grant
In vivo hematopoietic stem cell genetic modification by viral particles
Organization BOSTON CHILDREN'S HOSPITALLocation BOSTON, UNITED STATESPosted 9 Sept 2024Deadline 31 May 2028
NIHUS FederalResearch GrantFY2025AddressAutologousBaboonsBackBiodistributionBiologyBlood DiseasesBlood Precursor CellBone MarrowBone Marrow GraftingBone Marrow Reticuloendothelial SystemBone Marrow TransplantBone Marrow TransplantationBone marrow failureCD132CRISPRCRISPR/Cas systemCell BodyCell CompartmentationCell CompartmentationsCell Surface ReceptorsCellsClinicalClustered Regularly Interspaced Short Palindromic RepeatsCo-StimulatorCollectionCommon Cytokine Receptor Gamma ChainCommon Cytokine Receptor γ ChainCommon Gamma ChainCommon γ ChainComplexCostimulatorDNA IntegrationDNA TherapyDNA editorDNA mutationDiseaseDisorderDorsumDoseERVsEndogenous RetrovirusesEngineeringEngraftmentEnhancersEpidermal Thymocyte Activating FactorFrequenciesGamma Chain Interleukin 2 ReceptorGene DeliveryGene ModifiedGene TransferGene Transfer ClinicalGenesGeneticGenetic ChangeGenetic EngineeringGenetic Engineering BiotechnologyGenetic Engineering Molecular BiologyGenetic InterventionGenetic defectGenetic mutationGlycoproteinsGoalsGuide RNAHERVsHb SS diseaseHbSS diseaseHematologic DiseasesHematological DiseaseHematological DisorderHematopoietic Progenitor CellsHematopoietic Stem Cell MobilizationHematopoietic progenitor MobilizationHematopoietic progenitor cell MobilizationHematopoietic stem cellsHemoglobinHemoglobin S DiseaseHemoglobin sickle cell diseaseHemoglobin sickle cell disorderHigh Dose ChemotherapyHumanHuman Endogenous RetrovirusesIL-2IL2 ProteinIL2RGIL2RG geneIMD4In VitroIndividualInterleukin 2Interleukin 2 PrecursorInterleukin 2 Receptor GammaInterleukin IIInterleukin-2Interleukine 2Interleukine 2 PrecursorInterleukine IILentiviral VectorLentivirus VectorLymphocyte Mitogenic FactorMarrow TransplantationMediatingMedicalMendelian diseaseMendelian disorderMendelian genetic disorderMiceMice MammalsMitogenic FactorModalityModern ManModificationMurineMusMutationMyeloablative ChemotherapyPapioPatientsPhenotypePoint MutationPopulationProceduresProductionProgenitor CellsReceptor ProteinRecombinant DNA TechnologyRetroviral VectorRetrovirus VectorRibonucleoproteinsRiskSCIDXSCIDX1SafetySchwachman-DiamondSevere Combined ImmunodeficiencySevere Combined Immunodeficiency SyndromeSevere Combined Immunologic DeficiencyShwachman-DiamondShwachman-Diamond syndromeSickle Cell AnemiaSpecificityStressSystemT cell growth factorT-Cell Growth FactorT-Cell Stimulating FactorT-CellsT-LymphocyteTechnologyTestingTherapeuticTherapeutic Gene EditingThymocyte Stimulating FactorToxic effectToxicitiesTransgenesTransplantationVHHVHH antibodyValidationVeinsViralVirusVirus-like particleX-Linked Severe Combined ImmunodeficiencyXSCIDbase editorblood cell progenitorblood disorderblood progenitorblood stem cellblood-forming stem cellbone progenitorbone stem cellboyscamelid antibodycamelid based antibodycamelid derived antibodycamelid derived fragmentcamelid heavy chain only Abscamelid immunoglobulincamelid single chain antibodycamelid variable heavy chaincell transductioncellular transductionchemotherapyclinical validationcombined T and B cell inborn immunodeficiencyfirst in manfirst-in-humanflexibilityflexiblegRNAgene correctedgene correctiongene editorgene manipulationgene modificationgene repair therapygene therapygene transplantationgene transplantation for gene therapygene-based therapygene-editing therapygenetic manipulationgenetic therapygenetically engineeredgenetically manipulategenetically modifiedgenetically perturbgenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenome editorgenome mutationgenomic correctiongenomic therapygenotoxicityhematopoietic progenitorhematopoietic stem and progenitor cell mobilizationhematopoietic stem progenitor cellhemopoietic progenitorhemopoietic stem cellhumanized micehumanized mouseimprovedin vivoinnovateinnovationinnovativemanufacturemonogenic diseasemonogenic disordernanobodiesnanobodynew technologynew therapeutic approachnew therapeutic interventionnew therapeutic strategiesnew therapy approachesnew treatment approachnew treatment strategynovelnovel technologiesnovel therapeutic approachnovel therapeutic interventionnovel therapeutic strategiesnovel therapy approachparticlepatient populationperipheral bloodpre-clinicalpreclinicalprogenitor cell replacementreceptorscreeningscreeningssdAbsickle cell diseasesickle cell disordersickle diseasesicklemiasingle domain antibodiessingle-gene diseasesingle-gene disorderstem cell gene therapystem cell replacementstem cellssuccesstechnology platformtechnology systemtherapeutic editingtherapeutic genome editingtherapeutic targetthymus derived lymphocytetooltransduced cellstransgenetransplantvalidationsvariable heavy chain antibodyvectorvirus-like nanoparticlesviruslike particle
Description preview
PROJECT SUMMARY
Hematopoietic stem cell (HSC) gene therapies with lentiviral or CRISPR based gene modification are
demonstrating favorable safety profiles and remarkable efficacy to treat severe monogenic blood disorders with
unmet clinical need, including X-linked Severe Combined Immunodeficiency (X-SCID) and Sickle Cell Disease
(SCD), with…
🔒
Start 7-day free trial — $29.99/mo →Full details available on the Agency plan
Unlock the complete grant description, eligibility criteria, contract value, evaluation details and apply link — plus alerts, pipeline tracking, and CSV export.
Agency Plan
7-day free trialUnlock procurement & grants
Upgrade to access active tenders from World Bank, UNDP, ADB and more — with email alerts and pipeline tracking.
$29.99 / month
- 🔔Email alerts for new matching tenders
- 🗂️Track tenders in your pipeline
- 💰Filter by contract value
- 📥Export results to CSV
- 📌Save searches with one click