The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to HER2.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C07K 16/32 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des produits de traduction des oncogènes
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Drug delivery agents comprising compounds that facilitate delivery of a wide range of pharmaceuticals and therapeutics; Drug delivery agents comprising biological preparations in the nature of non-viral platforms that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics for treatment of diseases and disorders Biotechnology research services; research and development of platform technologies for delivery of therapies and pharmaceuticals; research and development for the delivery of therapeutics; research and development regarding delivery of therapeutics
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Drug delivery agents comprising compounds that facilitate delivery of a wide range of pharmaceuticals and therapeutics; Drug delivery agents comprising biological preparations in the nature of non-viral platforms that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics for treatment of diseases and disorders Biotechnology research services; research and development of platform technologies for delivery of therapies and pharmaceuticals; research and development for the delivery of therapeutics; research and development regarding delivery of therapeutics
The present disclosure provides anti-tau antibodies and adeno-associated virus (AAV) particles with an AAV genome encoding anti-tau antibodies. Also provided are methods of using anti-tau antibodies and AAV particles for treatment and diagnosis of neurological indications.
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to HER2.
C07K 16/32 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des produits de traduction des oncogènes
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
12.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO DYSTROPHIA MYOTONICA PROTEIN KINASE
The disclosure relates to compositions and methods for modulating, e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, suspected of having, or having myotonic dystrophy type 1 or another DMPK-related disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
inter aliae.g.inter aliainter alia, for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a GBA1-related disorder, e.g., Parkinson's Disease (PD), Gaucher Disease (GD), Parkinson's Disease Dementia (PDD), Dementia with Lewy Bodies (DLB), or Lewy Body Dementia (LBD).
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
14.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO ATAXIN-2
e.g.e.g., reducing or eliminating, the expression of ATXN2 via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, suspected of having, or having spinocerebellar ataxia type 2 or another ATXN2-related disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
15.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO DYSTROPHIA MYOTONICA PROTEIN KINASE
The disclosure relates to compositions and methods for modulating, e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, suspected of having, or having myotonic dystrophy type 1 or another DMPK-related disorder or at least one symptom thereof.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
16.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to tau.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
The disclosure relates to small interfering RNA (siRNA) molecules targeting MAPT and adeno-associated viral (AAV) particles encoding the same for treating tauopathies.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
18.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO FRATAXIN DEFICIENCY
inter aliae.g.inter aliainter alia, for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a disorder associated with frataxin (FXN) deficiency, e.g., Friedreich's Ataxia (FA), or at least one symptom thereof.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
19.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO SYNTAXIN-BINDING PROTEIN 1 DEFICIENCY
The disclosure relates to compositions and methods for altering, e.g., enhancing, the level of STXBP1 protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of an STXBP1-related disorder, e.g., an STXBP1-related neurodegenerative or neuromuscular disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
20.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO CDKL5 DEFICIENCY
The disclosure relates to compositions and methods for altering, e.g., enhancing, the level of CDKL5 protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of a CDKL5-related disorder, e.g., a CDKL5-related neurodegenerative or neuromuscular disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.
The present disclosure relates to cannula systems for delivery of a therapeutic agent to parenchymal tissue of a target subject. In certain embodiments, the cannula system is for delivery of a therapeutic agent to parenchymal tissue in the spine of a target subject. In certain embodiments, the cannula system includes a cannula positioning guide (CPG) useful in the delivery of a therapeutic agent to a localized target tissue of a subject. The guide may be used in a surgical suite or during a surgical procedure.
