Current State of Human Gene Therapy: Approved Products and Vectors
Pharmaceuticals, 2023 In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs.
Aladdin Y. Shchaslyvyi +3 more
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Predicting potentially pathogenic effects of hRPE65 missense mutations: a computational strategy based on molecular dynamics simulations [PDF]
Journal of Enzyme Inhibition and Medicinal Chemistry, 2022 The human retinal pigment epithelium-specific 65-kDa protein (hRPE65) plays a crucial role within the retinoid visual cycle and several mutations affecting either its expression level or its enzymatic function are associated with inherited retinal ...
Giulio Poli +7 more
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Immunomodulation in Administration of rAAV: Preclinical and Clinical Adjuvant Pharmacotherapies [PDF]
Frontiers in Immunology, 2021 Recombinant adeno-associated virus (rAAV) has attracted a significant research focus for delivering genetic therapies to target cells. This non-enveloped virus has been trialed in many clinical-stage therapeutic strategies but important obstacle in ...
Wing Sum Chu, Joanne Ng
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The Multiple Criteria Qualitative Value-Based Pricing Framework "MARIE" for Novel Cell and Gene Therapy. [PDF]
Clin Pharmacol TherWhen setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies.
Takami A, Igarashi A.
europepmc +2 more sources
Photopharmacology: A new paradigm for vision restoration. [PDF]
Clin Transl MedClinical and Translational Medicine, Volume 16, Issue 8, August 2026.
Casson RJ.
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Modification chimique d’AAV pour la thérapie génique
, 2021 AAV, or adeno associated virus, are now becoming therapeutic products thanks to their properties. The hundreds of ongoing clinical trials demonstrated their efficiency and gene therapy treatments as Glybera, Luxturna and Zolgensma are now commercialised.
Leray, Aurélien
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A versatile toolkit for overcoming AAV immunity
Frontiers in Immunology, 2022 Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene therapy and has been widely used in >200 clinical trials globally.
Xuefeng Li +5 more
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Pharmaceutics, 2023 AAV gene therapy for ocular disease has become a reality with the market authorisation of LuxturnaTM for RPE65-linked inherited retinal degenerations and many AAV gene therapies currently undergoing phase III clinical trials. Many ocular disorders have a
Naomi Chadderton +12 more
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Gene-agnostic approaches to treating inherited retinal degenerations
Frontiers in Cell and Developmental Biology, 2023 Most patients with inherited retinal degenerations (IRDs) have been waiting for treatments that are “just around the corner” for decades, with only a handful of seminal breakthroughs happening in recent years.
Lindsey A. Chew +2 more
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Advanced therapy medicinal products (ATMPs) - An analysis of the global regulatory status. [PDF]
Br J Clin PharmacolAbstract Background A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process.
Cramer A +4 more
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