Pediatric Blood & Marrow Transplant & Cellular Therapy

Finished

Assaying Swine Serum Samples

Panoskaltsis-Mortari, A. (PI) & Ehrhardt, M. J. (Other Role)

USDA AGRICULTURAL RESEARCH SERVICE

6/6/24 → 8/5/24

Project: Research project

Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application

Blazar, B. R. (PI) & Hippen, K. L. (CoI)

STANFORD UNIVERSITY, CALIFORNIA INST. FOR REGENERATIVE MED.

1/1/24 → 12/31/24

Project: Research project

National Adrenoleukodystrophy (ALD) Registry

Gupta, A. O. (PI), Lund, T. C. (CoI) & Orchard, P. J. (CoI)

ASSOC OF PUBLIC HEALTH LABORATORIES, US Dept. of Health & Human Services HRSA

9/1/23 → 6/30/24

Project: Research project

Travel: ISID 2023 Travel Grant

Osborn, M. J. (PI)

INTL SOC FOR INVESTIGATIVE DERMATOLOGY

2/21/23 → 7/31/23

Project: Research project

Matrix Bound Vesicles for the Treatment of DUX4-mediated Muscle Pathology

Kyba, M. (PI)

FSHD CANADA FOUNDATION

2/1/23 → 6/30/23

Project: Research project

Collection of Sodium Heparin Plasma Samples from Subjects Undergoing Intravenous Busulfan Treatment

Orchard, P. J. (PI)

SALADAX BIOMEDICAL, INC

12/13/22 → 3/31/24

Project: Research project

Interrogation of mDUX expression in muscle in mice

Bosnakovski, D. (PI)

FACIOSCAPAULOHUMERAL (FSH) SOCIETY

12/5/22 → 12/4/24

Project: Research project

Mucopolysaccharidosis Newborn Screening meeting

Orchard, P. J. (PI) & Gupta, A. O. (CoI)

ASSOC OF PUBLIC HEALTH LABORATORIES, US Dept. of Health & Human Services HRSA

8/23/22 → 9/30/23

Project: Research project

A Randomized Trial of Roadmap 2.0

Ebens, C. (PI)

UNIVERSITY OF MICHIGAN

8/1/22 → 1/27/23

Project: Research project

Foundations for a Phase 1 clinical trial of cell-based therapy for Duchenne muscular dystrophy

Kang, P. B. (PI), Karachunski, P. I. (CoI), Perlingeiro, R. (CoI) & Wagner, J. E. (CoI)

NIH NIAMS NATL INST OF ARTHRIT

7/15/22 → 6/30/24

Project: Research project

Engineered immune cells for safer and more effective tumor therapy

Osborn, M. J. (PI)

ST. BALDRICK'S FOUNDATION

7/1/22 → 6/30/24

Project: Research project

Autologous Revertant Mosaic Fibroblasts for Wound Healing in Dystrophic Epidermolysis Bullosa

Tolar, J. (PI), Ebens, C. (CoI) & Osborn, M. J. (CoI)

EB RESEARCH PARTNERSHIP, INC.

6/1/22 → 5/31/23

Project: Research project

Collection of Gene Mutations for Laboratory Quality AssuranceNewborn Screening Accuracy Project

Gupta, A. O. (PI) & Orchard, P. J. (CoI)

SEQUOIA FOUNDATION, CENTERS FOR DISEASE CONTROL & PREVENTION

5/12/22 → 10/14/23

Project: Research project

Lysosomal Storage Disease Focused Training

Orchard, P. J. (PI) & Miller, B. S. (CoI)

SANOFI-AVENTIS US

11/8/21 → 11/7/23

Project: Research project

Generating protein-ligand interaction data on a series of putative DUX4 interactors

Kyba, M. (PI)

UNIVERSITY OF BASEL

11/1/21 → 1/31/22

Project: Research project

Severe Combined Immunodeficiency (SCID) Screening and Education

Ebens, C. (PI)

UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, US Dept. of Health & Human Services HRSA

8/1/21 → 7/31/23

Project: Research project

Dose Optimization of Nervonic Acid-a Potential Therapy to Alleviate Disease Progression in Adrenoleukodystrophy

Kartha, R. (PI) & Lund, T. C. (CoI)

NIH NAT'L CTR FOR ADVANCING TRAN SCIENCE

7/1/21 → 5/31/24

Project: Research project

Testing the effects of antifibrotic drugs on an FSHD mouse model and FSHD fibroadipoprogenitors: thefirst step toward antifibrotic therapy for FSHD

Bosnakovski, D. (PI)