A61B 17/00 - Instruments, dispositifs ou procédés chirurgicaux
A61B 90/30 - Dispositifs pour éclairer une zone chirurgicale, les dispositifs ayant une corrélation avec d’autres dispositifs chirurgicaux ou avec une intervention chirurgicale
The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
26.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO FRATAXIN DEFICIENCY
e.g.e.g., enhancing, the level of frataxin protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects who have, have been diagnosed with having, or are at risk of having a disorder associated with frataxin (FXN) deficiency, e.g., Friedreich's Ataxia.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
28.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO DYSTROPHIA MYOTONICA PROTEIN KINASE
The disclosure relates to compositions and methods for modulating, e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects who have, have been diagnosed with having, or are at risk of having myotonic dystrophy type 1 or another DMPK-related disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
29.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO CDKL5 DEFICIENCY
The disclosure relates to compositions and methods for altering, e.g., enhancing, the level of CDKL5 protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of a CDKL5-related disorder, e.g., a CDKL5-related neurodegenerative or neuromuscular disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
30.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO ATAXIN-2
e.g.e.g., reducing or eliminating, the expression of ATXN2 via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, suspected of having, or having spinocerebellar ataxia type 2 (SCA2) or another ATXN2-related disorder.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
31.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA 1 DEFICIENCY
inter aliae.g.inter aliainter alia, for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a GBA1-related disorder, e.g., Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or Lewy Body Dementia (LBD).
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
32.
COMPOSITONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 38/50 - Hydrolases (3) agissant sur des liaisons carbone-azote autres que des liaisons peptidiques (3.5), p. ex. asparaginase
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to tau.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
34.
AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE
The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/14 - Médicaments pour le traitement des troubles du système nerveux pour traiter les mouvements anormaux, p. ex. chorée, dyskinésie
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
The present invention relates to small interfering RNA (siRNA) molecules against the HTT gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating Huntington's Disease (HD) using the siRNA molecules and AAV vectors.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/14 - Médicaments pour le traitement des troubles du système nerveux pour traiter les mouvements anormaux, p. ex. chorée, dyskinésie
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
The disclosure relates to small interfering RNA (siRNA) molecules targeting MAPT and adeno-associated viral (AAV) particles encoding the same for treating tauopathies.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
e.g.e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having myotonic dystrophy type 1 or another DMPK-related disorder.
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to human tau.
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C40B 50/04 - Procédés de création de bibliothèques, p. ex. synthèse combinatoire utilisant des techniques de chimie combinatoire dynamique
42.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO FRATAXIN DEFICIENCY
inter aliae.g.inter aliainter alia, in the treatment of subjects who have, have been diagnosed with, or suspected of having a disorder associated with frataxin (FXN) deficiency, e.g., Friedreich's Ataxia.
inter aliae.g.inter aliainter alia, in the treatment of subjects who have, have been diagnosed with, or are at risk of having a GBA1-related disorder, e.g., Parkinson's Disease (PD), Gaucher Disease (GD), Parkinson's Disease Dementia (PDD), Dementia with Lewy Bodies (DLB), or Lewy Body Dementia (LBD).
e.g.e.g., enhancing, the expression of CDKL5 proteins via delivery using an adeno-associated virus (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having CDD, or another CDKL5-related disorder.
e.g.e.g., enhancing, the expression of STXBP1 proteins via delivery using an adeno-associated virus (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having STXBP1 encephalopathy, or another STXBP1-related disorder.
e.g.e.g., reducing or eliminating, the expression of ATXN2 via delivery of a modulatory polynucleotide using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having, spinocerebellar ataxia type 2 or another ATXN2-related disorder.
