Audentes, Loaded With $30M, Pursues Gene Therapy for Rare Diseases

7/26/13Follow @xconomy

Five years ago, hardly anybody would invest in a startup focused on gene therapies for ultra-rare diseases. But gene therapy has been on the comeback trail, and a San Francisco startup called Audentes Therapeutics has corralled $30 million to see if it can overcome some of the obstacles that have tripped up others in this field over the past 20 years.

Audentes announced last week that it raised the hefty sum of $30 million in a Series A venture financing from a trio of well-known healthcare investors—OrbiMed Advisors, Versant Ventures, and 5AM Ventures. That will be enough money to run the company for the next several years, to see if it can come up with promising data from clinical trials for a couple of rare diseases called X-linked myotubular myopathy and Pompe disease.

Gene therapy has tantalized biologists for a couple decades, and frustrated biotech and pharmaceutical companies. The idea, for those unfamiliar, is about modifying viruses to carry copies of genes into cells, where they can replace missing or faulty genes at the root cause of certain diseases. Billions of dollars were invested in companies large and small throughout the 90s and early 2000s, and after a series of safety concerns, there are still no FDA-approved gene therapy products. But Netherlands-based UniQure broke through in November with the first gene therapy approved in Europe, and growing scientific enthusiasm around a series of technical improvements helped propel Cambridge, MA-based Bluebird Bio (NASDAQ: BLUE) to one of the most successful biotech IPOs of the year.

While looking around for opportunities last year as an entrepreneur-in-residence at OrbiMed in New York, Matthew Patterson saw enough improvement in the underlying technology of gene therapy to give it a chance in rare muscle disorders.

“It became clear to me there’s been tremendous scientific advances in field,” Patterson says.

Still, tremendous scientific advancement doesn’t guarantee the next steps will be clear or obvious. The company name, which Patterson came up with, is derived from the Latin term for “one who has courage or boldness.” Some readers probably know the phrase “audentes fortuna juvat,” which translates to “fortune favors the bold.” He knows there is still plenty of skepticism in the industry of gene therapy, largely from veterans who have seen failure after failure.

“The theme of courage is very appropriate when we think about the patients we’re trying to serve,” he says. “I like the boldness, the effort it will take from everyone involved.”

Patterson is a veteran of the rare disease field, with past experience at Amicus Therapeutics (NASDAQ: FOLD), BioMarin Pharmaceuticals (NASDAQ: BMRN), and Genzyme. During his time at OrbiMed, he connected with … Next Page »

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