Personal information

gene therapy, Translational sciences, AAV, Neurodegenerative diseases, lysosomal storage diseases, fragile X syndrome, preclinical studies, clinical studies
France

Biography

Passionate about gene therapy and translational sciences, I currently lead non-clinical projects management at Généthon. Prior to Généthon, I was leading the translational sciences at Coave Therapeutics and at Lysogene where I have overseen the pre-clinical work that have advanced into clinical development up to pivotal phase for two rare central nervous system disorders as well as designing and testing gene therapy drug candidates for Fragile X, Gaucher/Parkinson, and ALS diseases. Before Lysogene, I worked as a research scientist at the Pasteur Institute focused on neurophysiopathology and gene therapy of Lysosomal Storage Diseases. I have a PhD in neurosciences from the University Paris Descartes (Paris V), a Master’s in genetics from University Pierre and Marie Curie (Paris VI) and a Clinical Research Associate certification.

Activities

Employment (4)

Généthon: Evry, FR

2023-11 to present | Head of Non-Clinical Projects Management (Program management)
Employment
Source: Self-asserted source
Michaël Hocquemiller

Coave Therapeutics: Paris, FR

2023-03 to 2023-07 | Vice President Translational Sciences (R&D)
Employment
Source: Self-asserted source
Michaël Hocquemiller

LYSOGENE: Neuilly sur Seine, FR

2010-09 to 2023-02 | VP Head of Non Clinical Development (R&D)
Employment
Source: Self-asserted source
Michaël Hocquemiller

Institut Pasteur: Paris, FR

2005-01 to 2009-06 | Scientist (Rétrovirus et Transfert Génétique)
Employment
Source: Self-asserted source
Michaël Hocquemiller

Education and qualifications (3)

Supsante: Paris, FR

2009 to 2010 | CRA (Clinical Reasearch)
Qualification
Source: Self-asserted source
Michaël Hocquemiller

Université Paris Descartes: Paris, FR

2005 to 2009 | PhD (Neurosciences)
Education
Source: Self-asserted source
Michaël Hocquemiller

Université Pierre et Marie Curie: Paris, FR

1999 to 2004 | Master (Genetic)
Education
Source: Self-asserted source
Michaël Hocquemiller

Works (15)

Tandem mass spectrometry-based assay for heparan-N-sulphatase in paediatric CSF: A potential pharmacodynamic biomarker for mucopolysaccharidosis type IIIA therapy

Clinica Chimica Acta
2024-10 | Journal article
Part of ISSN: 0009-8981
Contributors: Roberto Speziale; Michaël Hocquemiller; Xin Mei; Danilo Fabbrini; Savina Malancona; Karen Aiach; Ralph Laufer; Laura Orsatti
Source: Self-asserted source
Michaël Hocquemiller

Focal lesions following intracerebral gene therapy for mucopolysaccharidosis

Annals of Clinical and Translational Neurology
2023-05-11 | Newspaper article | Author
Part of ISSN: 2328-9503
Part of ISSN: 2328-9503
Contributors: Marianna Bugiani; Truus E.M. Abbink; Arthur W. D. Edridge; Lia van der Hoek; Anne E. J. Hillen; Niek P. van Til; Gino V. Hu‐A‐Ng; Marjolein Breur; Karen Aiach; Philippe Drevot et al.
Source: Self-asserted source
Michaël Hocquemiller

Design of a stable human acid‐β‐glucosidase: towards improved Gaucher disease therapy and mutation classification

The FEBS Journal
2023-02-21 | Newspaper article
Part of ISSN: 1742-464X
Part of ISSN: 1742-4658
Contributors: Sarka Pokorna; Olga Khersonsky; Rosalie Lipsh‐Sokolik; Adi Goldenzweig; Rebekka Nielsen; Yacov Ashani; Yoav Peleg; Tamar Unger; Shira Albeck; Orly Dym et al.
Source: Self-asserted source
Michaël Hocquemiller

Intra-CSF AAVrh10 vector administration corrects disease pathology in murine and feline models of GM1 gangliosidosis and achieves widespread distribution of beta-galactosidase in the CNS of Non-Human Primates.

Molecular Therapy - Methods & Clinical Development
2022-10 | Journal article
Contributors: Michaël Hocquemiller; Laura Giersch; Xin Mei; Amanda L. Gross; Ashley N. Randle; Heather L. Gray-Edwards; Judith A. Hudson; Sophia Todeasa; Lorelei Stoica; Douglas R. Martin et al.
Source: check_circle
Crossref

AAV-delivered diacylglycerol kinase DGKk achieves long-term rescue of fragile X syndrome mouse model.

EMBO molecular medicine
2022-04-04 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.

Molecular therapy. Methods & clinical development
2019-12-10 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA.

Human gene therapy
2019-06-04 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.

Human gene therapy
2016-07-01 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial.

Human gene therapy
2014-05-05 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

Storage vesicles in neurons are related to Golgi complex alterations in mucopolysaccharidosis IIIB.

The American journal of pathology
2010-10-29 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

GAP43 overexpression and enhanced neurite outgrowth in mucopolysaccharidosis type IIIB cortical neuron cultures.

Journal of neuroscience research
2010-01-01 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

Enhanced degradation of synaptophysin by the proteasome in mucopolysaccharidosis type IIIB.

Molecular and cellular neurosciences
2009-01-21 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

Directed evolution of motor neurons from genetically engineered neural precursors.

Stem cells (Dayton, Ohio)
2008-07-17 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

alpha-L-Iduronidase transport in neurites.

Molecular genetics and metabolism
2006-01-24 | Journal article
Source: Self-asserted source
Michaël Hocquemiller

Forced expression of the motor neuron determinant HB9 in neural stem cells affects neurogenesis.

Experimental neurology
2006-01-24 | Journal article
Source: Self-asserted source
Michaël Hocquemiller