genOway : News room

Major scientific breakthrough will deliver better medicines to the world
- Published in Science today -

genOway (Lyon, France), Europe's leading provider of transgenic cellular and animal models, and its partner INRA (Institut National de la Recherche Agronomique), announce that they have succeeded in producing the world's first cloned rats. Details of this major scientific breakthrough are published today in Science. The rat is one of the most widely used animal models in scientific research. Cloning will aid in the development of genetically modified rat models of greater predictability and quality. Scientific results obtained from these models will contribute to the development of innovative therapeutics for major pathologies such as cardiovascular diseases, cancers, obesity, diabetes and neurological disorders.

genOway has been able to clone the rat by applying a proprietary technology platform derived from nuclear transfer. The adult male and female cloned rats are fertile and exhibit no abnormalities. The data published in Science unambiguously demonstrates that the animals are clones and that the technology can be used to generate targeted mutations in the rat.

"The success we have announced today represents an important result for the pharmaceutical industry, as bio-pharmaceutical companies can now look forward to significant productivity increases in their R&D process by using genetically modified rats. This would save tremendous resources in the industry by optimizing management of the drug development pipeline. We are currently working on building an international consortium that would bring together pharmaceutical companies and biotech companies with the objective of developing knock-out and knock-in rat models targeting therapeutic areas of high unmet medical need, such as hypertension, obesity, diabetes and neurological disorders," said Alexandre Fraichard, CEO of genOway.

"The importance of this scientific breakthrough should not be underestimated given that numerous research teams have been working on rat cloning for years without significant results. The combination of genOway's expertise and INRA's capabilities has been very successful in controlling activation of rat oocytes, the critical parameter in rat reproduction. As a result, we have now mastered the only technology that can achieve targeted genetic modifications in rat. This paves the way for the development of sophisticated genetically-modified rat models which will be of immense benefit to scientists around the world," said Dr Jean-Paul Renard, Head of Reproduction and Developmental Biology, INRA.

genOway is now focusing on the generation of cloned animals with specific genetic modifications (knock-out and knock-in rats), that are designed to provide improved models for highly relevant therapeutic areas such as cardiovascular (hypertension, atherosclerosis…) and neuroscience (Alzheimer's disease, aging…). In parallel, the company is also working on new and better models for toxicology and pharmacology studies, two key steps in the drug development process.

Rats are physiologically closer to humans than most other species used in research, and are reliable models for the study of many human diseases, as well as for the discovery of new druggable targets and therapeutic molecules. "The vast amount of physiological data collected on the rat over decades makes it a research model of key importance for many human pathologies," says Prof. M.Lazdunski, a pharmacologist and Director of Molecular and Cellular Pharmacology Institute (CNRS, France).

The international scientific community eagerly awaits genetically modified rat models (see NIH report: http://www.nih.gov/science/models/rat/genomics). With genetically modified rat models, it is possible to combine the power of genetics with the power of rat physiology, but until now, genetic manipulation in the rat has been hampered by the lack of suitable technologies.

Fundraising consolidates the company's leading european position in the development of tailor-made transgenic mice and rats

genOway (Lyon, France), the leading European provider in transgenesis, announced today the completion of a € 3.3 million fundraising. The money raised will support the growth of the company, thus consolidating its European leading position in the development of tailor-made transgenic mice and rats. In the last four years, genOway has expanded its business to 13 European countries and is now working with major leading pharmaceutical companies such as GlaxoSmithKline, Merck Sharp & Dohme, Novartis, Pfizer, sanofi-synthelabo and Servier, as well as renowned non-profit academic research institutes including King's College of London, Ludwig Institute for Cancer Research and Pasteur Institute.

This new fundraising, led by Dassault Developpement, reaffirms the confidence of all the existing investors - Qualis SCA, Rhone Alpes Creation and Dassault Developpement - have in genOway's scientific solutions for pharmaceutical companies and research institutes.

The successful fundraising will enable the company to launch its new product portfolio in Europe (for example, the release of the 'Safe Transgenesis^TM, technology will guarantee the development of an unlimited number of transgenic animals with predefined characteristics). The competitive advantages of these new products are to reduce the risk and development time of transgenesis projects, which are crucial for the industry. The remaining funds will also foster a research programme to develop cloned rats and humanised models.

"Over the last two decades scientists have been burdened with exhausting procedures and unpredictable results of genetically modified animal models. With our three "safe and fast" technologies that will be launched this year, scientists will no longer have to face such obstacles," said Alexandre Fraichard, CEO of genOway.

"We trust in genOway because they are focused on meeting an existing need in the marketplace and because they are the only solid company to offer this expert service," explained Jean-Claude Lévêque from Dassault Developpement.

