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What’s next is Cell & Gene Therapy?

What’s next is Cell & Gene Therapy?
May 2, 2019 SueCarr

It’s no longer a secret that Cell & Gene Therapy is now considered the future of healthcare. That said, the number of market approvals has only just about hit double figures, each of which are still experiencing their own commercialisation hurdles that we are learning from every day.

Nonetheless we have completed the cycle 10 times over, we know we can do it and we have an infinite amount of gratitude to express to the animals, people and organisations involved in laying down the foundations for which we can build an infrastructure to cure the world forever.

The big question is – what happens next?

In this article, Head of Boyds’ Dublin office, Siobhan Gaynor draws on her wealth of experience in cell and gene therapy and gives her views on this huge transformation in medicine.  

The exciting recent developments in cell and gene therapy have yielded some fantastic medical advances. There are now stories of patients getting “cured” of rare diseases, responding to treatment at previously unheard-of rates and seeing a dramatic improvement in the quality of their lives. These recent advances in the cell and gene therapy (CGT) field are great examples of why I remain excited and privileged to play my part.

Beyond the huge potential for patients, the challenges that lay ahead are in embedding these therapies into medical practice. Continuing to innovate and improve patient outcomes and access are worth reflecting on as well.

In terms of medicine’s evolution, this new paradigm does require a changing model for healthcare providers, regulators, and payors, as well as the pharmaceutical/biotechnology industry and academia.  This is because these medicines can be complex to deliver to patients, are being developed to treat smaller and smaller patient numbers, are presenting significant manufacturing and supply challenges and, of course, these innovations do come with high financial costs, which must also be addressed.

Top Issues to be addressed in CGT evolution:

  • Manufacturing bottlenecks are emerging in terms of capacity and expertise for soon-to-be-launched CGT products. It is necessary to recognise that these new products require a much earlier investment and dialogue about large scale manufacturing than for more traditional medicines.  This requires a change in mindset by industry as well as more collaborations with academia and others around process development.
  • Delivery of CGT therapies to patients, in many cases, requires local adaptions and/or specialist expertise. This speaks to the need to develop centres of excellence in hospitals with associated infrastructure and training, in parallel with investment in research to facilitate easier delivery of these medicines.
  • Patient organisations have been the inspiration, impetus and origin for so many of these therapies, but their voices are being diluted as we arrive at the point of delivery. Relevant patient organisations should be consulted at every stage of medicines development by industry, academia, regulators and payors. In my direct experience they are the ones that know and understand their diseases and needs best.
  • Affordability of these new medicines will naturally be debated in many jurisdictions’ over the coming years. The question is, can we afford to provide all these new medicines as well as to continue to improve health outcomes generally? In order to capitalise on the many decades of public and private funding that has allowed us to deliver these potentially transformative medicines, we need to have serious debates on what we want from our health system and where our focus on science spending should lie.
  • Consultative approaches by regulators, orphan legislation, as well as some fast-tracked mechanisms have resulted in multiple approvals of CGTs as well as continued investment in this space, in the EU and US. However, as the volume of new therapies in development gathers pace, it is clear that the regulatory authorities also need to continually innovate, collaborate with developers and upskill in order to continue to contribute to the availability of new lifesaving medicines.  Additionally, I see the need for more joined up thinking and earlier dialogue between the medicine’s regulators and the public payors and/or private insurers.

Finally, it is worthwhile remembering that it takes on average 12-15 years to develop a drug, so a continued focus on investment in early stage research as well as efforts to shorten this timeline should help ensure that science will continue to deliver for patients.

The future of cell and gene therapy is bright, but we must make it brighter by continuing to innovate, collaborate, educate and communicate.


Siobhan has over 25 years’ experience in a wide variety of senior roles in clinical and translational health research, working in major pharmaceutical companies, biotechnology start-ups and academia.
She has held influential leadership roles at Genable, a private venture capital funded, ophthalmic gene therapy, development stage company. During her time here she successfully developed and executed the management, strategic and operational plans ahead of a successful sale to Spark Therapeutics between 2014-2016.
Siobhan then went on to become Spark’s global drug development lead for ocular gene therapy programs. She later became co-founder of Spark Europe’s foundation and development team and became their EU sponsor for MAA submission for Luxturna, the world’s first gene therapy to treat an inherited retinal disease.
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