Article

Bbarrons.com on 2021-01-11 16:20

BioMarin’s Study of Its Hemophilia Gene-Therapy Looked Good

The company hopes to persuade the FDA to consider approval based on one-year data—instead of requiring two years of follow-up on trial participants.

Related news

BBioMarin Slumps After FDA Rejects Its Hemophilia Gene Therapy barrons.com
Phase 1 , Wall Street
FDA blocks much-anticipated BioMarin hemophilia gene therapy dailyherald.com
San Rafael
Health Canada approves Pfizer's gene therapy for treatment of hemophilia CBC
Ottawa , Canada
Promising gene therapy against hemophilia rejected by FDA bizjournals.com
$3.5M gene therapy for hemophilia gets FDA approval seattletimes.com
Therapy for patients with hemophilia supplies gene essential to blood clots pressherald.com
Toronto , Massachusetts , Cambridge , Philadelphia
After surprise rejection of hemophilia gene therapy, BioMarin asks FDA to approve dwarfism drug bizjournals.com
A hemophilia patient hero at zero, BioMarin to seek FDA OK for gene therapy. But will the effect last?bizjournals.com
Death in CRISPR gene therapy study sparks search for answers staradvertiser.com
New York , Montour Falls
UniQure gets $450M upfront to license hemophilia B gene therapy bizjournals.com
BioMarin delays refiling approval request for hemophilia gene therapy bizjournals.com
Spark, Pfizer amend agreement for experimental hemophilia gene therapy bizjournals.com
St. Jude prepares to launch global hemophilia gene therapy trial bizjournals.com
New UNC spin-out targets hemophilia cure through gene therapy bizjournals.com
BFDA Delays Start of Sarepta’s Gene Therapy Trial barrons.com
Wall Street
Gene therapy to fight a blood cancer succeeds in major study reviewtimes.com
Florida , Tampa , Europe