Brainvectis received the Orphan Drug Designation (ODD)Â from the European Commission for BV-CYP01, its gene therapy drug candidate for the treatment of Huntingtonâ€™s disease.
Brainvectis will participate in the European Society of Gene and Cell Therapy, that will be held at Lausanne, Switzerland.
Nathalie Cartier will give an oral presentation on Tuesday 16th October during the CNS session entitled Â«Â Gene Therapy for Huntingtonâ€™s diseaseÂ Â».
Sandro Alves will give an oral presentation on friday 19th October during the CNS session entitled Â«Â CYP46A1 gene therapy alleviates spinocerebellar ataxia in mouse modelsÂ Â».
Sandro Alves participated in the Gene Therapy partnering day, held at the Institut des Maladies GĂ©nĂ©tiques, organized by Medicen/Imagine (Paris). Sandro Alves introduced Brainvectis in the session Â«Â Innovative Technological SolutionsÂ Â» as an example ofÂ start-up translating academic preclinical research into industrial development.
Brainvectis has closed a financing round of â‚¬ 1.1 million from private investors. The funds will allow the company to move forward its programs while preparing a next financing operation in 2018.
On november 21st,Â Head of preclinical research Dr. Sandro Alves presented Brainvectis as an example ofÂ spin-off translating academic preclinical research into industrial development.
NeurATRIS is a research infrastructure conceived to accelerate the translation of discoveries in basic research into medical innovations for the treatment of diseases of the nervous system.
Brainvectis will be incubated at IPEPS, theÂ Brain and Spine instituteâ€™s (ICM)Â startup incubator and the first innovation accelerator dedicated to brain diseases in France.
Nathalie Cartier will give an oral presentation during the Opening session Tuesday 17 october, entitled:
â€śClinical gene therapy for neurodegenerative diseases: Past, present, and futureâ€ť
Nathalie Cartier will give another talk on Wednesday 18 october during the session 1b: Ocular and central nervous system gene and cell therapy, entitled :
â€śAAV-mediated CYP46A1 gene therapy for Huntingtonâ€™s diseaseâ€ť
Sandro Alves will give a short presentation on Wednesday 18 october during session 2a: Ocular and central nervous system gene and cell therapy, entitled :
â€śAAV-CYP46A1 is beneficial in Alzheimerâ€™s disease: From mice to non-human primatesâ€ť
These results will be also presented in poster P221.
BrainVectis received a â‚¬ 350K innovation aid from BPI to pursue its Huntington’s disease development plan. This money willÂ 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.
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%.
Impairment in cholesterol metabolism is associated with many neurodegenerative disorders including Alzheimerâ€™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 thatÂ 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.