Clinical development of gene therapy: results and lessons from recent successes.

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🔗 View Article (PMID 27257611)

Published in Mol Ther Methods Clin Dev on May 25, 2016

Authors

Sandeep Rp Kumar1, David M Markusic1, Moanaro Biswas1, Katherine A High2, Roland W Herzog1

Author Affiliations

1: Department of Pediatrics and Powell Gene Therapy Center, University of Florida , Gainesville, Florida, USA.
2: Spark Therapeutics , Philadelphia, Pennsylvania, USA.

Associated clinical trials:

Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728 for HIV (Zinc-Finger) | NCT00842634

Genetically Engineered Lymphocyte Therapy in Treating Patients With B-Cell Leukemia or Lymphoma That is Resistant or Refractory to Chemotherapy | NCT01029366

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (LCA) | NCT00481546

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | NCT00643747

Precursor B Cell Acute Lymphoblastic Leukemia (B-ALL) Treated With Autologous T Cells Genetically Targeted to the B Cell Specific Antigen CD19 | NCT01044069

CAR T Cell Receptor Immunotherapy for Patients With B-cell Lymphoma | NCT00924326

Treatment of Relapsed or Chemotherapy Refractory Chronic Lymphocytic Leukemia or Indolent B Cell Lymphoma Using Autologous T Cells Genetically Targeted to the B Cell Specific Antigen CD19 | NCT00466531

Phase I/IIA Study of CART19 Cells for Patients With Chemotherapy Resistant or Refractory CD19+ Leukemia and Lymphoma (Pedi CART19) | NCT01626495

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B | NCT00979238

Anti-CD19 White Blood Cells for Children and Young Adults With B Cell Leukemia or Lymphoma | NCT01593696

Administration of Anti-CD19-chimeric-antigen-receptor-transduced T Cells From the Original Transplant Donor to Patients With Recurrent or Persistent B-cell Malignancies After Allogeneic Stem Cell Transplantation | NCT01087294

Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (rAAV2-ND4) | NCT01267422

Gene Therapy for Blindness Caused by Choroideremia | NCT01461213

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | NCT02122952

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 | NCT01208389

Gene Therapy for Metachromatic Leukodystrophy (TIGET-MLD) | NCT01560182

A Study of AAV-hAADC-2 in Subjects With Parkinson's Disease | NCT00229736

Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS) | NCT01515462

Redirected Auto T Cells for Advanced Myeloma | NCT01352286

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) | NCT02161380

Phase I/II Study to Assess the Safety and Activity of Enhanced TCR Transduced Autologous T Cells in Metastatic Melanoma | NCT01350401

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B | NCT01687608

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector | NCT01129544

MAGE-A3/12 Metastatic Cancer Treatment With Anti-MAGE-A3/12 TCR-Gene Engineered Lymphocytes | NCT01273181

ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene | NCT01639690

A Study Evaluating the Efficacy and Safety of LentiGlobin BB305 Drug Product in Beta-Thalassemia Major and Sickle Cell Disease | NCT02151526

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) | NCT01175239

Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency | NCT01380990

Autologous Transplant of EFS-ADA Modified Bone Marrow Cells for ADA-Deficient Severe Combined Immunodeficiency (SCID) | NCT01852071

Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL) | NCT02453477

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID2) | NCT01410019

A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects | NCT01745120

A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC (AADC) | NCT01395641

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