Rocket will also collaborate with investigators to implement an immunomodulatory regimen more closely reflecting that administered in the Phase 1 pediatric cohort
Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne
FDA requests Sarepta Therapeutics to suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths
This collaboration aims to bridge India’s talent and translational gap in this rapidly growing field by equipping professionals with practical expertise in the manufacturing of advanced therapies.
Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters
Giroctocogene fitelparvovec study meets primary and key secondary objectives of superiority compared to prophylaxis
A one-time dose of DURVEQTIX has reduced bleeds post-treatment compared to standard of care with a median annualized bleed rate (ABR) of zero bleeds (range 0 to 9.9)
The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment
Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant
The survey data presented in “The State of the Biopharmaceutical Industry 2024” report reveals that N=21 (18%) of industry professionals were convinced that CGT will dominate as the most important trend in pharma next year
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