Genethon

The Children's Hospital of Philadelphia

Institut National De La Sante Et De La Recherche Medicale

Sorbonne Université

Université D'evry-val-d'essonne

Association Institut De Myologie

Duke University

Fondazione Istituto Italiano Di Tecnologia

Fondazione Telethon

International Centre for Genetic Engineering and Biotechnology

Federico Mingozzi

Katherine A High

Giuseppe Ronzitti

Xavier Anguela

Marcela V Maus

Mustafa N Yazicioglu

Daniel J Hui

John Fraser Wright

Dwight D Koeberl

Junwei Sun

Otto-Wilhelm Merten

Bernd Hauck

Pasqualina Colella

Olga Zelenaia

Laura Cancedda

J Fraser Wright

Carole Masurier

Andrés Muro

Giulia Bortolussi

Yifeng Chen

Sang-Oh Han

Andrea Contestabile

Patrice Vidal

Evelyne Gicquel

Isabelle Richard

Philip Johnson

Bérangére Bertin

Fanny Collaud

Francesco Puzzo

The present invention relates to hybrid transcription regulatory elements to drive gene expression, in particular hybrid promoters, designed by the fusion of at least two transcription regulatory elements with different tissue selectivity, such as two promoters driving expression in different tissues in a tissue-selective manner.

Hybrid regulatory elements

The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.

Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism

Compositions and methods are provided for inhibiting T cell mediated destruction of virally transduced, trangene containing cells.

Compositions and methods for detection and modulation of T cell mediated immune responses against viral vectors utilized for gene therapy

The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.

Treatment of glycogen storage disease III

An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.

Method for reducing the expression of NKCC1 in a subject

The present invention relates to variants of acid-alpha glucosidase and uses thereof. Said variants are sequence-optimized and/or are linked to a heterogenous signal peptide.

Acid-alpha glucosidase variants and uses thereof

Virus vectors, virus particles, and methods and uses of screening for, detecting, analyzing and determining amounts of virus antibody, or neutralizing antibody activity of samples are provided. Such virus vectors, virus particles, and methods and uses are applicable to a broad range of virus types, such as lentiviruses, adenovirus, and adeno-associated virus (AAV) serotypes. Methods and uses include virus antibody screening, such as anti-virus immunoglobulins screened for, detected, analyzed and amounts determined.

AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies

The present invention relates to a method for removing undesired anti-AAV antibodies from a blood-derived composition.

Method for removing anti-AAV antibodies from a blood-derived composition

The present invention relates to variants of acid-alpha glucosidase and uses thereof.

Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.

Composition and methods for highly efficient gene transfer using AAV capsid variants

The invention provides viral vector formulations and methods of uses thereof for delivery of transgenes or therapeutic nucleic acids to human subjects. The formulations include a vector and suitable amounts of empty capsids, viral genome-containing capsids, or viral capsid proteins which are optionally chemically or structurally modified and which bind to neutralizing anti-AAV antibodies thereby reducing or preventing antibody-mediated clearance of the vector, but still allowing the genome-containing (therapeutic) vector to transduce target cells and achieve therapeutic gene transfer.

Virus vectors for highly efficient transgene delivery

The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.

Treatment of hyperbilirubinemia

This application relates to the fields of gene therapy and molecular biology. In accordance with the present invention, an adeno-associated virus (AAV) vector comprising an altered capsid protein is provided. More specifically, this invention provides adeno-associated viral vectors comprising protein capsid variants which accelerate vector breakdown and clearance, thereby reducing undesirable immune responses.

Growing community of inventors

Paris, France

Federico Mingozzi