CliniGene NoE. European network for the advancement of clinical gene transfer and therapy (EC-FP6-NoE)www.clinigene.eu. 2012November.
2.
Cohen-HaguenauerO.2012a. Centralised regulation of gene therapy in europe. The CliniBook: Clinical Gene Transfer State-of-the-Art. Cohen-HaguenauerO.EDK: Paris, 504–516.
3.
Cohen-HaguenauerO.2012b. The necessity for data sharing towards advancement of clinical translation; building up sample IMPD and substantiating master files. The CliniBook: Clinical Gene Transfer State-of-the-Art. Cohen-HaguenauerO.EDK: Paris, 517–527.
4.
Corrigan-CurayJ., Cohen-HaguenauerO., O'ReillyM.et al.2012. Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy: summary of a symposium sponsored by the NIH Office of Biotechnology Activities and the EC DG-research NoE for the Advancement of Clinical Gene Transfer and Therapy. Mol. Ther., 20:1084–1094.
5.
European Commission, DG Health and Consumers. [a] Advanced therapies medicinal products (ATMPs) definitions are placed under EU-regulation: Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC. http://ec.europa.eu/health/documents/eudralex/vol-1/index_en.htm. 2012July.
6.
European Commission, DG Health and Consumers. [b] Eudralex Vol 1, pharmaceuticals legislation, medicinal products for human use. Manipulation listed in Annex I to Regulation (EC) No 1394/2007, first indent of Article 2.1.c. http://ec.europa.eu/health/documents/eudralex/vol-1/index_en.htm. 2012July.
7.
European Commission, DG Health and Consumers. [c] Orphan Medicinal Products: the EU regulation on orphan medicinal products. Regulation (EC) No 141/2000. http://ec.europa.eu/health/human-use/orphan-medicines/index_en.htm. 2012November.
8.
European Commission, DG Health and Consumers. [d] Medicinal products for human use. Clinical trials regulation in Europe. http://ec.europa.eu/health/human-use/clinical-trials/index_en.htm. 2012July.
9.
European Commission, DG Health and Consumers. [e] Public Health. http://ec.europa.eu/health/index_en.htm. 2012July.
10.
European Medicines Agency. [a] Advanced therapies. www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/general_content_000504.jsp&mid=WC0b01ac058050f347. 2012July.
11.
European Medicines Agency. [b] Committee for Medicinal Products for Human Use (CHMP)www.ema.europa.eu/ema/index.jsp?curl=pages/about_us/general/general_content_000094.jsp&mid=WC0b01ac0580028c79. 2012July.
12.
European Medicines Agency. [c] Community clinical trial system (EudraCT) public home page. https://eudract.ema.europa.eu/. 2012July.
13.
European Medicines Agency. [d] Draft guideline on the risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to ATMPs. EMA/CAT/CPWP/686637/2011. www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2012/01/WC500120989.pdf. 2012July.
14.
European Parliament and Council. Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004. http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2006:378:0001:0019:en:PDF
15.
KentA., KingN.M., Cohen-HaguenauerO.2011. Toward a proportionate regulatory framework for gene transfer: a patient group-led initiative. Hum. Gene Ther., 22:126–134.
16.
KingN.M., Cohen-HaguenauerO.2008. En route to ethical recommendations for gene transfer clinical trials. Mol. Ther., 16:432–438.
17.
PreLex. Monitoring of the decision-making process between institutions. http://ec.europa.eu/prelex/apcnet.cfm?CL=en. 2012December.
18.
SchneiderC.K., SalmikangasP., JilmaB.et al.2010. Challenges with advanced therapy medicinal products and how to meet them. Nat. Rev. Drug Discov., 9:195–201.
