Monday, January 13, 2014

State of the industry briefing shows a field coming of age, ready to start benefiting patients.

The size of the crowd at the ARM briefing is a measure of the growth of the industry
It’s an auspicious start to the week when you begin your day at a conference and one of the first lines you hear from the podium is: “This industry is ready for prime time.” In this case, the words were spoken by Geoff MacKay chair of the Alliance for Regenerative Medicine (ARM) at the group’s fourth annual State of the Industry briefing at the BioTech Showcase which runs parallel with the massive JP Morgan life sciences meeting that comes to San Francisco every January.

Both gatherings aim to connect early-stage companies with investors or partners to help carry their products through to routine patients, not just those in clinical trials. MacKay, whose day job is as CEO of Organogeneis, led off with some stats to support his conclusion. He noted that regenerative medicine companies had attracted $1.3 billion in outside investment in 2013 and that there were 575 potential cell and gene therapies in the development pipeline. He also cited the nine IPOs, or Initial Public stock Offerings, this past year in the field.

MacKay also shared some data from a survey ARM conducted among 17 major pharmaceutical companies. He said that to his surprise, and mine, 100 percent had some investment in regenerative medicine. More than 60 percent were using stem cells for drug discovery, something we have written about often, and something we knew was happing at big pharma, but at even more firms, nearly 70 percent, were investing directly in cell-based therapies.

Two panel discussions followed that opening from MacKay. One focused on companies that were expected to report out late stage clinical trial data this year using various approaches including patients’ own cells, donor cells, and gene therapy to activate a person’s innate stem cells. A repeated refrain from today’s session, and from an ARM retreat I attended yesterday, has been that this late stage data is critical to moving the industry forward and to attracting investment. The second panel highlighted this with remarks from leaders of four companies that have attracted outside investment.

At CIRM we have been excited that the new year has begun with two of our grantees attracting outside investment. Capricor entered an agreement with Janssen Biotech, a division of giant Johnson & Johnson for its stem cell heart therapy. And Sangamo BioSciences announced a deal with Biogen Idec to advance its stem cell gene therapy for Beta-thalassemia.

Let’s hope the good news keeps coming.

Don Gibbons

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