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Gene Therapy update

American Society of Gene & Cell Therapy (ASGCT) 2009-2010 President Ken Cornetta presents up to date membership concerns in his recent Editorial about the ASGCT mission of moving gene and cell technologies into clinical practice, (1) observing that:

Clearly, the NIH leaders are aware of the advances in our field. …We pointed out that many of the recent clinical successes were achieved in Europe, which reflects, in part, on the manner in which translational research is funded in the United States. Rather than viewing preclinical development, vector manufacture, toxicology studies, and early-phase clinical trials as a continuum, most institutes require investigators to submit multiple, independent grant applications for each element of translational research. This system often leads to a catch-22 scenario and challenges the five-year limit on many grants. …
Regulatory oversight topics he discusses include:
-now in translational research, investigators routinely perform work that includes animal studies, recombinant DNA, and human research, by consolidating review committees, the quality of a review can be improved, also resulting, in a considerable cost savings,
- difficulty for investigators to identify the most relevant and efficient pathway to move translational research forward, also of concern to NIH leaders,
- adapting to licensed gene and cell therapy products is a challenge that we all welcome.
- stem cell science and cell therapies are rapidly growing.
Some other int'l perspectives on gene therapy:
- European Medicines Agency (EMA) regulatory initiatives, including a draft concept paper on the development of a guideline on the risk-based approach (according to an EC directive) applied to Advanced Therapy Medicinal Products (2)
- International Conference on Harmonization (ICH) Steering Committee established its Gene Therapy Discussion Group (GTDG) to lead these activities within the ICH.(3) Objectives include proactively setting out principles that may have a beneficial impact on harmonizing regulations of gene therapy products and developing new ways of communication to ensure that the outcomes of ICH are well understood and widely disseminated such as Public ICH gene therapy workshops, establish a publicly available ICH gene therapy web page.
-FDA requested public comment as a part of its preparations for the June 2010 ICH meeting. (4)
(1)http://www.nature.com/mt/journal/v18/n8/full/mt2010158a.html 2010 Gene Therapy Year in Review,
(2) http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2010/01/WC500069264.pdf, Committee for Medicinal Products for Human Use(12/09)
(3) http://www.ich.org/cache/compo/276-254-1.htmlhttp://www.ich.org/cache/compo/276-254-1.html
The next formal meeting of ICH GTDG will take place in parallel with the ICH SC and EWG meetings in Japan,Nov 2010.
(4) http://www.fda.gov/Drugs/NewsEvents/ucm204924.htm Also there, see Page 7 of 20 slides: Global Harmonization Efforts beyond ICH,by Justina A. Molzon, MS Pharma, JD, FDA/CDER/OEP.

Excerpted from the ICH press release about their June, 2010 meeting, below are brief examples of main achievements.
Quality of Pharmaceuticals -This meeting was highlighted by major progress in the global implementation of the ICH Guidelines Q8 (Pharmaceutical Development), Q9 (Quality Risk Management) and Q10 (Pharmaceutical Quality System) in an integrated way. To this end the ICH Quality Implementation Working Group (IWG) held its first training workshop in Tallinn. The training consisted of a case study representing the different phases of the life cycle of a pharmaceutical product. The workshop was appreciated by the 240 participants from 34 countries, with 100 assessors and inspectors from 32 different health authorities and 140 industry experts representing 45 companies. The training will also be held in the other two ICH regions, with the next workshop in Washington , D.C. on 6-8 October 2010, followed by Tokyo on 25-27 October 2010.
Further progress was made on the harmonization of pharmacopeial text in the three regions, which will reduce testing requirements for industry.
Non-Clinical Safety -The ICH Steering Committee approved the establishment of an EWG for a new multidisciplinary topic (M7) on Genotoxic Impurities. The guideline will describe the evaluation, qualification and control of these impurities in medicines during development and after licensing.
Clinical Safety and Efficacy- addressing issues arising from the E7 guideline on Studies in Support of Special Populations: Geriatrics.

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