UC Berkeley backs oral gene therapy company targeting the gut in $1.5M seed round

DNA
The company's first target is a single-gene inherited disorder that causes hundreds of polyps to form in the colon, by using a nonviral nanoparticle that mimics enteroviruses to bypass mucus barriers. (Pixabay)

A UC Berkeley-backed preclinical company, DNAlite Therapeutics, has gathered $1.5 million in seed money to pursue oral gene therapies that can slip through the mucus barrier that typically blocks access to the gastrointestinal tract.

Its first target is familial adenomatous polyposis, a rare, inherited disorder that causes hundreds of polyps to form in the walls of the colon, eventually turning malignant unless the large intestine is removed.

DNAlite’s nonviral nanoparticle platform mimics certain properties of enteroviruses—a common cause of the stomach flu, as well as polio, meningitis and other diseases—in order to deliver a functional copy of a defective gene to the gastrointestinal epithelium.

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“Using a nonviral system allows us to overcome packaging capacity and immune response, which is a common challenge with current viral vectors,” DNAlite’s VP of business development and operations Xixi Zhu told FierceBiotech. The company has “several strategies” to overcome the volatile, acidic environment of the stomach and small intestine, she said.

After proving itself against diseases involving a single defective gene, the company plans to move on to more prevalent GI conditions such as inflammatory bowel disease, Zhu said, adding that the delivery platform can be adapted to other insulated tissues, such as the respiratory tract, sinuses, reproductive tract and the eyes.

The Berkeley Catalyst Fund led the seed round, with participation from the university, SVE Capital, Blue Bear Ventures, SOSV and the Baldota family. In addition, the San Francisco-based company received an investment from the Chinese pharma company BrightGene Bio-Medical Technology.

DNAlite’s immediate plans include refining its delivery system and demonstrating efficacy in an animal model.

While attending UC Berkeley, co-founders Mubhij Ahmad and Timothy Day conducted their initial experiments at the university’s core facility, as well as through contract research organizations off-site in their spare time, Zhu said, with DNAlite holding its own patents since its founding in 2016.

“That being said, we were lucky to be a part of the UC system as they have enabled us to receive tremendous support and progress quickly with our programs,” she said.