Pediatric Blood &amp; Marrow Transplant &amp; Cellular Therapy

UNIV. OF TX M.D. ANDERSON CANCER CENTER

10/25/22 → 12/31/26

Project: Research project

REVEAL - Research Evaluating Vagal Excitation and Anatomical Linkages.

Osborn, J., Crawford, P., Darrow, D., Drawz, P. E., Dudley, S., Eberly, L., Fonkoue, I. T., Henry, T. R., Hering, B. J., Ikramuddin, S., Johnson, M. D., Keller-Ross, M. L., Lim, K. O., Lim, H. H., Loavenbruck, A. J., McCarty, C. A., Meyer, M., Mueller, B. A., Murray, T., Nahas, Z., Netoff, T. I., Panoskaltsis-Mortari, A., Park, M. C., Puchalska, P., Ramachandran, S., Singh, A., Talkachova, A., Tignanelli, C. J. & Walczak, T. S.

National Institutes of Health

9/23/22 → 8/31/25

Project: Research project

To explore the molecular and cellular effects of transient DUX4 expression in skeletal muscle

Bosnakovski, D. & Magli, A.

Assoc of Public Health Laboratories

9/1/22 → 6/30/27

Project: Research project

Mucopolysaccharidosis Newborn Screening meeting

Orchard, P. J. & Gupta, A. O.

8/23/22 → 4/30/23

Project: Research project

A Randomized Trial of Roadmap 2.0

University of Michigan

8/1/22 → 7/31/25

Project: Research project

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

Kang, P. B., Karachunski, P. I., Perlingeiro, R. & Wagner, J. E.

ST. BALDRICK'S FOUNDATION

7/15/22 → 6/30/24

Project: Research project

Engineered immune cells for safer and more effective tumor therapy

Osborn, M. J.

7/1/22 → 6/30/23

Project: Research project

In Vivo Prevention of Murine GVHD

Blazar, B. R. & Panoskaltsis-Mortari, A.

NIH Nat'l Inst of Allergy & Infect Dis

7/1/22 → 6/30/27

Project: Research project

Autologous Revertant Mosaic Fibroblasts for Wound Healing in Dystrophic Epidermolysis Bullosa

Tolar, J., Ebens, C. & Osborn, M. J.

EB RESEARCH PARTNERSHIP, INC.

6/1/22 → 5/31/23

Project: Research project

Enhancing Treg Therapeutic Efficacy in GVHD

Blazar, B. R., Griffin, T. J., Hippen, K. L. & Osborn, M. J.

National Heart, Lung, and Blood Institute

5/15/22 → 3/31/26

Project: Research project

Collection of Gene Mutations for Laboratory Quality AssuranceNewborn Screening Accuracy Project

Gupta, A. O. & Orchard, P. J.

Sequoia Foundation

5/12/22 → 5/14/23

Project: Research project

A Phase I Open Label Study to Evaluate the Safety andTolerability of ISP-001 in Patients with MucopolysaccharidosisType I

Orchard, P. J. & Miller, B. S.

IMMUSOFT CORPORATION

12/9/21 → 1/1/26

Project: Research project

Lysosomal Storage Disease Focused Training

SANOFI-AVENTIS US

11/8/21 → 11/7/23

Project: Research project

Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial, with Cross-Over, of Posoleucel (ALVR105) for the Treatment of Adenovirus Infection in Pediatric and Adult Participants Receiving Standard of Care Following Allogeneic Hematopoietic Cell Transpl

ALLOVIR, INC

10/25/21 → 10/31/26

Project: Research project

Severe Combined Immunodeficiency (SCID) Screening and Education

University of California San Francisco

8/1/21 → 7/31/23

Project: Research project

Penetrating the "Black Box": Prediction of Early Bronchiolitis obliterans in Pediatric Hematopoietic StemCell Transplant Recipients

Goldfarb, S. & MacMillan, M. L.

Cincinnati Children's Hospital

7/15/21 → 4/30/26

Project: Research project

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

Kartha, R. & Lund, T. C.

NIH NAT'L CTR FOR ADVANCING TRAN SCIENCE

7/1/21 → 5/31/23

Project: Research project

Sensory Function and Chronic Pain in Cerebral Palsy

Symons, F. J., Erickson, D. J., Panoskaltsis-Mortari, A., Simone, D. A., Walk, D. & Wilcox, G. L.

NIH NAT'L INST OF CHILD HEALTH/HUMAN DEV

5/14/21 → 2/28/26

Project: Research project

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

Kartha, R., Cloyd, J. C. & Lund, T. C.