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
C12N 9/02 - Oxydoréductases (1.), p. ex. luciférase
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
45 - Services juridiques; services de sécurité; services personnels pour individus
Produits et services
Biological preparations for gene therapy; adeno-associated
virus (AAY) gene therapy preparations and pharmaceuticals;
biological preparations for treatment of neurological
diseases and disorders; antibody and gene therapy
preparations for treatment of disease, namely, neurological
diseases; medical preparations and pharmaceuticals for
treatment of neurological diseases via antibody, gene
replacement, gene knockdown, or vectorized antibodies;
antibody and gene therapy preparations for neurological
diseases; nucleic acids encoding polypeptides for gene
therapy; drug delivery agents comprising compounds that
facilitate delivery of a wide range of pharmaceuticals and
biotherapeutics; drug delivery agents comprising capsids
viral platforms, and non-viral platforms, that facilitate
delivery of a wide range of pharmaceuticals and
biotherapeutics; pharmaceutical for the treatment of
neurodegenerative diseases and diseases of the central
nervous system, alzheimer's disease, amyotrophic lateral
sclerosis (als), parkinson's disease, friedreich's ataxia,
huntington's disease, and prion disease. Biotechnology research services; research and development of
platform technologies for delivery of therapies; research
and development of platform technologies for delivery of
gene therapies, antibody therapies and pharmaceuticals;
research and development regarding the use of rna-based
functional screening platforms to discover, research and
develop novel capsids for genetic delivery of therapies and
pharmaceuticals; pharmaceutical research and development in
the field of gene therapy and antibody therapy;
pharmaceutical research and development for the treatment of
neurological diseases and disorders; scientific research in
the field of gene therapy and antibody therapy;
pharmaceutical research services; research and development
of pharmaceutical preparations for non-viral and gene
therapy; research and development of pharmaceutical
preparations for neurological diseases; biotechnology
research services; research and development in the
pharmaceutical and biotechnology fields; research and
development regarding the delivery of therapies across the
blood-brain barrier; research and development regarding the
non-viral and viral vectors for the delivery of therapeutic
agents; research and development regarding the
identification and targeting of specific cells and tissues
for gene therapy; providing medical and scientific research
information in the field of clinical trials and gene
therapy; providing a website featuring medical information
and medical research; providing scientific research
information in the field of clinical trials; hosting a
website featuring medical research information. Medical services provided for clinical trials; providing
patient care for clinical trials; providing medical
information in the field of gene therapy; medical treatment
of neurological diseases, neurodegenerative diseases, and
diseases of the central nervous system, including
alzheimer's disease, amyotrophic lateral sclerosis (als),
parkinson's disease, friedreich's ataxia, and huntington's
disease, and prion disease. Collaboration services in the field of platform technologies
for delivery of therapies and pharmaceuticals relating to
intellectual property, namely, licensing of intellectual
property.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Biological preparations for gene therapy; adeno-associated
virus (aav) gene therapy preparations and pharmaceuticals;
biological preparations for treatment of neurological
diseases and disorders; gene therapy preparations for
treatment of disease, namely, neurological diseases; medical
preparations and pharmaceuticals for treatment of
neurological diseases via gene replacement, gene knockdown,
or vectorized antibodies; gene therapy preparations for
neurological diseases; nucleic acids encoding polypeptides
for gene therapy; drug delivery agents comprising compounds
that facilitate delivery of a wide range of pharmaceuticals
and biotherapeutics; drug delivery agents comprising capsids
that facilitate delivery of a wide range of pharmaceuticals
and biotherapeutics. Pharmaceutical research and development in the field of gene
therapy; pharmaceutical research and development for the
treatment of neurological diseases and disorders; scientific
research in the field of gene therapy; pharmaceutical
research services; research and development of
pharmaceutical preparations for gene therapy; research and
development of pharmaceutical preparations for neurological
diseases; biotechnology research services; research and
development of platform technologies for genetic delivery of
therapies and pharmaceuticals; research and development
services in the field of platforms technologies for genetic
delivery of therapies and pharmaceuticals; research and
development in the pharmaceutical and biotechnology fields;
research and development regarding the use of rna-derived
functional screening platforms to discover, research and
develop novel capsids for genetic deliver of therapies and
pharmaceuticals; research and development regarding the
identification and targeting of specific cells and tissues
for gene therapy delivery. Medical treatment of neurological diseases via gene
replacement, gene knockdown, or vectorized antibodies,
namely, Parkinson's disease, Huntington's disease,
Friedreich's ataxia, amyotrophic lateral sclerosis (ALS),
Alzheimer's disease, tauopathies, synucleinopathies, and
neurodegenerative disease.
The present disclosure relates to compositions, methods and processes for the formulation and for the administration of a gene therapy agent using parvovirus e.g., adeno-associated virus (AAV) to the CNS, CNS tissues, CNS structures or CNS cells.
