Finding solutions for affordable and accessible cell and gene therapies

The California Institute for Regenerative Medicine (CIRM) is not alone in seeking a way to make cell and gene therapies more affordable and accessible.

The Innovative Genomics Institute (IGI) just completed a year-long effort to consider how treatments could be made more accessible. This effort involved creating a task force comprised of 30 participants to develop options. The participants hail from diverse fields including genome engineering, economics, manufacturing, venture capital, intellectual property and more.

The task force’s recommendations were published recently in a report titled: Making Genetic Therapies Affordable and Accessible.

The report describes the gene therapy landscape and discusses research, development and manufacturing factors that impact the cost of treatments. The authors also suggest ways to reduce these costs and include discussion of how government funding in general, and CIRM funding specifically, can augment some of these development costs.

One specific model proposed by the authors involves a development partnership between academic researchers, non-profit medical research organizations and public benefit corporations. Through this model, the aim would be to significantly reduce the costs of therapy delivery. The report also includes a set of policy recommendations deemed important supporting this outcome.

The progress made since 2004, when stem cell research was still in its nascent stages, is truly remarkable. Within a relatively brief period, the focus has shifted from the exploration of stem cell potential to the development of therapies for clinical trials.
Now, we are looking to the next stage of this journey to increase access to the availability of innovative treatments for patients.

IGI’s work and a broader body of knowledge will be taken under consideration by CIRM’s Access and Affordability Working Group as they seek ways to enable access to CIRM-funded treatments for all Californians.

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genomics
gene therapy
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