news on AAV trial death
on death of patient in AAV-vector trial
setbacks in gene therapy trials
news in the debate on gene therapy of SCID
Report on the Life Sciences 2006 meeting
The ANGT took part in the annual meeting in Salzburg from September 25th to 27th, along with the other Austrian societies representing the Life Sciences.
The Gene Therapy session was a resounding success, with a blend of established, internationally renowned scientists and young home-grown talent providing insights into various aspects of gene therapy from the research bench to clinical application.
The session was opened with the Austrianova Gene Therapy Lecture, which was this year delivered by Professor Srinivasan Chandrasegaran from the John Hopkins University in Baltimore, USA. He is the founding father of the highly promising Zinc Finger Nuclease (ZFN) technology and gave an excellent talk taking the audience on a journey from the very early days of his discovery through to potential future applications such as gene therapies for X-SCID and HIV.
This plenary lecture was followed by a short talk from Dr. Sundar Durai from Pondicherry University in India, who continued in the ZFN vein by presenting his work on systems for the high-throughput selection of functional Zinc Finger Proteins (ZFPs), which can then be used for the generation of novel ZFNs.
The second short talk was given by Dr. Karoline Lipnik from the University of Veterinary Medicine in Vienna, Austria, who shared her latest findings on the novel anti-tumour effects of the human guanylate binding protein (GBP)-1.
This was then followed by the really stimulating plenary lecture of Stefano Ferrari from the Veneto Eye Bank Foundation in Venice, Italy, which demonstrated the power of stem cells to regererated skin and eye injuries, and how a combination of the advances in tissue engineering, stem cell technology and the application of gene therapy to keratinocyte stem cells could produce a future therapy for Epidermolysis Bullosa (EB).
The session was wrapped up by a short talk from another homegrown “Nachwuchsforscher”, Irene Pfaffenzeller from the University of Natural Resources and Applied Life Sciences (BOKU) in Vienna, Austria, who shared the insights which she has gained from her work on the development of antibiotic-free plasmid production techniques, which could have far-reaching implications for gene therapy using non-viral delivery of the therapeutic gene.
Ark begins marketing authorisation application for glioma gene therapy product
London, UK, 1 September 2006 - Ark Therapeutics Group plc ('Ark' or the 'Company') today announces that it has filed its response to the first series of questions raised by the EMEA's scientific committee as part of the marketing authorisation application (MAA) review process for Cerepro(TM), its novel gene-based medicine for the treatment of operable high grade glioma (brain cancer). Ark's response has been filed in accordance with the standard procedure and time frame. The application for marketing approval was submitted by Ark in the second half of last year and, following validation of the submission by the EMEA, was accepted for review in October 2005.
To facilitate the response, Ark's Finnish manufacturing facility has manufactured the necessary 'conformity' batches of Cerepro(TM) to commercial supply specifications. The Phase II Cerepro(TM) study, which forms the main clinical evidence in the submission, has also been subject to a GCP inspection as a specific part of the MAA review process. In addition, Ark's headquarters has satisfactorily completed a full good clinical practice ('GCP') system inspection under the new EU pharmaceutical regulations.
Cerepro(TM) has completed three clinical studies during its development to date: a Phase I study establishing safety and posology (dosing and method of administration) and two safety and efficacy studies. Cerepro(TM) treatment produced an average extension of 7.5 months of life, almost doubling survival time in a disease where most patients will only live for around eight months.
Cerepro(TM) has Orphan Drug Status in Europe and the USA and is the first gene medicine in the world (1) to undergo a formal MAA review.
Dr Nigel Parker, CEO of Ark, commented: 'This review is significant because it gives clarity to the regulatory requirements and standards that need to be met for gene-based medicines, a new and exciting class of biological drugs in which Ark is rapidly becoming recognised as a world leader. We continue to make significant advances with Cerepro(TM) and look forward to providing further updates on its overall progress in due course.'
Notes (1) ex-China
Life Sciences 2006, Salzburg, September 25th- 27th
The ANGT is again teaming up with the other Austrian life-sciences based societies (ÖGBM, ÖGGGT and ÖGBT) to take part in the 2006 Life Sciences meeting, which will be held at the University of Salzburg from the 25th to the 27th of September. Invited speakers working in the field of gene therapy will include Srinivasan Chandrasegaran (USA) and Michele De Luca (Italy). This meeting will also provide Austrian scientists with the chance to present their work , both in oral and poster presentation formats. Abstracts can be submitted until the 10th of July, and registration for the meeting at a reduced fee is possible until August 28th. More information can be found on the meeting homepage using the following link: http://www.ogbm.org/Jahrestagung.
The results of a clinical trial published
The results of a clinical trial published in the April edition of Nature Medicine provides a novel insight into the role of retroviral insertional mutagenesis in gene therapy. Until now, the activation of non-target genes due to the random nature of integration has always been viewed as an unavoidable and purely negative side effect of using retroviruses as gene therapy vectors. This new study, which has apparently cured two adult men from the rare immunodeficiency, chronic granulomatous disease (CGD), however, indicates that the serendipitous activation of genes that promoted proliferation and differentiation of the treated cells played an important role in the success of the trial. The original paper can be downloaded here , and an editorial on the subject published in the same journal can be downloaded here.
therapy and oncolytic viral therapy products approved in China