Donald Kohn

Primary Immune Deficiency Treatment Consortium

National Institute of Allergy and Infectious Diseases · U54

PI · $1.7M · University of California, San Francisco

Training in Developmental Hematology

National Heart, Lung, and Blood Institute · T32

Contact PI · $2.7M · University of California Los Angeles

Disseminating Curative Biological Therapies for Rare Pediatric Diseases

National Center for Advancing Translational Sciences · U01

PI · $6.7M · Boston Children's Hospital

EFS-ADA Lentiviral Vector Transduction of Bone Marrow CD34+ Cells for ADA-SCID

National Institute of Allergy and Infectious Diseases · U01

Contact PI · $3.2M · University of California Los Angeles

Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

Principal investigator · Active not recruiting

Rocket Pharmaceuticals Inc.

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

Principal investigator · Terminated · Phase 1

Rocket Pharmaceuticals Inc.

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Principal investigator · Completed · Phase 1, Phase 2

Rocket Pharmaceuticals Inc.

Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

Principal investigator · Unknown

National Institute of Allergy and Infectious Diseases (NIAID)