CALBIO15: Gene Therapy in "Second Honeymoon" Phase as Companies, Investors Swarm

Published: Mar 04, 2015

CALBIO2015 Gene Therapy in
March 2, 2015
By Riley McDermid, Breaking News Sr. Editor

Gene therapy is undergoing a “second honeymoon” with investors, large and small, though the infrastructure to produce new products may be having a hard time keeping up with scientific advances currently underway worldwide, said experts at a panel in San Francisco on Monday.

The panel, “A Second Honeymoon for Gene Therapy: As Investors, Venture Capitalists and Pharma Fall Back In Love with the Space,” took place at CALBIO. With over 100 California life science companies and investors in attendance, the conference has become a favorite for discussions involving opportunities and scale in the rapidly innovating world of science.

Joan M. Robbins, senior director for cancer biology, Tocagen Inc.; Edward Lanphier, chief executive officer, Sangamo BioSciences ; Sasha Kamb, senior vice president of research, Amgen . The panel was moderated by Barrie Carter, vice president at Vector Biology, BioMarin Pharmaceutical Inc. .

A white-hot biotech market and new technology has sparked a renewed confidence from investors, venture capitalists and Big Pharma in doubling down on gene therapy. In the last year, multiple gene therapy companies have completed successful IPOs, including Avalanche Biotechnologies and Bluebird Bio , while the European Union’s approval of the first gene therapy in the western world, UniQure ’s Glybera, has only added to the excitement.

Panelists said Monday that they certainly were taking a closer look at the space, though they didn’t necessarily know if it could maintain the same level of investor interest as 2014. But that doesn’t mean certain companies won’t immediately attract attention, particularly in the gene therapy space, said participants. Amgen in particular is looking for a partner with that sort of background or existing pipeline, said Kamb.

“We're very interested in gene therapies. Many obstacles are now being overcome,” said Kamb. “The future is now, or at least just around the corner, for gene therapy.”

Amgen doesn't directly have a gene therapy investment currently, but they are interested in it, said Kamb.

Other panelists said the regulatory environment appears to finally catching up to science, and that has made them more willing to put money into the space.

"The regulatory environment for gene therapy has definitely improved. It's more defined. It’s more informed," said Lanphier. “Now we are hitting a physical facilities/infrastructure roadblock versus a technical roadblock.”

Robbins, with Tocagen, agreed, saying finding the right companies and timing it for the right market always remains a challenge, if one that can have a significant reward when done correctly.

“The key to gene therapy is likely pairing the right virus with the right payload for the right indication,” said Robbins, who said Tocagen’s own investment in a brain cancer therapeutic company has raised over $100 million in private funding so far. “The more success that we all have, the more manufacturing infrastructure for gene therapy can be built up.”

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