4 ms·
My son also has hemophilia A and I'm fascinated by the possibility that he may someday join one of these trials. The increase in the pipeline of gene therapies
by akuafo 9y ago
My son also has hemophilia A and I'm fascinated by the possibility that he may someday join one of these trials.
The increase in the pipeline of gene therapies just in the past few months has been incredible.
Here are the ones I'm tracking:
BioMarin valoctocogene roxaparvovec (BMN-270)
Spark SPK-8011
Sangamo (Pfizer) SB-525
Bioverativ BIVV-001
Shire SHP-654 (BAX-888)