Somatic Cell Genome Editing (SCGE) Program Phase 2: Platform Clinical Trials of Somatic Genome Editing for Multiple Diseases (UG3/UH3) is sponsored by NIH Office of Strategic Coordination (Common Fund). Supports platform clinical trials of somatic genome editing for multiple diseases, aiming to demonstrate the safety and efficacy of genome editing therapeutics in diverse disease contexts.

Funded Research | NIH Common Fund Somatic Cell Genome Editing (SCGE) Somatic Cell Genome Editing (SCGE) IND-enabling Studies for Platform Clinical Trials of Genome Editors in Multiple Diseases (U01 Clinical Trial Not Allowed) RFA-RM-24-001 PI Name Institution Name Title BAUER, DANIEL EVAN BOSTON CHILDREN'S HOSPITAL Robust verification of genetic variant-associated candidate off-target sites HE, MEI UNIVERSITY OF FLORIDA Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs TARANTAL, ALICE F UNIVERSITY OF CALIFORNIA AT DAVIS Validated Immunoassays to Accelerate Therapeutic Genome Editing INDs IND-enabling Studies for Platform Clinical Trials of Genome Editors in Multiple Diseases (U01 Clinical Trial Not Allowed) RFA-RM-24-001 PI Name Institution Name Title AHRENS-NICKLAS, REBECCA CLARE (contact) MUSUNURU, KIRAN CHILDREN'S HOSP OF PHILADELPHIA Personalized prime editing as a platform for hepatic inborn errors of metabolism CHEN, ZHENG-YI MASSACHUSETTS EYE AND EAR INFIRMARY AAV-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 Name Institution Name Title FREEDMAN, BENJAMIN SOLOMON UNIVERSITY OF WASHINGTON Utility of Human Organoids for Safety and Efficiency Evaluations of Genome Editing Therapeutics GIANNIKOPOULOS, PETROS (contact) GURKAN, UMUT A UNIVERSITY OF CALIFORNIA BERKELEY A 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 UNIVERSITY Genome sequencing for evaluating the efficacy, specificity, and safety of human genome editing TSAI, SHENGDAR ST.

JUDE CHILDREN'S RESEARCH HOSPITAL Ultra-sensitive, unbiased, high-throughput, biochemical CHANGE-seq genome-wide activity and gRNA sequencing assays for therapeutic genome editing INDs TSAI, SHENGDAR ST.

JUDE CHILDREN'S RESEARCH HOSPITAL Sensitive, 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 Name Institution Name Title DOUDNA, JENNIFER A UNIVERSITY OF CALIFORNIA, BERKELEY Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats LUTZ, CATHLEEN M (contact) MOURO PINTO, RICARDO JACKSON LABORATORY Preclinical Genome Editing for Rare Neurological Diseases PERANTEAU, WILLIAM H (contact) MUSUNURU, KIRAN CHILDREN'S HOSP OF PHILADELPHIA Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases SAHA, KRISHANU UNIVERSITY OF WISCONSIN-MADISON The 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 Name Institution Name Title JIANG, YONG-HUI (contact) BERRY-KRAVIS, ELIZABETH MARA ZHOU, JIANGBING YALE UNIVERSITY A 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 Name Institution Name Title DWINELL, MELINDA R MEDICAL COLLEGE OF WISCONSIN Translational 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 Name Institution Name Title SHEPPARD, TERRY L.

KEYSTONE SYMPOSIA Precision 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 Name Institution Name Title BANKIEWICZ, KRYSTOF S (contact) MURTHY, NIREN OHIO STATE UNIVERSITY Development of a nanoparticle-based gene editing technology for neurological applications CHAIKOF, ELLIOT BETH ISRAEL DEACONESS MEDICAL CENTER Delivery Technologies for In Vivo Genome Editing DAHLMAN, JAMES (contact) SANTANGELO, PHILIP J GEORGIA INSTITUTE OF TECHNOLOGY Highly Specific ZFN-Based HSC Gene Editing Therapies Identified By In Vivo Barcode Nanoparticle Screens And Rationally Designed Mrna LEONG, KAM W COLUMBIA UNIVERSITY HEALTH SCIENCES Focused 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, JIANGBING YALE UNIVERSITY Novel 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 Name Institution Name Title ASOKAN, ARAVIND DUKE UNIVERSITY Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.

SALTZMAN, W.

