Sigilon and MIT researchers are developing a novel cell therapy designed to control bleeding and to forestall undesired immune responses.
The updated trial data shows three individuals with hemophilia B responding well to a one-time treatment with their investigational gene therapy AMT-061.
The interim trial data for SB-525 included results from the newest and highest dosing group.
This new inhibitor kit is designed to improve and standardize the laboratory measurement of FVIII inhibitors, allowing for enhanced diagnosis and routine screening.
Spark currently has investigational gene therapy candidates in clinical trials for both hemophilia A and hemophilia B.
The long-acting product will be available in the US after 2020.
Catalyst reported data from their clinical study of marzeptacog alfa (MarzAA), a subcutaneous factor VIIa therapy that is being developed for treatment of people with hemophilia A or B with inhibitors.
The donation includes product as well as integrated care development training.
The trials is for uniQure investigational gene therapy treatment for patients with severe and moderately severe hemophilia B.
The case was ruled in favor of Genentech.
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