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Funded Research

IND-enabling Studies for Platform Clinical Trials of Genome Editors in Multiple Diseases (U01 Clinical Trial Not Allowed) RFA-RM-24-001
PI NameInstitution NameTitle
AHRENS-NICKLAS, REBECCA CLARE (contact) 
MUSUNURU, KIRAN 
CHILDREN'S HOSP OF PHILADELPHIAPersonalized prime editing as a platform for hepatic inborn errors of metabolism
CHEN, ZHENG-YIMASSACHUSETTS EYE AND EAR INFIRMARYAAV-mediated editing to treat human autosomal dominant hearing loss DFNA41 and DFNA2

 

Technologies and Assays for Therapeutic Genome Editing INDs (U01, Clinical Trial Not Allowed) RFA-RM-22-014
PI NameInstitution NameTitle
FREEDMAN, BENJAMIN SOLOMONUNIVERSITY OF WASHINGTONUtility of Human Organoids for Safety and Efficiency Evaluations of Genome Editing Therapeutics
GIANNIKOPOULOS, PETROS (contact)
GURKAN, UMUT A
UNIVERSITY OF CALIFORNIA BERKELEYA Modality-Agnostic Potency Assay Enabling Both Ex Vivo and In Vivo Genome Editing Therapeutics for Sickle Cell Disease
SPENCER, DAVID H (contact)
DUNCAVAGE, ERIC J
WASHINGTON UNIVERSITYGenome sequencing for evaluating the efficacy, specificity, and safety of human genome editing
TSAI, SHENGDARST. JUDE CHILDREN'S RESEARCH HOSPITALUltra-sensitive, unbiased, high-throughput, biochemical CHANGE-seq genome-wide activity and gRNA sequencing assays for therapeutic genome editing INDs
TSAI, SHENGDARST. JUDE CHILDREN'S RESEARCH HOSPITALSensitive, unbiased, high-throughput, cellular GUIDE-seq-2 genome-wide activity assay for therapeutic genome editing INDs

 

IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, Clinical Trial Not allowed)  RFA-RM-22-015
PI NameInstitution NameTitle
DOUDNA, JENNIFER AUNIVERSITY OF CALIFORNIA, BERKELEYCorrection of Neurological Disease via Allele Specific Excision of Pathogenic Repeats
LUTZ, CATHLEEN M (contact)
ARBAB, MANDANA
GRAY, STEVEN J
LIU, DAVID R
MOURO PINTO, RICARDO
JACKSON LABORATORYPreclinical Genome Editing for Rare Neurological Diseases
PERANTEAU, WILLIAM H (contact)
MUSUNURU, KIRAN
CHILDREN'S HOSP OF PHILADELPHIAPostnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases
SAHA, KRISHANUUNIVERSITY OF WISCONSIN-MADISONThe CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies
VALLABH, SONIA MINIKEL  BROAD INSTITUTE, INC.Therapeutic editing to lower PrP in prion disease
 

 

Platform Clinical Trials of Somatic Genome Editing for Multiple Diseases (UG3/UH3, Clinical Trial Required) RFA-RM-22-016
PI NameInstitution NameTitle
JIANG, YONG-HUI (contact)
BERRY-KRAVIS, ELIZABETH MARA
ZHOU, JIANGBING
YALE UNIVERSITYA non-viral CRISPR-mediated genome editing delivery platform as a potential therapy for neurogenetic diseases

 

Somatic Cell Genome Editing Dissemination and Coordination Center (U24, Clinical Trial Not Allowed) RFA-RM-22-017
PI NameInstitution NameTitle
DWINELL, MELINDA RMEDICAL COLLEGE OF WISCONSINTranslational Coordination and Dissemination Center for the SCGE Consortium

 

NIH Support for Conferences and Scientific Meetings (Parent R13 Clinical Trial Not Allowed) PA-21-151
PI NameInstitution NameTitle
SHEPPARD, TERRY L.KEYSTONE SYMPOSIAPrecision Genome Engineering

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-023
In September 2022, the awards below transitioned into their UH3 phase
PI NameInstitution NameTitle
BANKIEWICZ, KRYSTOF S (contact)
MURTHY, NIREN
OHIO STATE UNIVERSITYDevelopment of a nanoparticle-based gene editing technology for neurological applications
CHAIKOF, ELLIOTBETH ISRAEL DEACONESS MEDICAL CENTERDelivery Technologies for In Vivo Genome Editing
DAHLMAN, JAMES (contact)
SANTANGELO, PHILIP J
GEORGIA INSTITUTE OF TECHNOLOGYHighly Specific ZFN-Based HSC Gene Editing Therapies Identified By In Vivo Barcode Nanoparticle Screens And Rationally Designed Mrna
LEONG, KAM WCOLUMBIA UNIVERSITY HEALTH SCIENCESFocused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders
WILSON, ROSS (contact)
DOUDNA, JENNIFER A
UNIVERSITY OF CALIFORNIA BERKELEY   Cas9 RNP delivery to immune cells in vivo via molecular targeting
ZHOU, JIANGBINGYALE UNIVERSITYNovel grafted terpolymers for targeted delivery of CRISPR/Cas9- mediated precise genome editing to the brain