LIGAND PHARMACEUTICALS INC

5/1/21 → 4/30/23

Project: Research project

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

Whitley, C. B., Lund, T. C., Orchard, P. J. & Ou, L.

MINA THERAPEUTICS LTD

5/1/21 → 4/29/23

Project: Research project

An Open Label, Prospective, Historically controlled, Nonrandomized Clinical Study of OTL-200 for the Treatment of Patients with Early Symptoms of Early Juvenile (EJ) Metachromatic Leukodystrophy (MLD)

Assoc of Public Health Laboratories

ORCHARD THERAPEUTICS

12/1/20 → 11/25/25

Project: Research project

National Adrenoleukodystrophy (ALD) Registry

Orchard, P. J. & Gupta, A. O.

11/26/20 → 4/30/23

Project: Research project

Correction of Fanconi Anemia Mutations using Digital Genome Engineering

Moriarity, B. S., Wagner, J. E. & Webber, B. R.

Fanconi Anemia Research Fund, Inc.

10/1/20 → 3/31/23

Project: Research project

A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to < 18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSC

Smith, A. R.

ALEXION PHARMACEUTICALS

9/20/20 → 8/31/25

Project: Research project

Skin-targeted Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa

Tolar, J., Moriarity, B. S., Osborn, M. J. & Webber, B. R.

9/1/20 → 8/31/25

Project: Research project

Probing the activity of DUX4 in FSHD

Kyba, M.

Department of Veterans Affairs

8/18/20 → 7/31/25

Project: Research project

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

Osborn, M. J.

THE GENEVA FOUNDATION

6/16/20 → 6/15/23

Project: Research project

Evaluation of the Oral and Lung Microbiota and Inflammation in Chronic Obstructive Pulmonary DiseaseFrequent Exacerbators

Panoskaltsis-Mortari, A.

6/1/20 → 5/31/25

Project: Research project

A Phase I Study to Evaluate The Safety and Tolerability of Tandemly-Purified Allogeneic CD34+CD90+ HSC Administered Following Conditioning with AMG 191 to Achieve Engraftment and Immune Reconstitution in Patients with SCID

Ebens, C. & Smith, A. R.

JASPER THERAPEUTICS, INC.

5/1/20 → 4/30/25

Project: Research project

Development and Psychometric Testing of a Pediatric Chronic Graft-Versus-Host Disease (GVHD) Symptom Scale (PCSS)

MacMillan, M. L.

WESTAT, INC.

5/1/20 → 4/30/24

Project: Research project

An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients with Hematological Malignancies

Wagner, J. E. & Brunstein, C. G.

GAMIDA CELL LTD.

4/23/20 → 4/30/25

Project: Research project

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

Moriarity, B. S., Lund, T. C., McIvor, R. S., Orchard, P. J. & Webber, B. R.

NIH Nat'l Inst of Allergy & Infect Dis

3/1/20 → 2/28/25

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

MacMillan, M. L. & Neglia, J. P.

CHILDREN'S HOSPITAL LOS ANGELES

2/1/20 → 1/31/25

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.

ROCKET PHARMACEUTICALS, INC

11/1/19 → 11/30/24

Project: Research project

Lysosomal Disease Network

Whitley, C. B., Ahmed, A., Braunlin, E. A., Cortez, D., Eisengart, J., King, K. E., Nestrasil, I. & Orchard, P. J.

NIH NAT INST OF NEURO DISORDERS & STROKE

9/15/19 → 7/31/25

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., Karachunski, P. I., McKenna, D. H., Schumacher, R. J. & Wagner, J. E.

DUCHENNE UK

9/15/19 → 7/31/23

Project: Research project

A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

Gupta, A. O. & Smith, A. R.

Bluebird Bio, Inc.

9/1/19 → 7/31/24

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

Smith, A. R.

CHILDREN'S HOSPITAL LOS ANGELES

8/16/19 → 5/31/24

Project: Research project

Training in Lung Science

Wendt, C. H., Bitterman, P. B., Ingbar, D. H. & Panoskaltsis-Mortari, A.

National Heart, Lung, and Blood Institute

8/1/19 → 7/31/24

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

MAGENTA THERAPEUTICS

7/1/19 → 6/30/24

Project: Research project

Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Bluebird Bio, Inc.

7/1/19 → 6/30/24

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)

RHEACELL GMBH & CO KG

7/1/19 → 6/30/24

Project: Research project

Skeletal Muscle Stem Cells Derived from Teratomas

Kyba, M. & Gearhart, M. D.