Michaël Hocquemiller (0000-0003-2654-7250)

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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.

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Généthon: Evry, FR

2023-11 to present | Head of Non-Clinical Projects Management (Program management)

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Michaël Hocquemiller

https://orcid.org/0000-0003-2654-7250

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Biography

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Employment (4)

Généthon: Evry, FR

Coave Therapeutics: Paris, FR

LYSOGENE: Neuilly sur Seine, FR

Institut Pasteur: Paris, FR

Education and qualifications (3)

Supsante: Paris, FR

Université Paris Descartes: Paris, FR

Université Pierre et Marie Curie: Paris, FR

Works (15)

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

Focal lesions following intracerebral gene therapy for mucopolysaccharidosis

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

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.

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

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

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

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

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.

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

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

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

Directed evolution of motor neurons from genetically engineered neural precursors.

alpha-L-Iduronidase transport in neurites.

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