Realizing the ambition of Star Wars Medicine: widely available gene therapies that deliver outcomes

Written by Gavin Jones, Global Advisor, Rare Diseases on Wednesday 28th July 2021

Welcome to our third Thoughts & Outcomes Theme of 2021, where we will be revisiting the topic of gene therapy, the promise of which remains as compelling as ever. Throughout this theme we will be sharing blog pieces, articles, and a whitepaper. Keep an eye out for a panel webinar in September discussing the topic of gene therapy.

Despite some well-reported difficulties, which must be very challenging for rare communities, gene therapy continues to offer the potential to transform patients’ lives. More therapies are working their way through clinical development to patients but there is a long road ahead before gene therapies are established for many conditions and are considered as a standard of care. A lot of the conversations to date have understandably been focused on reimbursement, but we want to explore other aspects of realizing the ambition of gene therapy that will enable effective uptake. One particular area of focus for us will be educating and supporting informed consultations and decision-making to help deliver the promised outcomes.

To kick off this theme, I recently had the pleasure of speaking with Luke Pembroke, Creative Director of Haemnet, about his journey towards gene therapy and the film he created to capture it. I would recommend listening to the interview to get a sense of the magnitude of decision-making that patients and caregivers face as they consider the option of gene therapy. Please also take a look at Luke’s short film, which is both beautiful and inspiring.

Watch Lukes "A haemophilia gene therapy journey" video for the Rare Reach Festival HERE

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