Advocacy intelligence hub — real-time data for patient organizations
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. — NA
Skysona: FDA approved
To slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD)
Masonic Cancer Center, University of Minnesota
University of Milano Bicocca — NA
Genetix Biotherapeutics Inc.
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Skysona
(ELIVALDOGENE AUTOTEMCEL)Orphan drugGenetix Biotherapeutics Inc.
12.1 Mechanism of Action SKYSONA adds functional copies of the ABCD1 cDNA into patients' hematopoietic stem cells (HSCs) through transduction of autol...
Paul Orchard, MD, MD
University of Minnesota Medical Center
📍 MINNEAPOLIS, MN
Florian S Eichler, MD
Massachusetts General Hospital
📍 Boston, Massachusetts
Adeline Vanderver, MD
Children's Hospital of Philadelphia
📍 Los Angeles, California
Jakub M Antczak, MD
Jagiellonian University Medical College, Department of Neurology
📍 Krakow, Lesser Poland Voivodeship
Stephen Huhn, MD, MD
StemCells, Inc.
📍 STANFORD, CA
Kiran Bhirangi, MD
bluebird bio, Inc.
📍 Boston, Massachusetts