In addition, genOway has invested in newly renovated facilities in order to support the company's expected rapid growth. The new 1,200 square metre facility houses state-of-the-art laboratories and research equipment and have been fully financed by genOway in 2002 thus providing a debt-free infrastructure with no further spending. The local authority of "Grand Lyon" strongly supported the refurbishment of this facility.

The laboratories are staffed by scientific experts with years of experience in molecular biology and cellular biology and the company intends to expand its workforce further over the next few months. The facility has been functionally designed for ISO 9002 certification.

genOway S.A. (Lyon, France), the leading European provider in transgenesis and CopyRat Pty Ltd (Melbourne, Australia) a private biotechnology company, have joined forces to develop Knock-out - Knock-in rat models through a research partnership. Rat models are of prime importance for the development of new drugs in several large market therapeutic areas such as diabetes, cardiovascular diseases and neurological disorders.

Currently, the use of rat models has been limited by the lack of technologies to enable genetic modification of this animal. To overcome this limitation, genOway has achieved a leading position in the rat nuclear transfer field. One of the major achievements of genOway in this field was the generation of the first cloned rat embryo (introduced at the Cold Spring Harbor Meeting, NY, December 2001). The work was undertaken in collaboration with a team under Dr Jean-Paul Renard at the Institut National de la Recherche Agronomique in Paris.
CopyRat has developed a leading position in rat embryology and a technology platform that allows the isolation, culture and genetic modification of rat stem cells.

genOway will use CopyRat's stem cells in its proprietary cloning procedures. Combining both technologies will enable the companies to bypass the major technological barriers to develop rat models. A number of preliminary results have highlighted the potential of this partnership. The research is expected to take less than 12 months to complete. Financial terms have not been disclosed.

"Combining both technologies paves the way for the generation of targeted genetic manipulations in rats. Such rat models will strongly increase the efficiency of bio-pharmaceutical R&D by providing disease models of higher predictability. CopyRat and genOway are in an optimal position to provide the industry with these models," said Dr Fraichard, CEO of genOway.

"The development of this technology is essential to enable the development of rat models of human diseases such as diabetes, cardiovascular disease, respiratory disease and neurological disorders. Once developed, the rat models will play a critical role in the future development of improved treatments for these diseases," said CopyRat Chief Operating Officer, Dr Daniels.

CopyRat Pty Ltd is a spin out of Monash University, one of the world's leading research institutions in embryology and stem cell research. CopyRat has exclusive rights to intellectual property and know-how developed by Monash University in the areas of rat embryology and rat stem cells. CopyRat was established in August 2000 with the aim of becoming a world leading supplier of rat and cellular models of human disease to the biomedical industry and the academic community for medical research. CopyRat has developed a leading technology platform in rat embryology and the isolation and culture of rat stem cells, which will be critical to the development of the rat-cloning technology. Through its subsidiary company, IngenKO, CopyRat currently provides mouse gene manipulation services to clients in Australia, USA, Europe, Japan and Asia.

SERVIER and genOway S.A. have signed a multi target deal in the field of gene validation. This non-exclusive agreement covers the in vivo validation of gene targets identified at “Institut de Recherches Servier”. genOway will apply its proprietary target validation technologies to provide Servier with predictable results on the quality of its targets. Increasing the predictability (being closer to human physiology) is the key strength of genOway’s technologies. This one-year deal is renewable annually. Financial conditions and therapeutic areas of interest were not disclosed.

“This agreement reinforces genOway’s leading position in the field of in vivo and in vitro target validation and is further confirmation that genOway’s expertise and original intellectual property makes it a potential partner for most research based pharmaceutical and biotechnology companies” said Alexandre Fraichard, CEO of genOway.

“The collaboration with Servier is a strong signal from a key industry player that genOway’s technological platform provides pharmaceutical companies with rapid and relevant gene validation. In 2002, genOway’s partners will benefit from proprietary homologous recombination technology Rapid Knock Out. Using our Rapid KO technology, target validation programs will be shortened by several months ” continued Dr. Fraichard.

Servier is the largest privately-owned pharmaceutical company in France. It is the third ranked French drug group worldwide and the fourteenth in the European ranking of drug companies. Servier is a privately-owned company, established in 1954 by its founder and current Chairman, Jacques Servier, M.D. The Servier Group has 70 subsidiaries and markets it products in 140 countries with its main therapeutic products used to treat diabetes, cardiovascular disease, neuropsychiatric disorders, cancer, and bone and joint diseases. Servier has obtained regulatory approval for over30 medicines originating from their own research over the past 30 years."

French functional genomics company gen0way S.A. has announced the first successful post implantation inn vivo development of a cloned rat embryo. The company has conducted a joint nuclear transfer program to produce genetically manipulated rats, primarily focusing on Knock-out ( KO ) and Knock-in ( Kin ) rats.