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
53.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
The disclosure provides variant viral genomes, e.g., variant baculovirus genomes, and expression constructs for the variant genomes, as well as methods for producing the same. The variant viral genomes, e.g., variant baculovirus genomes, can be used, for example, to produce recombinant adeno- associated virus (AAV) particles.
e.g.in vitroin vivoin vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or related condition resulting from a deficiency in the quantity and/or function of GBA1 gene product or associated with decreased expression or protein levels of GCase protein.
C12N 9/24 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2)
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The disclosure relates to small interfering RNA (siRNA) molecules targeting MAPT and adeno-associated viral (AAV) particles encoding the same for treating tauopathies.
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Biological preparations for gene therapy; adeno-associated virus (aav) gene therapy preparations and pharmaceuticals; biological preparations for treatment of neurological diseases and disorders; gene therapy preparations for treatment of disease, namely, neurological diseases; medical preparations and pharmaceuticals for treatment of neurological diseases via gene replacement, gene knockdown, or vectorized antibodies; gene therapy preparations for neurological diseases; nucleic acids encoding polypeptides for gene therapy; drug delivery agents comprising compounds that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; drug delivery agents comprising capsids that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics. (1) Pharmaceutical research and development in the field of gene therapy; pharmaceutical research and development for the treatment of neurological diseases and disorders; scientific research in the field of gene therapy; pharmaceutical research services; research and development of pharmaceutical preparations for gene therapy; research and development of pharmaceutical preparations for neurological diseases; biotechnology research services; research and development of platform technologies for genetic delivery of therapies and pharmaceuticals; research and development services in the field of platforms technologies for genetic delivery of therapies and pharmaceuticals; research and development in the pharmaceutical and biotechnology fields; research and development regarding the use of rna-derived functional screening platforms to discover, research and develop novel capsids for genetic deliver of therapies and pharmaceuticals; research and development regarding the identification and targeting of specific cells and tissues for gene therapy delivery.
(2) Medical treatment of neurological diseases via gene replacement, gene knockdown, or vectorized antibodies, namely, Parkinson's disease, Huntington's disease, Friedreich's ataxia, amyotrophic lateral sclerosis (ALS), Alzheimer's disease, tauopathies, synucleinopathies, and neurodegenerative disease.
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs).
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. The production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles (e.g., rAAVs) which allow for the controlled expression of AAV structural (e.g., capsid) proteins, such as VP1, VP2, and VPS and the controlled expression of AAV nonstructural (e.g., replication) proteins, such as Rep78 and Rep52.
The disclosure provides compositions and methods for the Vectored Augmentation of the Destruction, Expression and/or Regulation of proteins, e.g., VA-DER systems and methods.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 16/00 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/08 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant de virus
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The present disclosure provides anti-tau antibodies. Also provided are methods of using anti-tau antibodies for treatment and diagnosis of neurological indications.
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
66.
COMPOSITIONS AND METHODS FOR CROSSING THE BLOOD BRAIN BARRIER
The disclosure relates to compositions and methods for the preparation, use, and/or formulation of active agents conjugated to ligands for increased crossing of the blood brain barrier.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
68.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAUOPATHY
The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present disclosure provides anti-tau antibodies and adeno-associated virus (AAV) particles with an AAV genome encoding anti-tau antibodies. Also provided are methods of using anti-tau antibodies and AAV particles for treatment and diagnosis of neurological indications.
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C07K 16/00 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
71.
COMPOSITIONS AND METHODS OF TREATING HUNTINGTON'S DISEASE
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an SMN protein, e.g., an SMN1 protein.
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to HER2.
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C07K 16/32 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des produits de traduction des oncogènes
75.
Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.
The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS).
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions, and formulations, including recombinant adeno-associated viruses (rAAV). The present disclosure presents cell culture mediums for use in producing adeno-associated viruses (AAV), such as AAV which comprise a polynucleotide encoding a payload. In certain embodiments, the cell culture medium comprises a hydrolysate mixture, L-glutamine, poloxamer 188 (e.g., 10% pluronic F-68), a lipid emulsion, and a cholesterol mixture. In certain embodiments, the production process and system use Spodoptera fmgiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs). In certain embodiments, the production process and system use Baculo viral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs.
