Robert Califf, MD, Nominated for FDA Commissioner
The professor of medicine at Duke University School of Medicine previously served as commissioner in 2016.
High Incidence of C Difficile Following Allogeneic Stem Cell Transplant
Patients with CDI also had more severe acute graft vs. host disease than those without CDI.
New Study Shows Potential of Gene Therapy for Hemophilia B
FIX activity was similar in participants without and with pre-existing neutralizing antibodies.
Sickle Cell Disease Gene Therapy on Hold After Adverse Events
Following 2 unexpected serious adverse events, the trial was put on clinical hold as of February 2021.
Promising New Gene Editing Techniques for Sickle Cell
Researchers observed 20-25% frequencies of this 4.9 kb deletion in HSPCs prior to xenotransplantation and at 17 weeks post-engraftment.
FDA Approves BCMA-Directed Cell Therapy for Multiple Myeloma
Multiple myeloma is currently incurable with many patients experiencing relapses.
New Gene Therapy Beneficial for Adolescent Patients with Cerebral Adrenoleukodystrophy
The investigators found the primary efficacy endpoints occurred in 20 evaluable patients (87%).
FLT201 Shows Promise as a Gene Therapy for Gaucher Disease
FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.
Preliminary SPK-8016 Data Shows Promise Treating Hemophilia A
The 1 patient who did not received immunomodulatory agents demonstrated the highest level of FVIII activity.
Gene Therapy Study for Monogenic Blindness Fails to Meet Endpoint
The researchers unexpectedly found sustained visual improvement in both eyes, despite only 1 eye being treated with AAV.
New Gene Therapy Shows Promise Treating DMD
Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.
Ongoing Trial Assesses New Anti-VEGF Gene Therapy Injection for nAMD
No serious adverse events reported through 24 weeks of a 52 week trial.
Bispecific CAR-T Cell Therapy Shows Promise Treating RRMM
During the annual ASH meeting, investigators present phase 1 data from a new dose-climbing trial.