Dr Alexandre FRAICHARD, Chief Executive Officer of genOway, said, « Up to now, no-one has succeeded in obtaining in vivo embryonic development using a cloned rat embryo. We achieved the key milestone of a living cloned rat embryo with no apparent abnormalities. The embryo was found in post mortem examination of the foster mother after 12.5 (twelve point five) days of development. In the past years, all previous attempts to clone rats have failed because the donor nucleus and the recipient ovocyte have not been “in phase”. These results demonstrate that we have in developing a technology that allows reprogramming of the rat nucleus.”

“Much work still remains to be done. But these results pave the way for further KO and Kin rats over the next two years. gen0way will be launching an ambitious program with our pharmaceutical an biotech partners to provide researchers with rat models which are of higher predictability and relevance for the treatment of a number of human diseases. Numerous therapeutic areas such as Cardiology and Neuroscience will benefit from this important innovation. Furthermore, the rat is the most commonly used model in toxicology studies. In the two coming years, we believe the development of a bank of several hundred KO rats is a realistic target” added Dr Fraichard.

This major breakthrough was announced by Dr Fraichard at the Physiological Genomics & Rat Models conference in Cold Spring Harbor Laboratory, New York, held from 6 to 9th December 2001. The work was undertaken in collaboration with a team under Dr Jean-Paul Renard at the Institut National de la Recherche Agronomique in Paris. The team has made a significant contribution to the nuclear transfer field over the past years in several species including cow, rabbit and mouse.

Cellectis S.A. AND genOway S.A. sign a licensing agreement for the use of homologous recombination

Cellectis SA, an offshoot of the Pasteur Institute specialised in genome engineering, has signed a non-exclusive world licensing agreement with genOway S.A., a biotechnology company specialised in the stem cell field.

The licence concerns Patent EP419621 covering the use of homologous recombination. The scope of this licence covers the development of animal and cell models.

Homologous recombination is the technological basis of all the methods of rational genome engineering, the speciality of Cellectis S.A. The process is underpinned by natural cell mechanisms of DNA maintenance and repair, present in all living organisms. The licensed technology permits the deletion or replacement of genes in living organisms. This already well-tried technology has shown its power for more than a decade and today forms a key technology for the study of genes.

Stem cells are the area in which genOway SA excels. Building on its background as supplier of technology to the biopharmaceutical industry, genOway SA develops a range of proprietary products for immunology, toxicology and neuroscience. In the area of gene validation and the screening of chemical entities, these products lend partner companies the means to increase the throughput of their research programmes and allow an essential quantum leap in the predictability of their drug development programmes. This licence totally frees genOway's partners and customers from all royalty claims.

SYN•X Pharma Inc. (CDNX: "SYY") and genOway S.A. have formed a collaboration to combine their respective expertise in proteomics and genomics. SYN•X, based in Toronto, Canada, has developed a proprietary Proteomics Discovery PlatformTM; genOway, based in Lyon, France, has a proprietary genomics platform to develop transgenic animals for identification of drug candidates. Together the companies will develop cardiovascular and central nervous system (CNS) drug targets using SYN•X’s extensive library of novel, proprietary proteins, which genOway will test for therapeutic value using its transgenic rodents created for cardiovascular and CNS research. The agreement will greatly accelerate the elucidation of proteins as potential therapeutics, thereby shortening the discovery and development process by several years.

This combined expertise in the drug discovery and development process will provide a more comprehensive research and development platform, new proprietary disease models and a broader range of discovery services than either could offer alone. Under the terms of the agreement SYN•X and genOway will share revenues from products, services and intellectual property developed jointly.

SYN•X Pharma has developed an extensive library of novel, proprietary proteins for CNS, cardiovascular diseases and stroke. Using information from the novel proteins discovered by SYN•X’s Proteomics Discovery PlatformTM for a particular disease, genOway will create a transgenic animal model or a "Knock-Out/Knock-In" animal model for pre-clinical research of drug targets. Both types of animal models will allow the testing of SYN•X’s proteins for therapeutic activity against disease. Together both companies will generate a pre-clinical model that mimics the disease in man and is capable of testing new therapeutic intervention.

"This collaboration is a marriage of strengths," said Sean McNicholas, President and Chief Executive Officer of SYN•X Pharma. "SYN•X and genOway exercise leadership in proteomics and genomics to quickly develop drug targets. We both bring expertise that is extremely valuable by itself, but together we can quickly and efficiently take our knowledge and meet the needs of pharmaceutical clients looking for specific therapeutics. Together we provide a totally integrated approach and bring pharmaceutical companies a drug that has been pre-clinically proven and is ready for human trials."

"Science is complex and the need for real expertise is growing rapidly," said Alexandre Fraichard, President and CEO of genOway S.A. "The alliance of two experts such as SYN X Pharma and genOway reflects a serious trend in the market to better match customer needs. Furthermore, the fact that this collaboration is done between a North American and a European entity reflects an important bridgehead for both companies."