://medcommsnetworking.com/presentations/mustill_041017.pdf

Tim’s Linkedin page is at https://www.linkedin.com/in/timmustillstrategist/

More about Astrocyte Consulting can be found at http://www.astrocyte.co.uk

Filming and technical direction by Mario Crispino, Freelance Cameraman & Editor

[For the avoidance of doubt: this video is intended to be freely accessible to all. Please feel free to share and use however you like. Cheers Peter Llewellyn, Director NetworkPharma Ltd and Founder of the MedComms Networking Community activity at http://www.medcommsnetworking.com]