Brainvectis will give presentations at the Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 17-20, 2017, in Berlin, Germany.

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Nathalie Cartier will give an oral presentation during the Opening session Tuesday 17 october, entitled:

“Clinical gene therapy for neurodegenerative diseases: Past, present, and future”


Nathalie Cartier will give another talk on Wednesday 18 october during the session 1b: Ocular and central nervous system gene and cell therapy, entitled :

“AAV-mediated CYP46A1 gene therapy for Huntington’s disease”


Sandro Alves will give a short presentation on Wednesday 18 october during session 2a: Ocular and central nervous system gene and cell therapy, entitled :

“AAV-CYP46A1 is beneficial in Alzheimer’s disease: From mice to non-human primates”

These results will be also presented in poster P221.

BrainVectis received a € 350K innovation aid from BPI

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BrainVectis received a € 350K innovation aid from BPI to pursue its Huntington’s disease development plan. This money will be used to expand the team and to finish preclinical studies, before a first in man trial in the end of 2019.

BPI innovation aid assists companies that carry out innovation projects involving industrial research and / or experimental development.


Brainvectis was laureate of the 2017 National Research Agency call for proposal

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Along with its academic partners, BrainVectis was selected during the call for proposal 2017 of the National Research Agency, for the development of its gene therapy program in Huntington’s disease.

The consortium will receive a grant of 534K euros to finance the development of the program for 2 years: development of BV-CYP01 biomanufacturing, injection studies in primate, and complete characterization of cholesterol metabolism in a cohort of Huntington patients.

Our partners are the CEA, the Institute of the Brain and the Marrow (ICM) and Nathalie Cartier’s laboratory (Inserm).

ANR mission is to finance French research projects. It is based on a competitive selection process that meets international standards. This year, the selection rate was 12.6%.

New results about how impaired brain cholesterol metabolism leads to neurodegeneration

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Impairment in cholesterol metabolism is associated with many neurodegenerative disorders including Alzheimer’s disease (AD). However, the lipid alterations underlying neurodegeneration and the connection between altered cholesterol levels and AD remains not fully understood.

In a new publication, Cartier et al. demonstrated that major membrane lipids, sphingolipids and specific enzymes involved in phosphatidylcholine and sphingolipid metabolism, were rapidly increased in the hippocampus of AAV-shCYP46A1 injected mice.

In conclusion, neuronal cholesterol accumulation induced by Cyp46a1 down-regulation in mouse hippocampus disrupts brain lipid homeostasis.

See the publication

At the end of FTTI, BrainVectis is selected among the 12 start-ups that have convinced investors the most

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France Tech Transfer Invest took place in Paris on the 1-2 of February, 2017. The event convened 41 technology transfer spin-offs from France selected by SATT the French Tech Transfer Accelerator Network, and selected investors in the field of digital tech, biotech, healthtech and energy. The event was a collaboration between the SATT, Tech Tour, Bpifrance and its EuroQuity platform and aims to efficiently and effectively support French tech transfer spinoff companies to meet and engage with international investors and corporate partners.

After two days, BrainVectis was awarded as one of the 12 start-ups that convinced investors the most, and was selected to give a talk at the European Venture Summit which will take place on 4 and 5 December 2017 in Düsseldorf.

New success in AAV-based gene therapy: Voyager Therapeutics Announces Positive Interim Phase 1b in Parkinson’s disease

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The interim surgical data from the Phase 1b study showed that VY-AADC01 continues to demonstrate safety with increasing coverage of the putamen (targeted region in the brain) in all the 10 Parkinson’s disease patients treated in the ongoing dose escalation trial.

Obtaining sufficient coverage of the putamen with VY-AADC01 is a key step towards potentially improving patients’ response to levodopa (levodopa is the standard of care treatment for Parkinson’s disease). Having reached an average coverage of 34% in the high-dose cohort, Voyager demonstrates that through direct injection in the brain one can achieve significant diffusion volumes for AAV vectors, confirming hopes of AAV-based gene therapy to treat efficiently neurodegenerative diseases.

See the details of the results here

BrainVectis CSO will speak at the ESGCT annual congress

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CSO Nathalie Cartier-Lacave will discuss on how AAV-CYP46A1 brain administration restores cholesterol metabolism and is neuroprotective in Huntington’s disease at the annual congress of The European Society of Gene and Cell Therapy (ESGCT) in Florence, on Friday, 21 October. ESGCT is a leading organization committed to support scientists and clinicians involved with basic and translational research on gene transfer and clinical applications of gene and cell therapy.