Institut National De La Sante Et De La Recherche Medicale

Centre National De La Recherche Scientifique

Novartis Ag

UniversitÉde Strasbourg

Sorbonne Université

Université Pierre Et Marie Curie (Paris 6)

University of California

University of Pennsylvania

Centre National De La Recherche Scientifique-cnrs

Inserm (institut National De La Sante Et De La Recherche Medicale)

Cnrs

Novartis Pharma Gmbh

Inserm

Sparingvision

Thierry Leveillard

Jose-Alain Sahel

David Hicks

Saddek Mohand-Said

Leah Caroline Thomas Byrne

Olivier Poch

Celine Jaillard

Jean Bennett

John G Flannery

Junwei Sun

Jeannette Bennicelli

Deniz Dalkara

Leah C Byrne

Valerie Fontaine

Valérie Fontaine

Jens Duebel

Emmanuelle Clerin-Lachapelle

Ludivine Perrocheau

Christo Kole

Hanen Khabou

Alain Van Dorsselaer

Hawa Camara

Céline Jaillard

Ram Fridlich

Therese Cronin

Xin Mei

José Alain Sahel

Jeanette Bennicelli

Thérèse Cronin

Frédéric Blond

José-Alain Sahel

John Flannery

Leah Byrne

Jose Alain Sahel

Alexandra Lyor Bouaziz

Géraldine Millet-puel

Najate Aït-ali Maamri

Géraldine Puel

Emmanuelle Clerin

The present invention relates to methods and composition for use in the treatment of retinal degeneration, in particular retinal degeneration due to retinal pigment epithelium dysfunction.

Transgenic RPE cells overexpressing OTX2 for the treatment of retinal degeneration

Several new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea are provided. The delivery modality relies on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector comprising a VP1 capsid protein as set forth in SEQ ID NO: 11 and the polynucleotide of interest under the control of the pR1.7 promoter as set forth in SEQ ID NO: 12 are also provided.

Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector

The present invention concerns a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a compound selected in the group comprising (i) a polypeptide comprising an amino acid sequence selected in the group comprising the amino acid sequence of the long isoform inof the RdCVF2 gene (SEQ ID NO: 10), orthologs, derivatives and fragments thereof, (ii) a polynucleotide coding for said polypeptide, (iii) a vector comprising said polynucleotide, and (iv) a host cell genetically engineered expressing said polypeptide; the use of such a composition for the manufacture of a medicament for treating and/or preventing a neurodegenerative disorder in a subject; and a method of testing a subject thought to have or be predisposed to having a neurodegenerative disorder.

Neuronal viability factor and use thereof in Alzheimer's disease

The invention relates to compounds which activate the BASIGIN signalling pathway, preferably agonists of BASIGIN, for the treatment of neurodegenerative disorders.

Method and pharmaceutical composition for use in the treatment of neurodegenerative disorders

The invention relates to the use of HNRNPC-expressing vectors for preventing and/or treating a tauopathy, such as Alzheimer's disease. The invention relates to methods for detecting a risk of developing a tauopathy such Alzheimer's disease in a patient, comprising the step of detecting the level of HNRNPC in a biological sample obtained from said patient.

Methods for the treatment and diagnosis of Alzheimer's disease and traumatic brain injuries

The present invention relates to the synergistic combination of the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.

Synergistic combination of neuronal viability factors and uses thereof

The present invention relates to methods and pharmaceutical compositions for the treatment of age-related macular degeneration (AMD). In particular the present invention relates to RdCVFL polypeptide or polynucleotide for use in the treatment of AMD.

Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (AMD)

The present disclosure concerns a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a compound selected in the group comprising (i) a polypeptide comprising an amino acid sequence selected in the group comprising the amino acid sequence of the long isoform inof the RdCVF2 gene (SEQ ID NO:10), orthologs, derivatives and fragments thereof, (ii) a polynucleotide coding for said polypeptide, (iii) a vector comprising said polynucleotide, and (iv) a host cell genetically engineered expressing said polypeptide; the use of such a composition for the manufacture of a medicament for treating and/or preventing a neurodegenerative disorder in a subject; and a method of testing a subject thought to have or be predisposed to having a neurodegenerative disorder.

Methods for treating cone dystrophy with neuronal viability factor, RDCVF2

Disclosed are methods and compositions for early diagnosis, monitoring and treatment of retinal dystrophy, age-related macular degeneration, Bardet-Biedel syndrome, Bassen-kornzweig syndrome, best disease, choroidema, gyrate atrophy, congenital amourosis, refsun syndrome, stargardt disease and Usher syndrome. In particular, the invention relates to a protein, termed 'Rdcvf1,' that is differentially transcribed and expressed in subjects suffering from retinal dystrophies and the like, such as retinal dystrophy and age-related macular degeneration compared with nonsufferers, antibodies which recognize this protein, and methods for diagnosing such conditions.

Methods of treating retinitis pigmentosa using nucleic acids encoding RDCVF1 or RDCVF2

This invention relates to methods and compositions for detection and treatment of neurodegenerative diseases. In particular, the invention relates to polypeptides that can protect against neuron degeneration, nucleic acid molecules that encode such polypeptides, and antibodies that recognize said polypeptides.

Neuronal viability factor and use thereof

Compositions comprising polynucleotides encoding RDCVF1 or RDCVF2

Methods of increasing RDCVF 1 or RDCVF 2 polypeptides in retinal cells

This invention relates to methods and compositions for detection and treatment of retinal degenerative diseases. In particular, the invention relates to polypeptides that can protect against cone degeneration, nucleic acid molecules that encode such polypeptides, and antibodies that recognize said polypeptides.

Trophic factor for the treatment of retinal degenerative diseases

Disclosed are methods and compositions for early diagnosis, monitoring and treatment of retinal dystrophy, age-related macular degeneration, Bardet-Biedel syndrome, Bassen-kornzweig syndrome, best disease, choroidema, gyrate atrophy, congenital amourosis, refsun syndrome, stargardt disease and Usher syndrome. In particular, the invention relates to a protein, termed 'Rdcvf1,' that is differentially transcribed and expressed in subjects suffering from retinal dystrophies and the like, such as retinal dystrophy and age-related macular degeneration compared with non-sufferers, antibodies which recognize this protein, and methods for diagnosing such conditions.

Retinal dystrophy-associated protein and uses thereof

Growing community of inventors

Paris, France

Thierry Leveillard