FRIENDS OF FSH RESEARCH

5/15/21 → 8/31/22

Project: Research project

Safety and efficacy of Nov340 delivery of SaRNA in treating MPS I disease

Whitley, C. B. (PI), Lund, T. C. (CoI), Orchard, P. J. (CoI) & Ou, L. (CoI)

MINA THERAPEUTICS LTD

5/1/21 → 4/29/23

Project: Research project

Nervonic Acid-a Potential Therapy to Alleviate Disease Progression in Adrenoleukodystrophy

Kartha, R. (PI), Cloyd, J. C. (CoI) & Lund, T. C. (CoI)

LIGAND PHARMACEUTICALS INC

5/1/21 → 10/31/23

Project: Research project

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Gupta, A. O. (PI)

THE CHILDREN'S HOSPITAL OF PHILADELPHIA, SHIRE HUMAN GENETIC THERAPIES, INC.

12/1/20 → 12/30/23

Project: Research project

National Adrenoleukodystrophy (ALD) Registry

Orchard, P. J. (PI) & Gupta, A. O. (CoI)

ASSOC OF PUBLIC HEALTH LABORATORIES, HRSA MATERNAL AND CHILD HEALTH (MCH)

11/26/20 → 8/31/23

Project: Research project

Next Generation Genome Editing for RDEB

Tolar, J. (PI) & Osborn, M. J. (CoI)

EB RESEARCH PARTNERSHIP, INC.

11/18/20 → 11/17/21

Project: Research project

Optimizing Editing and Delivery for Sickle Cell Disease

Osborn, M. J. (PI)

THE BROAD INSTITUTE, INC., BILL & MELINDA GATES FOUNDATION

10/28/20 → 1/31/23

Project: Research project

Correction of Fanconi Anemia Mutations using Digital Genome Engineering

Moriarity, B. S. (PI), Wagner, J. E. (CoI) & Webber, B. R. (CoI)

FANCONI CANCER FOUNDATION

10/1/20 → 3/31/23

Project: Research project

Transposon Engineered B Cell Therapy for Hunter Syndrome

Belur, L. (PI) & Lund, T. C. (CoI)

IMMUSOFT CORPORATION, NATIONAL INSTITUTES OF HEALTH (NIH)

9/30/20 → 9/29/22

Project: Research project

Data on outcomes with allogenic haematopoietic stem cell transplants in Mucopolysaccharidosis type I

Orchard, P. J. (PI), Eisengart, J. (CoI), Lund, T. C. (CoI) & Miller, B. S. (CoI)

ORCHARD THERAPEUTICS

9/9/20 → 5/6/22

Project: Research project

CRISPR/Cas9 Cellular Engineering for the Creation of Coagulation Factor-Based Resuscitation Fluid to Combat Hemorrhage and Coagulopathy

Osborn, M. J. (PI)

THE GENEVA FOUNDATION, U.S. DEPARTMENT OF DEFENSE

6/16/20 → 6/15/23

Project: Research project

Blue Rock Development Phase I

Wagner, J. E. (PI) & Brunstein, C. G. (CoI)

BLUEROCK THERAPEUTICS GP LLC

3/15/20 → 9/14/20

Project: Research project

Engineered B Cells as a Universal Platform for the Treatment of Enzymopathies

Moriarity, B. S. (PI), Lund, T. C. (CoI), McIvor, R. S. (CoI), Orchard, P. J. (CoI) & Webber, B. R. (CoI)

NIH Nat'l Inst of Allergy & Infect Dis

3/1/20 → 2/28/25

Project: Research project

Rheos compound study

Blazar, B. R. (PI)

RHEOS MEDICINES, INC.

2/11/20 → 10/11/22

Project: Research project

A phase II pilot trial to estimate survival after a non-total body irradiation (TBI) based conditioning regimen in patients diagnosed with B-acute lymphoblastic leukemia (ALL) who are pre-allogeneic hematopoietic cell transplantation (HCT) next-generatio

Ebens, C. (PI), MacMillan, M. L. (Prior Principal Investigator) & Neglia, J. P. (Prior Principal Investigator)

CHILDREN'S HOSPITAL LOS ANGELES, GATEWAY FOR CANCER RES

2/1/20 → 1/31/25

Project: Research project

Long Term Outcomes in Mucopolysaccharidosis Type I-Hurler (MPS I-H) PatientsAfter Hematopoietic Stem Cell Transplantation (HSCT)

Gupta, A. O. (PI)

SANOFI-AVENTIS US

1/9/20 → 12/31/20

Project: Research project

A Phase 2, single-arm, open-label study to evaluate the safety and efficacy of MGTA-456 in patients with sickle cell disease (SCD) undergoing hematopoietic stem cell transplantation (HSCT)

Smith, A. R. (PI)

MEDPACE, INC., MAGENTA THERAPEUTICS

1/1/20 → 12/31/20

Project: Research project

Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Following Hematopoietic Stem CellTransplantation (ACES)

Ebens, C. (PI)

CHILDREN'S HOSPITAL LOS ANGELES, CALIFORNIA INST. FOR REGENERATIVE MED.

