ARM State of the Industry Briefing gives clear evidence that regen med/advanced therapies are the next big thing in biotech

January 12, 2015 Erin Righetti

BTS_1.12.15-EdLanphierARMBiotech Showcase™ 2015 kicked off today with the Alliance for Regenerative Medicine’s (ARM) Regenerative Medicine and Advanced Therapies State of the Industry Briefing. Edward Lanphier, President and CEO of Sangamo BioScience and the new Chair of the Alliance for Regenerative Medicine gave introductory remarks that indicated that the excitement for gene therapies will continue to grow in 2015.

Regenerative medicine is a fascinating field and one of the fastest growing areas in biotech. The main therapeutic areas being developed fall under three categories: Gene and gene modified cell therapies, cell therapies, and tissue engineering. When you put all the regenerative medicine/advanced therapies in the blender, said Lanphier, what you have are technologies that allow us to think about the next paradigm of disease; not just treating disease, but curing disease.

According to Lanphier, the industry’s growth is robust. There are currently over 500 companies worldwide in the regenerative medicine and advanced therapies space, and over 200 of those global companies are ARM members. The breakdown by focus area is: 340 are focused in cell therapy, 180 in tissue engineering, and 140 in gene therapy, said Lanphier. For financing in the regen med/advanced therapies space, USD 6.3 billion was raised in 2014, as compared to USD 3 billion raised in 2013, for a year-over-year growth of 112%. From a priorities standpoint, regen med is really a bottoms-up industry focused on the prioritization process.

There are now over 40 approved products coming from these technologies, and over 650 ongoing clinical trials going on in the space. Lanphier expects to see significant milestones in 2015 in Phase II and III trials, with the formation of significant partnerships. Financing and validation are critically important to the industry, and the financing window appears to be opening up for advanced therapies.

Overall, 2014 was a big year, beginning with the first gene therapy product approved in Europe by UniQure. The company raised USD 91.8M in Feb 2014, said Lanphier. The IPO class of 2014 continues to perform; this is good news and investors want more. With big pharma value placed on outcomes, the maturation of regenerative medicine and advanced therapy pipelines means more corporate partnerships in 2015.ARM_Logo

As well, product development has taken off and regenerative medicine and cell therapy products are truly a global technology and have rapidly grown into an area of global development. The perception based on data is that gene therapy is not just a way of developing novel treatments but rather curative outcomes. And there is palpable excitement among the investor set over the potential.

“This industry is making a big mark globally,” said Lanphier. In particular, there has been an explosion of growth in advanced therapies in Japan. “Japan is really picking up the ball and running with it. They have a national mission to create an environment for accelerating conditional approvals, thus allowing companies to get through regulatory hurdles,” said Lanphier.

Lanphier stated that, despite the obvious value, there can be resistance to regenerative medicine and advanced therapies due to the challenging regulatory environment. “This is a big need for the future,” reiterated Lanphier.

Lanphier’s remarks were followed by the panel entitled “Gene therapy and genome editing: Defining the clinical and commercial requirements for success” that featured experts in the space in a discussion looking forward to the expected clinical, regulatory and commercial inflection points for the sector in 2015 and beyond. Speakers included Katrine Bosley – CEO, Editas Medicine; Larry Bullock – Chief Financial Officer, AGTC; Thomas Chalberg – Founder and CEO, Avalanche Biotechnologies; Steven Paul – CEO, Voyager Therapeutics; Josh Schimmer – Managing Director and Senior Research Analyst, Piper Jaffray; Jeffrey Walsh – COO, bluebird bio; and Kristina Zsebo – CEO, Celladon.

The ARM special briefing wrapped up with the panel of industry CEOs and senior executives projecting milestones and progress in the space for 2015. The panel, “Dose, delivery, data: Understanding the three D’s of Advanced Therapy drug development” featured Tony Fiorino – CEO, BrainStorm Cell Therapeutics; Adam Gridley – President and CEO, Histogenics; Paul Laikind – President and CEO, ViaCyte, Inc; C. Randal Mills – President and CEO, California Institute for Regenerative Medicine (CIRM); Jay Siegel – Chief Biotechnology Officer and Head, Scientific Strategy and Policy, Johnson & Johnson; and Paul Wotton – President and CEO, Ocata Therapeutics.

Look for coverage from the ARM Briefing on the Biotech Showcases web site, or online at partnering360® . And for additional coverage on Lanphier’s perspective, see The Bio Report’s interview ‘Moving from Chronic Therapies to Cures.’

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