United Kingdom Human Trials began November 2011:

United Kingdom Pre-clinical Studies:


Limited info on United Kingdom MHRA (Medicines and Healthcare products Regulatory Agency) guidances:

EU EMA Orphan Drug Designations: There are two EMA ODD favorable opinions for CHM AAV2 gene therapy both published on 15 July 2014:

US Human Trials at the University of Miami, Bascom Palmer Eye Institute using the UK AAV2/CHM vector:

Phase I/II Human trials at the Children's Hospital of Philadelphia began February 2015:
United States Pre-clinical Studies:

FDA Orphan Drug Designation for Children's Hospital of Pennsylvania and Spark Therapeutics, Inc. - adeno-associated viral vector, serotype 2, containing the human choroideremia gene encoding human Rab escort protein 1 - 9/12/2013: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm?Index_Number=404713

Canadian Human Trials using the UK AAV2/CHM vector:

AAV5 Human iPS, RPE models and pre-clincal studies at Inserm in Montpellier France
Nicolas Cereso, Pequignot, Robert, Becker, De Luca, Nabholz, Rigau, De Vos, Christian P Hamel, Kalatzis:
  • The characterization of genomic DNA from the fibroblasts of patient CHM1 reveals duplication and deletion of sequences at the intron 7-exon 8 junction that lead to loss of the intron 7 acceptor splice site and thus deletion of exon 8 - First creation of human CHM iPS and RPE models.
  • CHM-AAV5-_Hamel_Kalatzis_etal-Molecular Therapy-Apr 2014.pdf

AAV8 - IP owned by Regenx Bio http://regenxbio.com/ - The University of Pennsylvania is a co-founder of Regenx Bio as is Dr. James Wilson of U Penn (http://www.med.upenn.edu/gtp/vectorcore/):
  • AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis - Boye SL, Peshenko IV, Huang WC, Min SH, McDoom I, Kay CN, Liu X, Dyka FM, Foster TC, Umino Y, Karan S, Jacobson SG, Baehr W, Dizhoor A, Hauswirth WW, Boye SE - Hum Gene Ther. 2013 Feb - http://www.ncbi.nlm.nih.gov/pubmed/23210611
  • AAV8-mediated gene therapy for Choroideremia: Preclinical studies in in vitro and in vivo models - Aaron Black,Vidyullatha Vasireddy,Daniel C. Chung, Albert M. Maguire, Rajashekhar Gaddameedi, Tania Tolmachova, Miguel Seabra, Jean Bennett-Jrnl of Gene Medicine-June2014: http://www.ncbi.nlm.nih.gov/pubmed/24962736 Full text: http://onlinelibrary.wiley.com/doi/10.1002/jgm.2768/full