A reflection of the 11th Business of Regenerative Medicine

It was a sunny Philadelphia that greeted the 11th Business of Regenerative Medicine Symposium. Hosted at the University of Pennsylvania’s Similow Center for Translational Research, the two-day symposium revealed the broad range of insights into innovation, clinical translation and entrepreneurship within the regenerative medicine industry.

The event, including informative presentations (covering experience through to commercialisation), discussions of current trends, practical advice and the sharing of cutting-edge science, was a great success. For us at CCRM Australia, this experience, mesmerised by the science of regenerative medicine and awed by the bold approaches undertaken by physicians, engineers and scientists who are advancing the field – inspired us.

Major takeaways from the symposium

Kicking off with an opening address by the Food and Drug Administration (FDA), the symposium appropriately drew focus to the recent approvals of Novartis’ Kymriah (the first CAR-T cell therapy to receive FDA approval) and Kite Pharma’s Yescarta (a close second). The event also highlighted the University of Pennsylvania’s critical role in the development of Kymriah.

Amongst the many topics covered, there were a number that left strong impressions. The list includes:

  1. Reni Benjamin’s analysis of the current trends in the US Investment landscape and advice on raising capital.
  2. An explanation of the Multi-Luminance Mobility Test by Katherine High from Spark Therapeutics.
  3. Arnold Caplan’s passionate pitch to rename mesenchymal stem cells to medicinal signalling cells.
  4. A bioengineered device presented by Dan Huh that simulates a blinking eye.

However, as the Symposium continued, I found myself continually thinking about the Kymriah journey, presented by Pascal Touchon and Bruce Levin of both Novartis and University of Pennsylvania. This inspiring journey lead to a closer analysis on developmental pipeline for regenerative medicine therapies and what it means for other therapies in the future.

Unveiling the developmental pipeline for regenerative medicine therapies

While literature in the field estimates varying lengths of time from the point of clinical development to regulatory approval (ranging anywhere from 8 to 12 years), Kymriah’s record of five years to approval has, without a doubt, captured the interests of scientists, biopharmas and investors.

Over the next two days, possible reasons to explain this phenomenon became clear— great science begets efficacy and without great science, there would be no product. From that baseline, I would list my takeaway key messages to be the need to have 1) a multidisciplinary team, 2) good data, 3) a focus on manufacturing and 4) a partnership with the regulator.

Bringing a therapy or device to market is the sum total of basic research, clinical development, process engineering, commercial expertise, legal advice, capital raising and partnerships. This developmental pipeline is so difficult that a number of books have been written in the attempt of decoding this arduous effort. A multidisciplinary approach, perhaps akin to a ‘by design’ approach, presents many benefits, especially when it comes to problem solving.

It has been impressed frequently that bio-entrepreneurs have achieved success ‘by misadventure’. If our aim is increase the speed at which products enter the market in this highly regulated industry, (where we do not have the luxury of continuous product improvement as in the automobile industry), the formation of a multidisciplinary team at the onset may result in more benefits than cost.

Perhaps we see a reflection of this multidisciplinary collaboration in the form of disease teams and alpha clinics or through facilitation of public-private partnerships. A shortened time to market also means less time spent for process development and manufacturing to address the tenets of quality, safety, scalability, preservation and shipment.

Coupled with potentially low patient accruals for autologous therapies, having robust data is ever more crucial for characterisation, regulatory approval and subsequent compliance, attracting capital investment and potential problem solving in the future.

Lastly, the importance of a partnership with the regulator became more evident from an insightful presentation by Spark Therapeutics. Our lofty goals of regeneration and restoring function sometimes present challenges in determining the most appropriate endpoints, in finding distinct differentiation and in the protection of our value proposition(s) against competitors that develop large and small molecule therapies.

As a consequence, can there be an appropriate measure of functional testing that potentially addresses the above? The Multi-Luminance Mobility Test by Spark Therapeutics for their Luxturna gene therapy is an enlightening example of the possibilities that could arise and I highly encourage everyone to read.

A future in Asia Pacific

In addition to its lecture-style program, the Business of Regenerative Medicine Symposium further provided opportunities to network with peers and start-ups from the major hubs of California and Boston. No doubt there will be discussions leading to collaborations in the future.

However, it must also be noted that one cannot help but wonder if we will see rising numbers of biotechnology companies, spinouts and investments from and into the Asia Pacific region. Might there be a need to host a localised Business of Regenerative Medicine even? Given that the Asia Pacific market is more diverse in its regulations, business culture, and resource levels, I think it is worthwhile to explore this possibility.