Paul Orchard

Professor, Department of Pediatrics
Faculty Member, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy
BMT Specialist, Pediatric Blood and Marrow Transplant (BMT) Center
Director, Mucopolysaccharidosis (MPS) Center
Team Member, Leukodystrophy Center


Dr. Paul Orchard is the Medical Director of the Inherited Metabolic and Storage Disease Program and a Professor in the Department of Pediatrics, Division of Blood and Marrow Transplant & Cellular Therapy. Dr. Orchard's focus is in the use of hematopoietic stem cell transplantation and other cell therapies for inherited metabolic disorders, with a special interest in the inherited leukodystrophies, the mucopolysaccharidoses and osteopetrosis. His clinical research is in evolving new and combination therapies to decrease toxicity and improve outcomes, with a particular interest in the use of gene therapy approaches.

Research Summary

Hematopoietic Cell Transplantation of Genetic Disorders Transplant related toxicity and graft failure Use of reduced intensity preparative regimens Risk factors affecting outcomes following transplantation
Osteopetrosis Biology and genetics of osteopetrosis Correlations of clinical and genetic parameters to outcome and prognosis Hematopoietic transplantation and other therapies
Gene Therapy T cell engineering with "suicide genes" for transplantation Infusions of mismatched T cells with following genetic manipulation Treatment of genetic and metabolic diseases with gene therapy
Development of Alternative Cellular Therapy Use of mesenchymal stem cells for genetic diseases Infusions of immunologically active cell populations

Clinical Summary

Bone marrow transplants to treat genetic diseases; Osteopetrosis; Inherited conditions


MD, Brown University Hospital, Providence RI

Fellowships, Residencies, and Visiting Engagements

Residency in Pediatrics,
University of Wisconsin Hospital
Madison, Wisconsin, United States
Fellowship in Hematology-Oncology and Blood and Marrow Transplantation,
University of Minnesota Medical School
Minneapolis, Minnesota, United States

Licensures and Certifications

American Board of Hematology/Oncology
Pediatrics, American Board of Pediatrics

Honors and Recognition

Best Doctors in America®
Team Science Award, Department of Pediatrics, University of Minnesota
Minneapolis/St. Paul Magazine 2022 Health Care Hero, Innovator, Minnepaolis/St. Paul Business Journal
UMMC Clinical Research and Innovation Award
Minneapolis - St. Paul Magazine “Top Doctors”, Mpls. St. Paul Magazine

Media Appearances

Groundbreaking ALD Therapy, KSTP
5-year-old who received groundbreaking gene therapy travels back to Minnesota to pay it forward, KARE11
On the eve of historic medical treatment in MN, family's insurance pulls out, KARE11
The Balancing Act - Severe malignant osteopetrosis awareness, Lifetime
Selected Publications

