Sage Journals: Discover world-class research

Abstract

Industry Wire

Amsterdam Molecular Therapeutics (AMT) (Amsterdam, The Netherlands) entered into an agreement with the Institute Pasteur (Paris, France). AMT will support the manufacturing and testing of clinical lots of AAV serotype 5 encoding alpha-N-acetylglucosaminidase for a Phase I/II study in Sanfilippo B patients. Sanfilippo B, one of the mucopolysaccharidosis type III diseases, is a genetic disorder that leads to the accumulation of heparan sulfate glycosaminoglycans which in turn leads to neuronal dysfunction and cell death. This rare disease manifests itself in newborns and young children, whose lifespan often does not exceed the second decade. Currently supportive treatment is the only clinical option for these patients with stem and bone marrow, cell and gene therapies being considered.

Cell Medica (London, UK), a private consortium of groups in industry and academia focused on delivering cellular immunotherapies for treatment of infectious diseases and cancer, announced the signing of an exclusive licensing agreement and research interaction with the Center for Cell and Gene Therapy at Baylor College of Medicine in Houston, Texas. The deal is centered on the commercialization and development of cell-based treatment of cancers using oncogenic Epstein Barr virus vectors.

Global Industry Analysts, Inc (San Jose, CA), a publishing company that provides research reports to the investment, manufacturing and consultancy world, estimates the global market for gene therapy products to reach US$ 316 Million by 2015. The press report on the $4500 report cites the demand for novel therapies for unmet needs, the first products in the process of approval and the recent success in early stage trials as contributing factors.

Genzyme (Cambridge, MA) and Sanofi-Aventis (Paris, France) confirmed they are negotiating a merger deal based on contingent value right. This would mean that a share price of $80 would be offered instead of the previous offer of $69 per share if Genzyme meets predefined milestones. Central to this discussion is alemtuzumab, Genzyme's promising multiple sclerosis drug, which will be marketed as Lemtrada.

Vical (San Diego, CA) and AnGes (Osaka, Japan) announced that its phase 3 clinical trial of Collategene received the green light from the US Food and Drug Administration. Collategene is a DNA based angiogenesis product for patients suffering from advanced peripheral arterial disease. The hepatocyte growth factor gene is delivered locally to vasculature with restricted blood flow. AnGes, which licenses technology on this product from Vical, is pursuing late stage clinical trials for this product in Canada, Europe, Latin America, the US, and Japan.

Regulatory Wire

FDA Creates New Education Website for Gene and Cell Therapy

The FDA's office of cellular, tissue and gene therapies (OCTGT) has established a new website containing online educational courses for parties interested in developing gene and cell therapies for clinical application. There are currently eight learning modules on the site. Topics covered range from the basics of how to submit an investigational new device (IND) application, to guidance on the development of quality gene and cell therapy products, to the preclinical requirements that must be met for a successful IND submission. More information regarding this new initiative by OCTGT can be found at: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ucm232821.htm.

NIH and FDA to Hold Workshop on Pluripotent Stem Cells

The National Institutes of Health and the Food and Drug Administration are collaborating on a series of workshops on moving pluripotent stem cell therapies into the clinic. The first two-day workshop, “Pluripotent Cells in Translation: Early Decisions,” is scheduled for March 21 and 22 at Lister Hill Auditorium on the NIH campus in Bethesda, Maryland. The first day will cover pluripotency and its challenges, sources of pluripotent cells, and stem cell banks; the second day will cover making pluripotent stem cells into a product, product characterization, and emerging technologies. The meeting will be open to participation from industry, academic and clinical scientists, the FDA, and the NIH. Space is limited, and registration is required. The registration deadline is March 7, 2011. Online registration may be completed at: http://www.cvent.com/EVENTS/Info/Summary.aspx?e=942182d8-4b08-4a79-8f98-2a3c9df62678.

National Cancer Institute Overhauls Clinical Trial Program

The National Cancer Institute (NCI) announced major changes to be made in the long-established Clinical Trials Cooperative Group Program that conducts many of the trials of new cancer therapies in the United States. In a major transformation, NCI intends to consolidate the nine groups that currently conduct trials in adult cancer patients into four state-of-the-art entities that will design and perform improved trials of cancer therapies.

The NCI Cooperative Group program, founded over 50 years ago, involves more than 3,100 institutions and 14,000 investigators, and the program enrolls over 25,000 patients in clinical trials each year. Four pediatric groups were consolidated into one group a number of years ago, and that sole pediatric group will not be consolidated with other groups.

According to NCI officials¹, consolidation is intended to improve the efficiencies of operations centers, data management centers, and tumor banks, and the changes will take into consideration an assessment of all currently active cooperative groups. The current groups will also be given opportunities to comment on the proposed changes and to explore specific aspects of the reorganization plans in consultation with NCI leadership.

A list of the current 10 U.S.-based NCI Cooperative Groups can be found at: http://www.cancer.gov/cancertopics/factsheet/NCI/clinical-trials-cooperative-group.

http://www.nih.gov/news/health/dec2010/nci-23.htm.

a-0

European Medicines Agency Publishes ‘Road map to 2015’

The European Medicines Agency (EMA) has published its final 'Road map to 2015', coinciding with the 16th anniversary of its inauguration on January 26, 2011. The road map was drafted in consultation with the Agency's European partners, stakeholders, including patients' and doctors' organizations as well as pharmaceutical industry, and the public, to ensure a broad consensus on the best approach to be taken for the Agency to fulfill its public mandate to protect and promote public health in the European Union over the coming years.

The ‘Road map to 2015’ sets out the Agency's vision in further developing its role as a European public-health agency in the field of medicine. The new road map proposes priority areas for future actions to strengthen the Agency's role in protecting and promoting human and animal health in the European Union. At present the EMA's top three aims are: meeting public health needs, improving access to medicines and optimizing their safe use.

The road map highlights the increasing complexity of the EMA's tasks, specifically managing and harmonizing interactions between all of EMA's individual components; the Agency is made up of six scientific committees and 35 working parties and other groups to which it provides scientific support. A complete copy of the map can be found at: http://www.ema.europa.eu/docs/en_GB/document_library/Report/2011/01/WC500101373.pdf.