The disclosure provides compositions and methods for altering, e.g., enhancing, the expression of NPC1 and/or NPC2 proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having NPC1 disease or related condition resulting from a deficiency in the quantity and/or function of NPC1 and/or NPC2 proteins or associated with decreased expression or protein levels of NPC1 and/or NPC2 proteins.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
45 - Services juridiques; services de sécurité; services personnels pour individus
Produits et services
Biological preparations for gene therapy; adeno-associated virus (AAV) gene therapy preparations and pharmaceuticals; Biological preparations for treatment of neurological diseases and disorders; antibody and gene therapy preparations for treatment of disease, namely, neurological diseases; medical preparations and pharmaceuticals for treatment of neurological diseases via antibody, gene replacement, gene knockdown, or vectorized antibodies; antibody and gene therapy preparations for neurological diseases; nucleic acids encoding polypeptides for gene therapy; Drug delivery agents comprising compounds that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; Drug delivery agents comprising capsids viral platforms, and non-viral platforms, that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; pharmaceutical for the treatment of neurodegenerative diseases and diseases of the central nervous system, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Parkinson's disease, Friedreich's Ataxia, Huntington's disease, and prion disease Biotechnology research services; research and development of platform technologies for delivery of therapies; research and development of platform technologies for delivery of gene therapies, antibody therapies and pharmaceuticals; research and development regarding the use of RNA-based functional screening platforms to discover, research and develop novel capsids for genetic delivery of therapies and pharmaceuticals; Pharmaceutical research and development in the field of gene therapy and antibody therapy; Pharmaceutical research and development for the treatment of neurological diseases and disorders; scientific research in the field of gene therapy and antibody therapy; pharmaceutical research services; research and development of pharmaceutical preparations for non-viral and gene therapy; research and development of pharmaceutical preparations for neurological diseases; biotechnology research services; research and development in the pharmaceutical and biotechnology fields; research and development regarding the delivery of therapies across the blood-brain barrier; research and development regarding the non-viral and viral vectors for the delivery of therapeutic agents; research and development regarding the identification and targeting of specific cells and tissues for gene therapy; providing scientific research information in the field of clinical trials; hosting a website featuring medical research information Medical services provided for clinical trials; providing patient care for clinical trials; providing medical
information in the field of
gene therapy; providing a website featuring medical information;
medical treatment of neurological diseases, neurodegenerative diseases, and diseases of the central nervous system, including Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Parkinson's disease, Friedreich's Ataxia, and Huntington's disease, and prion disease Collaboration services in the field of platform technologies for delivery of therapies and pharmaceuticals, namely, licensing of intellectual property
C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
C12N 15/35 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
C07K 7/04 - Peptides linéaires ne contenant que des liaisons peptidiques normales
C12N 15/863 - Vecteurs poxviraux, p. ex. virus de la vaccine
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C40B 40/08 - Bibliothèques comprenant de l'ARN ou de l'ADN codant des protéines, p. ex. bibliothèques de gènes
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles comprising a payload (e.g., a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC) or a functional variant thereof). In certain embodiments, the production process uses Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAV particles.
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts tor enhanced tropism to a target tissue.
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The disclosure relates to compositions, formulations, and methods for the preparation, use, and/or formulation of adeno-associated virus capsid protein variants.
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 31/00 - Préparations médicinales contenant des ingrédients actifs organiques
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/00 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
90.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to tau.
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C07K 16/00 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
91.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
e.g.in vitroin vivoin vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to HER2.
C07K 16/32 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des produits de traduction des oncogènes
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
The present disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
The invention provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like pol peptides, for the prevention and/or treatment of diseases and/or disorders.
The invention provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) in the production of AAV particles. In certain embodiments, the production process and system allow for the controlled expression of AAV capsid proteins, such as VP1, VP2 and VP3.
The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
