Gene therapy startup bags cash to fund preclinical work in Huntington's, Alzheimer's

brain imaging

A startup based on the work of gene therapy pioneer Nathalie Cartier-Lacave has secured Series A funding to advance programs against Huntington's and Alzheimer's. The idea is to use gene therapies to lower neuronal cholesterol levels by increasing expression of the CYP46A1 enzyme.

BrainVectis, the Paris, France-based startup behind the programs, is years away from starting to learn whether the concept works in humans. The current goal is to start a Huntington's disease clinical trial in 2019, with an Alzheimer’s study slated to begin two years later. Given that the role of cholesterol in neurodegenerative disorders is still a topic of some debate and gene therapy is far from being an established approach, the project is ambitious, even accounting for the time BrainVectis has given itself to enter the clinic.

Yet, while BrainVectis may ultimately join the long list of companies to swing at Alzheimer’s and miss, the credentials of its founder lend support to the idea that its approach is worth investigating.

Cartier-Lacave is the director of research at French public research powerhouse Inserm and president of the European Society of Gene and Cell Therapy, positions she gained on the strength of a career spent turning gene therapy from a concept into a workable experimental therapeutic approach. Most notably, Cartier-Lacave worked for years on a program against adrenoleukodystrophy, leading to her and Patrick Aubourg running the first clinical trial of a gene therapy to use a lentiviral vector.

That gene therapy is now known as Lenti-D, one of the lead candidates in bluebird bio’s ($BLUE) pipeline. Bluebird is currently testing Lenti-D in a Phase II/III trial, but still points to the study run by Cartier-Lacave when making its case for the potential of the gene therapy.

Having completed its Series A, BrainVectis now has €1 million ($1.1 million) more to find out whether it can follow in the footsteps of bluebird. BrainVectis will use the money to run preclinical pharmacology tests. Jérôme Becquart, an Aventis veteran who has spent the past decade in and around the French biotech scene, has come on board as CEO, putting him at the head of the five-person team that is working to hustle the gene therapy asset to the clinic.

The team is piggybacking on years of work by Cartier-Lacave and her collaborators that led to papers in journals including Brain, Human Molecular Genetics and Molecular Therapy. Those papers built the case that the malfunctioning of neuronal cholesterol homeostasis is linked to neurodegenerative disorders, and that vectors encoding CYP46A1 may be able to improve outcomes by affecting tau pathology.