Selected Publications

Engelen, M., van Ballegoij WJC, Mallack, E. J., Van Haren, K. P., Köhler, W., Salsano, E., van Trotsenburg ASP, Mochel, F., Sevin, C., Regelman, M. O., Tritos, N. A., Halper, A., Lachmann, R. H., Davison, J., Raymond, G. V., Lund, T., Orchard, P. J., Kuehl, J. S., Lindemans, C. A., Caruso, P., Turk, B. R., Moser, A. B., Vaz, F. M., Ferdinandusse, S., Kemp, S., Fatemi, A., Eichler, F. S., Huffnagel, I. C., 2022. International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach.. Neurology,
Chiesa, R., Boelens, J. J., Duncan, C. N., Kühl, J. S., Sevin, C., Kapoor, N., Prasad, V. K., Lindemans, C. A., Jones, S. A., Amartino, H. M., Algeri, M., Bunin, N., Diaz-de-Heredia, C., Loes, D. J., Shamir, E., Timm, A., McNeil, E., Dietz, A. C., Orchard, P. J., 2022. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.. Blood advances, 6 (5): 1512-1524.
Wiersma, R., Gupta, A. O., Lund, T. C., Orchard, P. J., Miller, B. S., 2021. Adrenal Insufficiency in an Adolescent Boy With Type 1 Diabetes Mellitus - the Importance of Considering X-Linked Adrenoleukodystrophy. (ENDO 2021). Journal of Endocrine Society,
Hampe, C. S., Wesley, J., Lund, T. C., Orchard, P. J., Polgreen, L. E., Eisengart, J. B., McLoon, L. K., Cureoglu, S., Schachern, P., McIvor, R. S., 2021. Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement. Special Issue Lysosomal Storage Disorders: Molecular Basis and Therapeutic Approaches Biomolecules 2021, 11 (2): 189.
Gupta, A. O., Boelens, J., Ebens, C. L., Kurtzberg, J., Lund, T. C., Smith, A. R., Wagner, J. E., Wynn, R., Blazar, B. R., Orchard, P. J., 2021. Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders.. Bone marrow transplantation,
doi: PubMed ID: 33441980.
Lund, T. C., Doherty, T. M., Eisengart, J. B., Freese, R. L., Rudser, K. D., Fung, E. B., Miller, B. S., White, K. K., Orchard, P. J., Whitley, C. B., Polgreen, L. E., 2020. Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I. JIMD Reports,
Foy, A., Elizondo, E., Kroening, S., Rogers, D., Keeny-Jung, D., Nascene, D., Nestrasil, I., Orchard, P. J., Lund, T. C., Gupta, A. O., Pierpont, E. I., Characteristics of seizures among boys with standard risk childhood cerebral adrenoleukodystrophy treated with hematopoietic cell transplantation. (American Epilepsy Society meeting). American Epilepsy Society,
Drozdov, D., Long, S. E., Gupta, A. O., Lund, T. C., Boelens, J., Orchard, P. J., Bodyweight and absolute lymphocyte count based dosing of rabbit anti-thymocyte globulin results in early cd4+ immune reconstitution in patients with inborn errors of metabolism undergoing umbilical cord blood transplantation. (Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, February 15-19, 2023, Orlando, Florida). American Society of Transplant and Cellular Therapy,
Long, S. E., Lund, T. C., Gupta, A. O., Boelens, J., Orchard, P. J., Bodyweight/ absolute lymphocyte count based dosing of Anti-Thymocyte Globulin (ATG) results in early CD4+ immune reconstitution in patients with inborn errors of metabolism undergoing umbilical cord blood transplantation. (ASH, December 10 2022, New Orleans, LA). American Society of Hematology,
Firoozmand, A., O'Leary, D., Cao, Q., Gupta, A., Ebens, C., Maakaron, J., Betts, B., Lund, T., Bachanova, V., MacMillan, M., Miller, J., Orchard, P., Wagner, J., Vercellotti, G., Weisdorf, D., Dusenbery, K., Terezakis, S., Holtan, S., El Jurdi, N., Juckett, M., Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning with Cyclophosphamide/ Fludarabine/2 Gy Total Body Irradiation (TBI) preparative regimen with Tacrolimus, MMF with or without ATG for the Treatment of Hematological Ma. (ASH, December 10 2022, New Orleans, LA). American Society of Hematology,
Hoover, A., O'Leary, D., Cao, Q., Gupta, A., Ebens, C., Maakaron, J., Betts, B., Juckett, M., Lund, T., Bachanova, V., MacMillan, M., Miller, J., Orchard, P., Wagner, J., Vercellotti, G., Weisdorf, D., Dusenbery, K., Terezakis, S., Holtan, S., El Jurdi, N., Phase II Study of Myeloablative 8/8- or 7/8-Matched Allotransplantation with Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil: Marked Reduction in GVHD Risk Without Increase Relapse Risk Compared to Historical Cyclosporine/Methotrex. (ASH, December 10 2022, New Orleans, LA). American Society of Hematology,
El Jurdi, N., O'Leary, D., He, F., de For, T., Rashidi, A., Warlick, E., Gupta, A., Maakaron, J., Janakiram, M., Slungaard, A., Smith, A., Bachanova, V., Brunstein, C., MacMillan, M., Miller, J., Betts, B., Ebens, C., Stefanski, H., Lund, T., Orchard, P., Vercellotti, G., Weisdorf, D., Holtan, S., Low Incidence of Chronic Graft-versus-host Disease in Myeloablative Allogeneic Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide using Matched Related or Unrelated Donors: Phase II Study Interim Analysis.
Selected Presentations

