‘Huge Step Back’ for Hemophilia Gene Therapy, Expert Says of FDA Delay on Roctavian
The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential…
Articles by Joana Carvalho, PhD
FDA Delays Decision on Roctavian, Potential Hemophilia A Gene Therapy, for a Year or More
The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian,…
#ISTH2020 – Gene Therapy BAY 2599023 Showing Steady FVIII Activity, Safety in Trial
A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production of factor VIII (FVIII),  effectively…
#ISTH2020 – Eloctate Effective in Previously Untreated Hemophilia A Patients, Trial Shows
Treatment with Eloctate (efmoroctocog alfa) was well-tolerated and effective at preventing and treating bleeding episodes in children with severe…
#ISTH2020 – BioMarin’s Gene Therapy Drives Sustained FVIII Production
A single infusion of valoctocogene roxaparvovec, BioMarin Pharmaceutical’s investigational gene therapy, can lead to the sustained production of…
#ISTH2020 – FLT180a Gene Therapy Shows Promise for Hemophilia B Patients in Phase 1/2 Trial
A single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained increases in the activity of…
#ISTH2020 – Hemlibra Continues to Show Favorable Safety Profile, Early Phase 3b Trial Data Show
Note: This story was updated July 20, 2020, to note that Hemlibra binds to activated FIX and FX, rather…
#ISTH2020 – Gene Therapy SPK-8011 Lowers Bleeding Rate in Hemophilia A for up to 3 Years, Trial Shows
A single dose of the investigational gene therapy SPK-8011 is safe and leads to durable production of factor VIII…
Freeline Raises $120M for Pivotal Trial of FLT180a, a Gene Therapy Candidate for Hemophilia B
Freeline has raised $120 million to support the launch of a pivotal clinical trial of FLT180a, the…
CSL Behring Acquires Rights to Market AMT-061, Gene Therapy for Hemophilia B
CSL Behring has acquired exclusive global rights to commercialize AMT-061, a potential gene therapy for hemophilia B now in…