grant

Novel strategy to block AAV neutralizing antibody activity

Organization NABGEN, INC.Location DURHAM, UNITED STATESPosted 1 Apr 2023Deadline 31 Aug 2028
NIHUS FederalResearch GrantFY202519S Gamma Globulin3-D structure3-dimensional structure3D structure7S Gamma GlobulinAAV vectorAAV-based vectorAcuteAd vectorAddressAdeno-Associated VirusesAdenoviral VectorAdenovirus VectorAdoptionAdoptive TransferAffectAffinityAmino AcidsAnimal ModelAnimal Models and Related StudiesAnimalsAntibodiesAntibody titer measurementAntigensApheresisAran-Duchenne diseaseAutoimmune StatusAutoimmunityB blood cellsB cellB cellsB-CellsB-LymphocytesB-cellBindingBinding ProteinsBiologicalBlindnessBlood Coagulation DisordersBlood Component RemovalBlood PlasmaBlood SerumBody TemperatureCancer TreatmentCanine SpeciesCanis familiarisCapsidCellular TropismChemicalsChronicClinicClinicalClinical TrialsCoagulation DisorderCoagulopathyCommunicationCruveilhier diseaseDNA TherapyDependoparvovirusDependovirusDevelopmentDiseaseDisorderDogsDogs MammalsDoseDrug TherapyDrugsEngineeringEnsureEnzyme GeneEnzymesEperythrozoonExclusionFDA approvedFactor VIII DeficiencyFailureFeedbackFree EnergyFutureGene DeliveryGene Transfer ClinicalGenetic DiseasesGenetic InterventionGraft RejectionHaemobartonellaHemapheresisHemophiliaHemophilia AHigh PrevalenceHumanIND FilingIND applicationIND packageIND submissionIgAIgGIgMImmune GlobulinsImmune responseImmunizeImmunoglobulin AImmunoglobulin GImmunoglobulin MImmunoglobulinsIn VitroIndividualInvestigational New Drug ApplicationLigand Binding ProteinLigand Binding Protein GeneM proteinMalignant Neoplasm TherapyMalignant Neoplasm TreatmentMarketingMedicationMedicineMendelian diseaseMendelian disorderMendelian genetic disorderMethodsMiceMice MammalsModern ManModificationMolecular InteractionMurineMusMuscular DystrophiesMycoplasmaMyodystrophicaMyodystrophyNatureOrphan DiseaseOrphan DrugsPatientsPharmaceutical PreparationsPharmacological TreatmentPharmacotherapyPhasePheresisPlasmaPlasma SerumPopulationPre IND FDA meetingPre-IND mtgPreparationPrimatesPrimates MammalsProceduresProductionProtein BindingProtein EngineeringProteinsProtocolProtocols documentationPublishingQOLQualifyingQuality of lifeRare DiseasesRare DisorderResidualResidual stateReticuloendothelial System, Serum, PlasmaSTTRSafetySerotypingSerumSmall Business Technology Transfer ResearchSpinal Muscular AtrophyStandardizationSurfaceTechnologyTestingTherapeuticTherapeutic UsesTimeTransplant RejectionTransplantation RejectionVirionVirus Particleadeno associated virus groupadeno vectoradeno-associated viral vectoradeno-associated virus vectoradenovectoraminoacidanaloganti-cancer therapyantibody titeringbiologicbleeding disorderbound proteincancer therapycancer vaccinationcancer-directed therapycanineclinical developmentclotting disordercostdesigndesigningdevelopmentaldomestic dogdrug interventiondrug treatmentdrug/agentefficacy studygene repair therapygene therapygene-based therapygenetic conditiongenetic disordergenetic protein engineeringgenetic therapygenomic therapyhost responseimmune modulatory interventionimmune system responseimmunogenimmunointerventionimmunological interventionimmunoresponseimprovedimproved outcomein vitro activityin vivolead candidatemanufacturemanufacturing runmodel of animalmonogenic diseasemonogenic disordermortalitymultiple myeloma M Proteinmuscle dystrophymutantnano particlenano-sized particlenanoparticlenanosized particleneutralizing antibodynew approachesnon-human primatenonhuman primatenovelnovel approachesnovel strategiesnovel strategyorphan disorderpatient screeningpharmaceutical interventionpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticspre-IND consultationpre-IND discussionpre-IND meetingpre-Investigational New Drug meetingpreparationspreventpreventingproduction runprotein designrational designsafety and feasibilityside effectsingle-gene diseasesingle-gene disordersuccesstech developmenttechnology developmentthermolabilitythermostabilitythree dimensional structuretissue tropismtooltransgene expressiontumor vaccinationvectorvirus tropismvision lossvisual loss
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Description preview

Adeno-associated virus (AAV) vectors have been successfully applied in clinical trials in patients with different
genetic disorders. However, the high prevalence of neutralizing antibodies (Nabs) is one of major concerns to

limit the broad AAV application in clinics. In humans, approximately 50% of the population has Nabs. Several

approaches have…

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Novel strategy to block AAV neutralizing antibody activity — NABGEN, INC. | UNITED STATES | Apr 2023 | Dev Procure