Literature DB >> 20712582

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

J Fraser Wright1, Jennifer Wellman, Katherine A High.   

Abstract

Recombinant adeno-associated virus (AAV) -based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A recent milestone has been the safety and efficacy observed using recombinant AAV2 expressing retinal pigment epithelial associated 65KDa protein for Leber Congenital Amaurosis. This review summarizes manufacturing and characterization of 'AAV2-hRPE65v2', the vector used in one completed Phase I/II clinical trial. Regulatory challenges and strategies that were successfully used for this groundbreaking trial are described.

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Year:  2010        PMID： 20712582     DOI： 10.2174/156652310793180715

Source DB:  PubMed          Journal:  Curr Gene Ther        ISSN： 1566-5232            Impact factor:   4.391


  17 in total

Review 1.  Progress and challenges in viral vector manufacturing.

Authors:  Johannes C M van der Loo; J Fraser Wright
Journal:  Hum Mol Genet       Date:  2015-10-30       Impact factor: 6.150

2.  Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Authors:  Stephen Russell; Jean Bennett; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Sarah McCague; Dominique Cross; Kathleen A Marshall; Jean Walshire; Taylor L Kehoe; Hannah Reichert; Maria Davis; Leslie Raffini; Lindsey A George; F Parker Hudson; Laura Dingfield; Xiaosong Zhu; Julia A Haller; Elliott H Sohn; Vinit B Mahajan; Wanda Pfeifer; Michelle Weckmann; Chris Johnson; Dina Gewaily; Arlene Drack; Edwin Stone; Katie Wachtel; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2017-07-14       Impact factor: 79.321

Review 3.  Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.

Authors:  Federico Mingozzi; Katherine A High
Journal:  Nat Rev Genet       Date:  2011-05       Impact factor: 53.242

4.  Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles.

Authors:  Da Sun; Rebecca M Schur; Avery E Sears; Song-Qi Gao; Amita Vaidya; Wenyu Sun; Akiko Maeda; Timothy Kern; Krzysztof Palczewski; Zheng-Rong Lu
Journal:  Mol Ther       Date:  2019-09-12       Impact factor: 11.454

Review 5.  Interactions of nanomaterials and biological systems: Implications to personalized nanomedicine.

Authors:  Xue-Qing Zhang; Xiaoyang Xu; Nicolas Bertrand; Eric Pridgen; Archana Swami; Omid C Farokhzad
Journal:  Adv Drug Deliv Rev       Date:  2012-08-17       Impact factor: 15.470

6.  Progress in the development of novel therapies for choroideremia.

Authors:  Jasmina Cehajic Kapetanovic; Maria I Patrício; Robert E MacLaren
Journal:  Expert Rev Ophthalmol       Date:  2019-12-26

Review 7.  Therapeutic approaches to muscular dystrophy.

Authors:  Aurélie Goyenvalle; Jane T Seto; Kay E Davies; Jeffrey Chamberlain
Journal:  Hum Mol Genet       Date:  2011-03-24       Impact factor: 6.150

8.  SAFETY AND TOLERABILITY OF MRI-GUIDED INFUSION OF AAV2-hAADC INTO THE MID-BRAIN OF NON-HUMAN PRIMATE.

Authors:  Waldy San Sebastian; Adrian P Kells; John Bringas; Lluis Samaranch; Piotr Hadaczek; Agnieszka Ciesielska; Michael Macayan; Phillip J Pivirotto; John Forsayeth; Sheryl Osborne; J Fraser Wright; Foad Green; Gregory Heller; Krystof S Bankiewicz
Journal:  Mol Ther Methods Clin Dev       Date:  2014-10-15       Impact factor: 6.698

Review 9.  Manufacturing of recombinant adeno-associated viral vectors for clinical trials.

Authors:  Nathalie Clément; Joshua C Grieger
Journal:  Mol Ther Methods Clin Dev       Date:  2016-03-16       Impact factor: 6.698

10.  Gene Therapy for Duchenne muscular dystrophy.

Authors:  Julian Ramos; Jeffrey S Chamberlain
Journal:  Expert Opin Orphan Drugs       Date:  2015-10-06       Impact factor: 0.694
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