MARK (contact) GLAZER, PETER M YALE UNIVERSITY Poly(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 Name Institution Name Title JARVIS, THALE CROSS KEYSTONE SYMPOSIA Precision 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 Name Institution Name Title BULTE, JEFF W JOHNS HOPKINS UNIVERSITY Non-Invasive Tracking of Genome-Corrected iPS cells in ALS RONALD, JOHN ANDREW UNIVERSITY OF WESTERN ONTARIO Non-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 DAVIS Innovative Translational Imaging Technologies to Monitor Genome Edited Cells in Vivo VANDSBURGER, MORIEL UNIVERSITY OF CALIFORNIA BERKELEY Molecular 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 Name Institution Name Title ASOKAN, ARAVIND DUKE UNIVERSITY Evolving High Potency AAV Vectors for Neuromuscular Genome Editing CHEN, ZHENG-YI (contact) XU, QIAOBING MASSACHUSETTS EYE AND EAR INFIRMARY Efficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles DEVERMAN, BENJAMIN E BROAD INSTITUTE, INC. Novel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous system GAO, GUANG-PING (contact) XUE, WEN UNIV OF MASSACHUSETTS MED SCH WORCESTER Develop combinatorial non-viral and viral CRISPR delivery for lung diseases GONG, SHAOQIN (contact) SAHA, KRISHANU UNIVERSITY OF WISCONSIN-MADISON Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain MCCRAY, PAUL B UNIVERSITY OF IOWA Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides SALTZMAN, W.

MARK (contact) GLAZER, PETER M YALE UNIVERSITY Poly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung SONTHEIMER, ERIK J (contact) WOLFE, SCOT A UNIV OF MASSACHUSETTS MED SCH WORCESTER Enhancing 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 Name Institution Name Title BULTE, JEFF W JOHNS HOPKINS UNIVERSITY Non-Invasive Tracking of Genome-Corrected iPS cells in ALS RONALD, JOHN ANDREW UNIVERSITY OF WESTERN ONTARIO Non-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 DAVIS Innovative Translational Imaging Technologies to Monitor Genome Edited Cells in Vivo VANDSBURGER, MORIEL UNIVERSITY OF CALIFORNIA BERKELEY Molecular 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 Name Institution Name Title GERSBACH, CHARLES A DUKE UNIVERSITY Epigenome Editing Technologies for Treating Diverse Disease GLAZER, PETER M (contact) SALTZMAN, W.

MARK YALE UNIVERSITY PNA 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 Name Institution Name Title BANKIEWICZ, KRYSTOF S (contact) MURTHY, NIREN OHIO STATE UNIVERSITY Development of a nanoparticle-based gene editing technology for neurological applications BAO, GANG (contact) SUH, JUNGHAE RICE UNIVERSITY Velcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacity CHAIKOF, ELLIOT BETH ISRAEL DEACONESS MEDICAL CENTER Delivery Technologies for In Vivo Genome Editing CURIEL, DAVID TERRY WASHINGTON UNIVERSITY Endothelial-targeted adenovirus for organ-selective gene editing in vivo DAHLMAN, JAMES (contact) SANTANGELO, PHILIP J GEORGIA INSTITUTE OF TECHNOLOGY Highly 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 DAVIS Cell-specific nanocarrier with endocytic and endosomolytic activities for therapeutic genome editing LEONG, KAM W COLUMBIA UNIVERSITY HEALTH SCIENCES Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders TILTON, JOHN CHRISTIAN CASE WESTERN RESERVE UNIVERSITY In vivo delivery of CRISPR Cas9-guide RNA nucleoprotein complexes using the nanoPOD platform WILSON, ROSS (contact) DOUDNA, JENNIFER A UNIVERSITY OF CALIFORNIA BERKELEY Cas9 RNP delivery to immune cells in vivo via molecular targeting YI, GUOHUA TEXAS TECH UNIVERSITY HEALTH SCIS CENTER Novel CRISPR-Cas9 protein delivery to T cells in vivo by targeting CD7 ZHOU, JIANGBING YALE UNIVERSITY Novel 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 Name Institution Name Title FREEDMAN, BENJAMIN SOLOMON UNIVERSITY OF WASHINGTON Improving the Safety of Genome Editing With Human Kidney Organoids GERSBACH, CHARLES A.