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-016
In September 2022, the awards below transitioned into their UH3 phase
PI NameInstitution NameTitle
ASOKAN, ARAVIND    DUKE UNIVERSITYEvolving High Potency AAV Vectors for Neuromuscular Genome Editing.
SALTZMAN, W. MARK (contact) 
GLAZER, PETER M
YALE UNIVERSITYPoly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung

 

NIH Support for Conferences and Scientific Meetings (Parent R13 Clinical Trial Not Allowed) PA-21-151
PI NameInstitution NameTitle
JARVIS, THALE CROSSKEYSTONE SYMPOSIAPrecision Genome Engineering

 

Innovative Technologies to Non-Invasively Monitor Genome Edited Cells In Vivo (UH2/UH3 Clinical Trial Not Allowed) RFA-RM-18-025

In September 2021, the awards below transitioned into their UH3 phase.
PI NameInstitution NameTitle
BULTE, JEFF WJOHNS HOPKINS UNIVERSITYNon-Invasive Tracking of Genome-Corrected iPS cells in ALS
RONALD, JOHN ANDREWUNIVERSITY OF WESTERN ONTARIONon-Invasive Monitoring of CRISPR/Cas-Edited Chimeric Antigen Receptor T (CAR-T) Cells with Reporter Gene-Based Magnetic Resonance Imaging and Positron Emission Tomography
TARANTAL, ALICE F (contact)
SEGAL, DAVID J
UNIVERSITY OF CALIFORNIA AT DAVISInnovative Translational Imaging Technologies to Monitor Genome Edited Cells in Vivo
VANDSBURGER, MORIELUNIVERSITY OF CALIFORNIA BERKELEYMolecular MRI for in vivo tracking of gene editing and gene edited cells

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-016

In September 2021, the awards below transitioned into their UH3 phase.
PI NameInstitution NameTitle
ASOKAN, ARAVINDDUKE UNIVERSITYEvolving High Potency AAV Vectors for Neuromuscular Genome Editing
CHEN, ZHENG-YI (contact) 
LIU, DAVID R 
XU, QIAOBING
MASSACHUSETTS EYE AND EAR INFIRMARYEfficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles
DEVERMAN, BENJAMIN EBROAD INSTITUTE, INC.Novel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous system
GAO, GUANG-PING (contact) 
ANDERSON, DANIEL G 
XUE, WEN 
UNIV OF MASSACHUSETTS MED SCH WORCESTERDevelop combinatorial non-viral and viral CRISPR delivery for lung diseases
GONG, SHAOQIN (contact) 
EMBORG, MARINA 
LEVINE, JON E 
ROY, SUBHOJIT 
SAHA, KRISHANU
UNIVERSITY OF WISCONSIN-MADISONEnabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain
MCCRAY, PAUL BUNIVERSITY OF IOWADelivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
SALTZMAN, W. MARK (contact) 
GLAZER, PETER M 
YALE UNIVERSITYPoly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung
SONTHEIMER, ERIK J (contact) 
KHVOROVA, ANASTASIA 
WATTS, JONATHAN K 
WOLFE, SCOT A 
UNIV OF MASSACHUSETTS MED SCH WORCESTEREnhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors

 

Innovative Technologies to Non-Invasively Monitor Genome Edited Cells In Vivo (UH2/UH3 Clinical Trial Not Allowed) RFA-RM-18-025
PI NameInstitution NameTitle
BULTE, JEFF WJOHNS HOPKINS UNIVERSITYNon-Invasive Tracking of Genome-Corrected iPS cells in ALS
RONALD, JOHN ANDREWUNIVERSITY OF WESTERN ONTARIONon-Invasive Monitoring of CRISPR/Cas-Edited Chimeric Antigen Receptor T (CAR-T) Cells with Reporter Gene-Based Magnetic Resonance Imaging and Positron Emission Tomography
TARANTAL, ALICE F (contact)
SEGAL, DAVID J
UNIVERSITY OF CALIFORNIA AT DAVISInnovative Translational Imaging Technologies to Monitor Genome Edited Cells in Vivo
VANDSBURGER, MORIELUNIVERSITY OF CALIFORNIA BERKELEYMolecular MRI for in vivo tracking of gene editing and gene edited cells