12/12/19 → 12/19/21

Project: Research project

A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects with Fanconi Anemia Subtype A

MacMillan, M. L. (PI)

ROCKET PHARMACEUTICALS, INC

11/1/19 → 11/30/24

Project: Research project

Creation and Characterization of a Biopolymer-bioink Hybrid Tissue Engineered Vascular Graft

Panoskaltsis-Mortari, A. (PI)

MEDTRONIC INC

10/1/19 → 2/28/21

Project: Research project

Preclinical Studies in Pluripotent Stem Cell-Derived Myogenic Progenitors to Enable a First-in-Human Phase 1 Safety/Dose Escalation Trial for Duchenne Muscular Dystrophy

Perlingeiro, R. (PI), Karachunski, P. I. (CoI), McKenna, D. H. (CoI), Schumacher, R. J. (CoI) & Wagner, J. E. (CoI)

DUCHENNE UK

9/15/19 → 7/31/23

Project: Research project

An Open-Label, Single-Arm, Multicenter Study of Combination anti-CD3/CD7 Immunotoxin (T-Guard) for Steroid-Refractory Acute Graft-versus-Host Disease.

MacMillan, M. L. (PI)

NATIONAL MARROW DONOR PROGRAM

9/13/19 → 3/31/21

Project: Research project

Biomarkers in MPS

Lund, T. C. (PI)

SHIRE HUMAN GENETIC THERAPIES, INC.

9/3/19 → 6/25/20

Project: Research project

Services Agreement Rider for Painless Bone Marrow Harvest Project

Wagner, J. E. (PI)

NATIONAL MARROW DONOR PROGRAM

9/1/19 → 6/19/20

Project: Research project

MASTER: National Marrow Donor Program Services Agreement

Wagner, J. E. (PI)

NATIONAL MARROW DONOR PROGRAM

9/1/19 → 6/19/20

Project: Research project

An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Itacitinib in Combination With Corticosteroids for the Treatment of Steroid-Naive Acute Graft-Versus-Host Disease inPediatric Subjects

MacMillan, M. L. (PI)

INCYTE CORPORATION

9/1/19 → 8/31/23

Project: Research project

Exposure-response of combination pre-transplant conditioning (cPTC) regiments in pediatric patientsundergoing hematopoietic cell transplantation (HCT) Master PK Protocol

Gupta, A. O. (PI)

UNIVERSITY OF CALIFORNIA, SAN FRANCISCO

9/1/19 → 6/30/24

Project: Research project

An open-label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of min-102 treatment on disease progression prior to human stem cell transplant (hsct)

Orchard, P. J. (PI)

MINORYX THERAPEUTICS

8/22/19 → 7/1/21

Project: Research project

MT2019-09: A randomized trial of low versus moderate exposure busulfan for infants with severecombined immunodeficiency (SCID) receiving TCR????+/CD19+ depleted transplantation: A Phase II studyby the Primary Immune Deficiency Treatment Consortium (PID

Ebens, C. (PI) & Smith, A. R. (Prior Principal Investigator)

CHILDREN'S HOSPITAL LOS ANGELES, NIH Nat'l Inst of Allergy & Infect Dis

8/16/19 → 7/31/24

Project: Research project

An Interventional, Multicenter, Single Arm, Phase I/IIA Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)

Ebens, C. (PI)

RHEACELL GMBH & CO KG

7/1/19 → 6/30/24

Project: Research project

IMD-002: A FOLLOW-UP STUDY TO EVALUATE THE SAFETY AND CLINICAL OUTCOMES OF PATIENTS WITH NON-MALIGNANT DISEASES WHO HAVE UNDERGONE HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH MGTA-456

Orchard, P. J. (PI)

MAGENTA THERAPEUTICS

7/1/19 → 6/30/24

Project: Research project

Development of Cellular Products for Allogeneic Transplantation

Wagner, J. E. (PI) & Blazar, B. R. (CoI)

BLUEROCK THERAPEUTICS GP LLC

6/19/19 → 6/18/22

Project: Research project

Training for the Adult Subspecialist in Inherited Metabolic Disorders

Braunlin, E. A. (PI) & Orchard, P. J. (CoI)

GENZYME CORPORATION

6/17/19 → 6/17/19

Project: Research project

Pediatric Blood & Marrow Transplant & Cellular Therapy

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