The milestone reflects KFSH’s ongoing efforts to advance its gene therapy program, bridging clinical care with translational ...
A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.
A groundbreaking gene therapy trial has successfully restored hearing in individuals with a rare form of congenital deafness.
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Base-Editing Gene Therapy Shows Promise in Sickle Cell Disease
Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin ob ...
With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit. | Rocket ...
With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients.
Modifying hematopoietic stem cells to correct the underlying genetic defect, followed by reinfusion of the modified cells ...
Upon approval, Rocket has been awarded a priority review voucher, which currently fetch around $200m on the secondary market.
A new gene therapy, Celosia's CTx1000, is now being tested for ALS in a small clinical trial in Australia. The one-time treatment targets toxic TDP-43 protein clumps, a key driver of most cases of ALS ...
Discusses FDA Approval of KRESLADI Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1 March 27, 2026 8:30 ...
Early biomarker and safety data show encouraging signals with plans to present additional data in 2H 2026 AviadoBio secures ...
After years of hospital stays, these kids are thriving thanks to a gene therapy that just earned accelerated FDA approval.
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