 

Expanding the Human Genome Engineering Repertoire (U01 Clinical Trial Not Allowed) RFA-RM-18-024
PI NameInstitution NameTitle
GERSBACH, CHARLES ADUKE UNIVERSITYEpigenome Editing Technologies for Treating Diverse Disease
GLAZER, PETER M (contact)
LY, DANITH H
SALTZMAN, W. MARK
YALE UNIVERSITYPNA Nanoparticles for Gene Editing In Vivo

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-023 
PI NameInstitution NameTitle
BANKIEWICZ, KRYSTOF S (contact)
MURTHY, NIREN
OHIO STATE UNIVERSITYDevelopment of a nanoparticle-based gene editing technology for neurological applications
BAO, GANG (contact)
LAGOR, WILLIAM RAYMOND
SUH, JUNGHAE
RICE UNIVERSITYVelcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacity
CHAIKOF, ELLIOTBETH ISRAEL DEACONESS MEDICAL CENTERDelivery Technologies for In Vivo Genome Editing
CURIEL, DAVID TERRYWASHINGTON UNIVERSITYEndothelial-targeted adenovirus for organ-selective gene editing in vivo
DAHLMAN, JAMES (contact)
SANTANGELO, PHILIP J
GEORGIA INSTITUTE OF TECHNOLOGYHighly Specific ZFN-Based HSC Gene Editing Therapies Identified By In Vivo Barcode Nanoparticle Screens And Rationally Designed Mrna
LAM, KIT S (contact)
CHENG, R.HOLLAND
UNIVERSITY OF CALIFORNIA AT DAVISCell-specific nanocarrier with endocytic and endosomolytic activities for therapeutic genome editing
LEONG, KAM WCOLUMBIA UNIVERSITY HEALTH SCIENCESFocused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders
TILTON, JOHN CHRISTIANCASE WESTERN RESERVE UNIVERSITYIn vivo delivery of CRISPR Cas9-guide RNA nucleoprotein complexes using the nanoPOD platform
WILSON, ROSS (contact)
DOUDNA, JENNIFER A
UNIVERSITY OF CALIFORNIA BERKELEYCas9 RNP delivery to immune cells in vivo via molecular targeting
YI, GUOHUATEXAS TECH UNIVERSITY HEALTH SCIS CENTERNovel CRISPR-Cas9 protein delivery to T cells in vivo by targeting CD7
ZHOU, JIANGBINGYALE UNIVERSITYNovel grafted terpolymers for targeted delivery of CRISPR/Cas9- mediated precise genome editing to the brain

 

Development of Cell and Tissue Platforms to Detect Adverse Biological Consequences of Somatic Cell Genome Editing (U01 Clinical Trial Not Allowed) RFA-RM-18-022
PI NameInstitution NameTitle
FREEDMAN, BENJAMIN SOLOMONUNIVERSITY OF WASHINGTONImproving the Safety of Genome Editing With Human Kidney Organoids
GERSBACH, CHARLES A. (contact)
BURSAC, NENAD
TRUSKEY, GEORGE A
DUKE UNIVERSITYMicrophysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes
HINSON, JOHN TRAVISUNIVERSITY OF CONNECTICUT SCH OF MED/DNTHuman cardiac microtissues with innate immune sensing to study adverse consequences of genome editing
MORIZANE, RYUJI (contact)
LEWIS, JENNIFER A.
SABBISETTI, VENKATA
MASSACHUSETTS GENERAL HOSPITALVascularized kidney organoids on chip for efficacy and toxicity testing of somatic genome editing

 

Large Animal Testing Centers for Evaluation of Somatic Cell Genome Editing Tools (U42 Clinical Trial Not Allowed) RFA-RM-18-014
PI NameInstitution NameTitle
TARANTAL, ALICE F (contact) 
SEGAL, DAVID J 
UNIVERSITY OF CALIFORNIA AT DAVISNonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools
WELLS, KEVIN DALE (contact) 
PRATHER, RANDALL S
UNIVERSITY OF MISSOURI-COLUMBIASwine Somatic Cell Genome Editing (SCGE) Center

 

 

Somatic Cell Genome Editing Dissemination and Coordination Center (U24 - Clinical Trial Not Allowed) RFA-RM-18-018
PI NameInstitution NameTitle
DWINELL, MELINDA R (contact) 
SHIMOYAMA, MARY E 
MEDICAL COLLEGE OF WISCONSINDissemination and Coordinating Center for the SCGE Consortium

 

