by CheckRare Staff | Feb 2, 2023
P.J. Brooks, Ph.D., Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the two main types of gene therapy currently in development: ex vivo and in vivo. Gene therapy...
by CheckRare Staff | Feb 1, 2023
Ying Huang, PhD, CEO of Legend Biotech, discusses updated results from the CARTITUDE-1 study and data from two cohorts in the CARTITUDE-2 study. Both of these studies are evaluating cilta-cel in patients with relapsed/refractory multiple myeloma. These data...
by CheckRare Staff | Jan 31, 2023
Conditioning Agent (JSP191) For Stem Cell Transplants Shows Promise in Patients With Fanconi Anemia Ronald Martell, President, and CEO of Jasper Therapeutics, discusses the positive data from the ongoing phase 1/2 study testing JSP191 as a conditioning...
by CheckRare Staff | Jan 27, 2023
Adeline Vanderver, MD, Program Director of the Leukodystrophy Center in the Division of Neurology at Children’s Hospital of Philadelphia (CHOP), and Co-Principal Investigator of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), lists key...
by CheckRare Staff | Jan 26, 2023
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