Ceregene: Catching an updraft in gene therapy
(UPDATED: See below.)
Ceregene, a San Diego biotech at work on a gene therapy for Parkinson’s disease, has so far raised $28 million in a third funding round and last week struck a development partnership with Genzyme that resulted in a $25 million up-front payment and potential payments of another $125 million plus royalties.
Those are some surprisingly large numbers for gene therapy, the experimental practice of inserting new genes into the human body in hopes that their activity will make up for a defective or malfunctioning natural gene. The technique once served as a poster child for biotechnology’s promise of curing genetic disease, but crashed and burned when early efforts failed or, in a few tragic cases, proved harmful to patients. One infamous trial involving a rare genetic disease led to the 1999 death of 18-year-old Jesse Gelsinger, after which interest in the field dropped precipitously.
Now enthusiasm for gene therapy may once again — tentatively, at least — be on the upswing. Ceregene’s focus lies in genes that can deliver so-called neurotrophic factors, which are naturally occuring proteins that protect brain, spinal and nerve cells against damage, prevent programmed cell death, and stimulate the growth of new neurons.
While researchers have long considered neurotrophic factors a possible way to treat degenerative neural diseases such as Parkinson’s disease and Alzheimer’s disease, the proteins themselves don’t make promising drugs — largely because they’re too large to cross the blood-brain barrier. Some researchers have experimented with delivering similar proteins directly into the brain via invasive shunts or catheters, but the results have been unimpressive and the cost and difficulty of the procedure would likely limit its widespread use in any case.
Ceregene’s technology involves adeno-associated viruses that have been modified to carry genes for particular neurotrophic factors and disabled from reproducing naturally. These viruses are designed to carry the genes into at-risk cells — say, dopamine-producing neurons in Parkinson’s patients — and then “install” the carried gene into cellular DNA, where the cell’s own natural machinery will activate the gene and begin to produce neurotrophic factors.
In an early-stage trial involving just 12 Parkinson’s patients, administration of Ceregene’s gene therapy CERE-120 was associated with a 36 percent reduction in symptoms 12 months after the gene-loaded virus was injected into the volunteers’ brains. That trial didn’t have the most rigorous controls necessary to protect against investigator bias and placebo effect, so it’s impossible to draw too many conclusions from it. Ceregene is currently at work on a 51-patient follow-up trial that may produce data by the fall of 2008.
The promising results still intrigued Genzyme, an early pioneer in gene therapy for cystic fibrosis, who two years earlier had bought out much of the gene-therapy business of the struggling biotech Avigen, which also has a gene-therapy treatment for Parkinson’s disease.
Last week, Genzyme agreed to pay Ceregene 50 percent of the late-stage development costs for CERE-120 plus up to $150 million in cash in exchange for all rights to the treatment outside the U.S. and Canada. That’s a fairly hefty sum for a treatment that hasn’t even completed mid-stage trials and which also depends on such a relatively untested technique as gene therapy. Genzyme has other irons in the gene-therapy fire as well; today, Applied Genetic Technologies announced that it received $2 million from the big biotech as a milestone payment for its development of a gene therapy for a particular form of blindness.
Meanwhile, Ceregene has also raised $28.1 million in an open third funding round, VentureWire reports (subscription required). Investors in the round include Investor Growth Capital, Alta Partners, California Technology Ventures, Hamilton BioVentures, MPM Capital and Cell Genesys, Ceregene’s former corporate parent.
UPDATE: Added MPM Capital to the investors list, per Ceregene CEO Jeff Ostrove’s comment.
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About the Author, David P. Hamilton
David Hamilton has been writing for VentureBeat LifeScience since April 2007. He formerly spent 14 years as a reporter for the Wall Street Journal in its San Francisco and Tokyo bureaus. Prior to that, he spent several years as a reporter at Science Magazine and as a reporter/researcher for the New Republic, both in Washington.
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Jeff Ostrove