Marketing Therapies Trials 
Drug Discovery World
SEPTEMBER 11, 2024
A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The trial also tested the safety profile of the treatment. The gene therapy itself caused mild inflammation that was treated with steroids.
Drug Discovery World
SEPTEMBER 2, 2024
DDW’s Megan Thomas discusses the future of the cell and gene therapy (CGT) sector with industry experts, who predict what to expect in 2024 and beyond. Meeting this need will become a major determinant as to what role CGT plays in the healthcare market.”
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Drug Discovery World
FEBRUARY 21, 2024
DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. PS : Most, if not all, current therapies are focused on MCI. RA: How does SNK01 work?
Drug Discovery World
JUNE 23, 2023
Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The accelerated approval follows a vote of support from the FDA Cellular, Tissue and Gene Therapies Advisory Committee and the granting of priority review status.
Drug Discovery World
JULY 12, 2024
AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy. The UK MHRA Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market, facilitating patient access.
Drug Discovery World
JUNE 1, 2023
A Nature publication confirmed that there are currently 12 FDA-approved ADCs on the market, and nine of these secured FDA approval in the past six years 2. DDW’s Megan Thomas explores three trends in the market. Varying sources suggest that China has been one to watch over recent years and continues to be going forward.
Drug Discovery World
JANUARY 30, 2024
Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.
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