Selected Presentations

Orchard, P. J., (Author & Presenter) "The Inherited Leukodystrophies: Can We Do Better?", Pediatric Grand Rounds, University of Minnesota, Department of Pediatrics, Minneapolis, Minnesota, United States. August 10, 2022.
Orchard, P. J., (Author), Lund, T. C., (Author) "Blood Stem Cell Transplantation & Gene Therapy for Hurler Syndrome", United Leukodystrophy Foundation (ULF) Scientific Symposium , Itasca, Illinois, June 24, 2022.
Orchard, P. J. "Krabbe Disease", Newborn Screening Advisory Committee - Spring Meeting, Minnesota Department of Health, Saint Paul, Minnesota, May 31, 2022.
Kaczmarcyk, A., Lasio, L., Viskochil, D., Longo, N., Lund, T., Orchard, P. J., Yang, A., Chang, I., C., Pedro, H., Aliu, E., Siemon, A., Mori, M., Dickson, P., Al-Hertani, W., Ahrens-Nicklas, R., Pasquali, M. "Analysis of urine heparan sulfate and its non-reducing ends for the follow-up of abnormal newborn screening for MPS1", SIMD 43rd Annual Meeting, Society for Inherited Metabolic Disorders, Orlando, Florida, April 10, 2022.
Orchard, P., (Panelist) "Metachromatic Leukodystrophy and Newborn Screening", Minnesota State Legislature, Orchard Therapeutics and CureMLD, St. Paul, Minnesota, February 24, 2022.
Orchard, P. J. "Emerging Developments in the Treatment of Cerebral Adrenoleukodystrophy", Newsteps New Disorders & Short-Term Follow-up National Meeting, Association of Public Health Laboratories, August 26, 2021.
Orchard, P. J., Miller, B. S. "Adrenoleukodystrophy: Advancements in the Care of a Children with a Rare Disease", Pediatric Grand Rounds, University of Minnesota Medicine School, Department of Pediatrics, Minneapolis, Minnesota, July 14, 2021.
Takahashi, T., Illamola, S., Jennissen, C. A., Long, S. E., Lund, T. C., Orchard, P. J., Long-Boyle, J. R., Gupta, A. O. "Busulfan Dose Recommendation in Inherited Metabolic Disorders: Population Pharmacokinetic Analysis", PRESS 2021, University of Minnesota Department of Pediatrics, April 23, 2021.
Orchard, P. J. "Clinical aspects of lentiviral gene therapy: What to expect?", 17th Annual WORLD Symposium - Satellite Symposia, Avrobio, Inc., February 10, 2021.
Orchard, P. J., Duncan, C. N., Eichler, F. S., De Oliveira, S., Thrasher, A. J., Sevin, C., Amartino, H. M., Smith, N. J., Kühl, J., Kenney-Jung, D. L., Chiesa, R., Dalle, J., Xu, J., Dietz, A. C., McNeil, E., Aubourg, P., Williams, D. A. "Elivaldogene Autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Updated Results from the Phase 2/3 ALD-102 Study and First Report on Safety Outcomes from the Phase 3 ALD-104 Study", 2021 Transplantation & Cellular Therapy Meetings, ASTCT and CIMBTR, February 09, 2021.
Pillai, N., Orchard, P. J., Lund, T. C., Ahrens-Nicklas, R., Elsea, S., Whitley, C. B. "Evaluation of the Effectiveness of Bone Marrow Transplantation in Multiple Sulfatase Deficiency", 17th Annual WORLD Symposium, February 09, 2021.
Orchard, P. J., Gupta, A. O. "ALD National Registry Update", ALD Standards of Care Meeting 2021, Aidan Jack Seeger Foundation, Brooklyn, New York, January 14, 2021.
Van Haren, K., Bonkowsky, J., Lund, T. C., Orchard, P. J., Parker, A. "Panel: Future Therapies - gene therapy for cerebral ALD, gene therapy for AMN, small molecule therapy", ALD Family Weekend, The Painted Turtle, May 03, 2020.
Grants and Patents

Grants and Patents


Chimeric Genes Facilitating the Expression of NGF Receptor and Cystosine Deaminase Gene Function. Issued: 2001