Biotech Introduces First Gene Therapy, Beats Pharma

By Prei Dy, | March 29, 2017

Biotech beats pharma on gene therapy.  (YouTube)

Biotech beats pharma on gene therapy. (YouTube)

While it would seem that pharma has beaten biotech across any industry, several CEOs believe the otherwise in terms of "The World's First Gene Therapy."

Glybera, the world's first gene therapy, received marketing authorization from the European Medicines Agency in 2012. Developed by uniQure, Glybera was made as a one-off cure for a rare hereditary acute pancreatitis disease called the lipoprotein lipase deficiency. The therapy managed to decrease the blood fat levels all nearly all patients on the clinical trial, an evidence that proved durable over a long period.

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Successfully therapies like the Glybera are usually snapped by pharmas even before they reach the market, but biotech did it this time. Although biotech may seem to be outmatched by pharma in almost every aspect, innovation is an exception, Labiotech pointed.

"Biotech is typically first in highly innovative fields," Matt Kapusta, uniQure CEO, said. "There's a lot of risk and investment that goes into cutting edge technology, but that's the nature of the biotech industry."

Pharmaceutical giants with aging pipelines are always aggressive on discovering something new, spending a staggering $87 billion on research and development in 2016 alone. Now, pharma is starting to tap to biotech to transform its portfolio and seek innovative ways to new indications.

On the other hand, biotech also needs help from the cash-rich pharma to successfully push its therapies to Phase III as it costs billions to develop a drug, something that a small biotech with negative cash flow could not afford.

"Typically, pharma is more earnings-conscious and thinks that if a biotech can prove an innovative technology works, then the company will collaborate or acquire its way in," Kapusta said. Biotech are usually well-positioned to team up with pharma after it proves its lead candidate in Phase II.

"For a long time, gene therapy and rare disease were of no interest to pharma," Frederic Revah, Genethon CEO, said. He also added that gene therapy has paved way for a broader market. And unlike common diseases that affect a larger patient population, gene therapy paved way for a broader market, although longer clinical regulatory pathways and bigger investment are needed.

"It's kind of a beauty contest, but the designation has proven to be very stimulatory... it means you get FDA love, and who doesn't want that?" Timothy Cote, CEO of consulting company Cote Orphan, said.

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