(contact) TRUSKEY, GEORGE A DUKE UNIVERSITY Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes HINSON, JOHN TRAVIS UNIVERSITY OF CONNECTICUT SCH OF MED/DNT Human cardiac microtissues with innate immune sensing to study adverse consequences of genome editing MORIZANE, RYUJI (contact) SABBISETTI, VENKATA MASSACHUSETTS GENERAL HOSPITAL Vascularized 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 Name Institution Name Title TARANTAL, ALICE F (contact) SEGAL, DAVID J UNIVERSITY OF CALIFORNIA AT DAVIS Nonhuman Primate Testing Center for Evaluation of Somatic Cell Genome Editing Tools WELLS, KEVIN DALE (contact) PRATHER, RANDALL S UNIVERSITY OF MISSOURI-COLUMBIA Swine Somatic Cell Genome Editing (SCGE) Center Somatic Cell Genome Editing Dissemination and Coordination Center (U24 - Clinical Trial Not Allowed) RFA-RM-18-018 PI Name Institution Name Title DWINELL, MELINDA R (contact) SHIMOYAMA, MARY E MEDICAL COLLEGE OF WISCONSIN Dissemination 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 Name Institution Name Title HEANEY, JASON D (contact) LAGOR, WILLIAM RAYMOND BAYLOR COLLEGE OF MEDICINE BCM-Rice resource for the analysis of somatic gene editing in mice MURRAY, STEPHEN A (contact) LUTZ, CATHLEEN M JACKSON LABORATORY The 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 Name Institution Name Title CARLSON, DANIEL FRED RECOMBINETICS, INC. Development of Swine Reporter Models for Testing Somatic Cell Genome Editing Tools FENG, GUOPING MASSACHUSETTS INSTITUTE OF TECHNOLOGY Knockin marmoset reporters for non-invasive measuring of genome-editing efficiency HENNEBOLD, JON D OREGON HEALTH & SCIENCE UNIVERSITY Rhesus 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 Name Institution Name Title KIANI, SAMIRA UNIVERSITY OF PITTSBURGH AT PITTSBURGH Multicell type human liver on chip microphysiological platform to examine CRISPR- based gene modulation MCDEVITT, TODD C J.

DAVID GLADSTONE INSTITUTES Human microtissues for in situ detection and functional measurement of adverse consequences caused by genome editing SAHA, KRISHANU (contact) SKALA, MELISSA CAROLINE UNIVERSITY OF WISCONSIN-MADISON Single Cell Profiling To Define Biomarkers Of Photoreceptor Dysfunction After Gene Editing Within PSC-Derived Organoids TSAI, SHENGDAR ST.

JUDE CHILDREN'S RESEARCH HOSPITAL A 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 Name Institution Name Title ASOKAN, ARAVIND DUKE UNIVERSITY Evolving High Potency AAV Vectors for Neuromuscular Genome Editing CHEN, ZHENG-YI (contact) XU, QIAOBING MASSACHUSETTS EYE AND EAR INFIRMARY Efficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles DEVERMAN, BENJAMIN E BROAD INSTITUTE, INC. Novel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous system GAO, GUANG-PING (contact) XUE, WEN UNIV OF MASSACHUSETTS MED SCH WORCESTER Develop combinatorial non-viral and viral CRISPR delivery for lung diseases GHIRAN, IONITA CALIN BETH ISRAEL DEACONESS MEDICAL CENTER Bioengineered red blood cells as extracellular vesicle-mediated delivery platforms for gene editing machinery GONG, SHAOQIN (contact) SAHA, KRISHANU UNIVERSITY OF WISCONSIN-MADISON Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain MCCRAY, PAUL B UNIVERSITY OF IOWA Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides SALTZMAN, W.

MARK (contact) GLAZER, PETER M YALE UNIVERSITY Poly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung SONTHEIMER, ERIK J (contact) WOLFE, SCOT A UNIV OF MASSACHUSETTS MED SCH WORCESTER Enhancing 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 Name Institution Name Title DOUDNA, JENNIFER A (contact) BANFIELD, JILLIAN UNIVERSITY OF CALIFORNIA BERKELEY Expanding CRISPR-Cas editing technology through exploration of novel Cas proteins and DNA repair systems EKKER, STEPHEN CARL (contact) CLARK, KARL J MAYO CLINIC ROCHESTER Building the mitochondrial genome editing repertoire LIU, DAVID R BROAD INSTITUTE, INC. Expanding the Scope of Base Editing This page last reviewed on