Gene-addition and gene-editing therapies are currently in clinical development.
NEW ORLEANS – Children and young adults with non-beta0/beta0 thalassemia achieved transfusion independence in two trials.
The Recombinant DNA Advisory Committee will now be known as the Novel and Exceptional Technology and Research Advisory Committee.
From the Journals
In the phase 1/2 study, seven infants had normal immune cells 3-4 months after treatment, while the eighth infant responded after a gene therapy...
Movers in Medicine
Hematology and oncology researchers will receive awards at the 2019 American Society of Pediatric Hematology/Oncology conference.
Two of eight patients treated with the investigational gene therapy SB-525 have achieved normal factor VIII levels, according to interim study...
HOUSTON – Results from the small study provide a “proof of concept” for the use of gene therapy in X-linked chronic granulomatous disease,...
PRAGUE – The biggest question for gene therapy today is durability, according to Dr. John Pasi.
European approvals of CAR T-cell therapy are prompting a closer look at economic issues, outcomes data, and future directions.
PRAGUE – Early data shows clinically meaningful factor IX activity in men with hemophilia B.
A final decision on the proposal is expected by May 2019.