AskBio Acquires BrainVectis and Potential Gene Therapy for Huntington’s

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by Vanessa Pataia |

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AskBio announced that it has acquired BrainVectis, a company developing gene therapies for Huntington’s disease and other neurodegenerative conditions.

The two companies will work to advance BrainVectis’ gene therapy programs for neurodegenerative diseases associated with problematic fat metabolism in the brain, including a lead candidate therapy for Huntington’s called BV-CYP01

“Our acquisition of BrainVectis offers us an extraordinary opportunity to work together to potentially transform patients’ lives,” including Huntington’s, “a devastating and irreversible disease for which no effective treatments exist,” Sheila Mikhail, CEO and co-founder of AskBio, said in a press release.

People with Huntington’s disease have low levels of a protein called cholesterol 24-hydroxylase (CYP46A1) in the brain. This protein has an important role in transforming cholesterol molecules into a form that allows them to be moved out of the brain and eliminated from the body.

Without enough CYP46A1 protein, cholesterol accumulates in the brain and the cholesterol pathway is slowed. This dysfunction in cholesterol metabolism has been linked to neurodegenerative processes in Huntington’s and diseases like Alzheimer’s, as cholesterol is thought to contribute to several areas of cognition, such as learning and memory.

BrainVectis is working on gene therapies that increase the levels of CYP46A1 protein in the brain. Its lead candidate, BV-CYP01, works by using a virus engineered to be harmless, called the adeno-associated virus (AVV), as a vector to carry and deliver the gene for the CYP46A1 protein into brain cells, so they can produce higher amounts of this protein.

In animal models of Huntington’s disease, BV-CYP01 has been shown to restore normal brain cholesterol metabolism. 

“We have seen promising preclinical results with BV-CYP01, and combining our program with gene therapy leader AskBio is ideal as we progress toward clinical studies,” said Nathalie Cartier-Lacave, MD, founder of BrainVectis.

AskBio holds large knowledge of AVV vectors, which will help to optimize the design of an AVV vector for BrainVectis’ BV-CYP01 therapy and similar programs. 

AskBio reports that its AVV manufacturing Pro platform will also allow for a safe and quick, large-scale production of the therapy once it is ready.

“AskBio has the resources to accelerate the development of our gene therapies for patients who desperately need treatment options,” Cartier-Lacave said.

BV-CYP01 was designated an orphan medicine in the European Union to support its development and possible approval.

Financial terms of the acquisition were not disclosed; BrainVectis will operate as a wholly owned subsidiary of AskBio.