Overcoming the blood-brain barrier for gene therapy delivery with AAV capsids

June 2, 2022 -- The blood vessels surrounding the brain are much more restrictive than the body’s other blood vessels in keeping out potentially harmful molecules. This protection provided by the blood-brain barrier keeps people healthy, but it also makes it difficult for gene therapies to penetrate the blood vessels, according to Amy Pooler, PhD, vice president of neuroscience at Sangamo Therapeutics.

As a result, current gene delivery to the central nervous system (CNS) continues to be a challenge, Pooler told ScienceBoard.net at the American Society of Gene & Cell Therapy (ASGCT) 2022 annual meeting in Washington, DC. However, Sangamo's adeno-associated virus (AAV) capsids are designed to overcome the blood-brain barrier, ensuring CNS access while minimizing exposure to a patient's preexisting anti-AAV antibodies.

"What we want to do is engineer novel capsids that can penetrate into the brain," Pooler said. "Sangamo scientists developed a novel platform that I'm really excited about. It's called SIFTER, Selection In vivo for Transduction and Expression of RNA. And what's really exciting about this part is the expression. We know that AAV can travel around the body but it's not enough just for it to get to the target tissue. It also has to get inside the cell and express the therapeutic payload. In our case, it's the zinc fingers."

Two new novel capsids were discovered using the SIFTER technique by Sangamo's David Ojala, PhD, senior scientist for AAV engineering. At ASGCT 2022, Ojala presented pre-clinical data on a study of the genetically engineered AAV capsid platform for delivery to the CNS after cerebrospinal fluid administration.

A poster presentation at the meeting also discussed the first applications of the SIFTER platform to Sangamo's blood-brain barrier program, which is "looking to deliver intravenously in order to achieve widespread brain delivery," Pooler said.

Watch the video below to learn more.

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