Rodent Testing Centers for Development of Reporter Systems and Evaluation of Somatic Cell Genome Editing Tools (U42 Clinical Trial Not Allowed) RFA-RM-18-012
PI NameInstitution NameTitle
HEANEY, JASON D (contact) 
DICKINSON, MARY E 
LAGOR, WILLIAM RAYMOND
BAYLOR COLLEGE OF MEDICINEBCM-Rice resource for the analysis of somatic gene editing in mice
MURRAY, STEPHEN A (contact) 
LUTZ, CATHLEEN M
JACKSON LABORATORYThe Jackson Laboratory Gene Editing Testing Center (JAX-GETC)

 

Development of Large Animal Reporter Systems for Testing Somatic Cell Genome Editing Tools (U24 Clinical Trial Not Allowed) RFA-RM-18-013
PI NameInstitution NameTitle
CARLSON, DANIEL FREDRECOMBINETICS, INC.Development of Swine Reporter Models for Testing Somatic Cell Genome Editing Tools
FENG, GUOPINGMASSACHUSETTS INSTITUTE OF TECHNOLOGYKnockin marmoset reporters for non-invasive measuring of genome-editing efficiency
HENNEBOLD, JON D OREGON HEALTH & SCIENCE UNIVERSITYRhesus Macaque Somatic Cell Gene Editing Resource

 

Development of Cell and Tissue Platforms to Detect Adverse Biological Consequences of Somatic Cell Gene Editing (U01 Clinical Trial Not Allowed) RFA-RM-18-015
PI NameInstitution NameTitle
KIANI, SAMIRAUNIVERSITY OF PITTSBURGH AT PITTSBURGHMulticell type human liver on chip microphysiological platform to examine CRISPR- based gene modulation.
MCDEVITT, TODD C J. DAVID GLADSTONE INSTITUTESHuman microtissues for in situ detection and functional measurement of adverse consequences caused by genome editing
SAHA, KRISHANU (contact) 
GAMM, DAVID M 
ROY, SUSHMITA 
SKALA, MELISSA CAROLINE 
UNIVERSITY OF WISCONSIN-MADISONSingle Cell Profiling To Define Biomarkers Of Photoreceptor Dysfunction After Gene Editing Within PSC-Derived Organoids
TSAI, SHENGDAR ST. JUDE CHILDREN'S RESEARCH HOSPITALA novel human T-cell platform to define biological effects of genome editing

 

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-016
PI NameInstitution NameTitle
ASOKAN, ARAVINDDUKE UNIVERSITYEvolving High Potency AAV Vectors for Neuromuscular Genome Editing
CHEN, ZHENG-YI (contact) 
LIU, DAVID R 
XU, QIAOBING
MASSACHUSETTS EYE AND EAR INFIRMARYEfficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles
DEVERMAN, BENJAMIN EBROAD INSTITUTE, INC.Novel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous system
GAO, GUANG-PING (contact) 
ANDERSON, DANIEL G 
XUE, WEN 
UNIV OF MASSACHUSETTS MED SCH WORCESTERDevelop combinatorial non-viral and viral CRISPR delivery for lung diseases
GHIRAN, IONITA CALIN BETH ISRAEL DEACONESS MEDICAL CENTERBioengineered red blood cells as extracellular vesicle-mediated delivery platforms for gene editing machinery
GONG, SHAOQIN (contact) 
EMBORG, MARINA 
LEVINE, JON E 
ROY, SUBHOJIT 
SAHA, KRISHANU
UNIVERSITY OF WISCONSIN-MADISONEnabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain
MCCRAY, PAUL BUNIVERSITY OF IOWADelivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
SALTZMAN, W. MARK (contact) 
GLAZER, PETER M 
YALE UNIVERSITYPoly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung
SONTHEIMER, ERIK J (contact) 
KHVOROVA, ANASTASIA 
WATTS, JONATHAN K 
WOLFE, SCOT A 
UNIV OF MASSACHUSETTS MED SCH WORCESTEREnhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors

 

Expanding the Human Genome Engineering Repertoire (U01 Clinical Trial Not Allowed) RFA-RM-18-017
PI NameInstitution NameTitle
DOUDNA, JENNIFER A (contact) 
BANFIELD, JILLIAN 
UNIVERSITY OF CALIFORNIA BERKELEYExpanding CRISPR-Cas editing technology through exploration of novel Cas proteins and DNA repair systems
EKKER, STEPHEN CARL (contact) 
CLARK, KARL J
MAYO CLINIC ROCHESTERBuilding the mitochondrial genome editing repertoire
LIU, DAVID RBROAD INSTITUTE, INC.Expanding the Scope of Base Editing

 

This page last reviewed on December 5, 2024