AskBio Takes Over French Gene Therapy Company to Treat Alzheimer’s and Huntington’s

The Paris-based BrainVectis, a biotech developing gene therapies for Huntington’s disease and Alzheimer’s disease, has been acquired by the US clinical-stage gene therapy company AskBio. 

AskBio’s gene therapy experience and manufacturing capacity will help to accelerate BrainVectis’ lead candidate for Huntington’s into phase I testing. In return, AskBio gets to expand its list of target indications. 

AskBio recognized the scientific merit of the work at BrainVectis as an opportunity to strengthen our central nervous system clinical pipeline,” Robin Fastenau, VP of Communications for AskBio, told me. No financial details about the acquisition were disclosed.

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BrainVectis’ lead candidate focuses on increasing the expression of a protein called CYP46A1. This enzyme is key for turning excess cholesterol into a derivative that can be cleared from the brain into the blood. It is also reduced in Huntington’s and Alzheimer’s patients, allowing toxic levels of cholesterol to build up in the brain. By increasing the levels of this enzyme, BrainVectis aims to restore normal cholesterol metabolism and improve the clinical outcome.

So far, BrainVectis’ lead gene therapy candidate has shown proof-of-concept in animal models of Huntington’s. It also received orphan drug designation from the European Commission last year. 

According to Nathalie Cartier-Lacave, CEO and Founder of BrainVectis, AskBio offers a strong cell-line manufacturing process. In particular, it can manufacture a range of viral vectors, including BrainVectis’ vector of choice: the adeno-associated virus.

This powerful adeno-associated virus technology and Askbio’s expertise in clinical applications will allow us to rapidly go to clinical application in patients,” Cartier-Lacave told me. 

There are currently no approved treatments able to slow down the progress of Huntington’s and Alzheimer’s. Combined with aging populations in developed countries, these debilitating diseases are creating a healthcare challenge. Many companies are trying — and sometimes failing — to develop drugs able to stop the progression of Alzheimer’s, for example, as such a drug could make a huge impact on society.  

According to Cartier-Lacave, Huntington’s disease is the first target for the company going forward. The disease is caused by a mutation in a gene called HTT that is important for the function of nerve cells. It’s a very severe disease for which we think the treatment may not only decrease the toxic mutated protein, but also preserve neurons from death,” she added. 

Gene therapies are becoming ever more popular in the biotech industry for their potential to tackle previously incurable conditions. BrainVectis is one of several companies aiming gene therapies at the brain; another is the French company Lysogene. However, there are major challenges with developing gene therapies for the brain, for example, getting the therapy past the blood-brain barrier and into the brain tissue.

AskBio might be up to the challenge, as it has taken several gene therapies to the clinic. Its proprietary treatment for the neuromuscular indication Pompe disease is currently in phase I/II, and it has licensed its technology to several big pharmaceutical companies. Those currently in clinical development include treatments for Duchenne muscular dystrophy (Pfizer), hemophilia (Takeda), and spinal muscular atrophy (AveXis), which was approved by the FDA last year, and is awaiting marketing approval by the EMA.


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