Pipeline Chart

Transgene is focused on discovering and developing a broad pipeline of innovative immunotherapies in the areas of cancer and infectious diseases. This pipeline includes both products deriving from Transgene’s internal research, and particularly from its core viral vector technology, and from in-licensing activities.

Pipeline

Collaborations

Innovation through collaboration is a key driver at Transgene, and the Company has leveraged its R&D capabilities and its portfolio of products and processes through collaborative programs with a variety of external organizations. Transgene has a strong track record of fruitful interactions with academic and private partners, having been involved in more than a dozen grant-supported collaborative programs during the past three years. Some representative examples are highlighted below, and Transgene’s Open Innovation team is always looking for new collaborative opportunities, both at the local and international levels, to enhance and support the Company’s pipeline.

National collaborative projects

ADNA (Advanced Diagnostics for New Therapeutic Approaches) (ongoing). The ADNA program is coordinated by Institut Mérieux and supported by BPIfrance, the French public investment bank. The program brings together four companies specialized in diagnostics (bioMérieux and GenoSafe) and therapeutics (Transgene and Genethon) with the global aim of developing personalized medicines for cancer, infectious diseases and genetic diseases. The program is focused on pharmacogenomic approaches and the development of biomarkers to achieve early diagnosis and improve understanding of how diseases evolve and how individual patients respond to treatment. Transgene’s specific contribution is to develop immunological biomarkers to enable new therapeutic vaccines to be prescribed to patients who are most likely to benefit from them. More information on the ADNA program can be found at www.institut-merieux.com. The ADNA program has been extremely valuable to Transgene in contributing to the identification of potential predictive biomarkers for some of its development programs, including TG4010, which is in late-stage clinical development for the treatment of non-small cell lung cancer.

ONCOVACCINE (ongoing). Funded by ANR, French National Research Agency, the goal of this program is to develop a new generation of oncolytic vaccinia viruses with enhanced therapeutic indices. The rationale is to identify specific intracellular factors that limit the replication in tumor cells and that could be specifically targeted by a dedicated arming of the virus. Project partners are two renowned academic laboratories, IGBMC (UMR7104 CNRS/UdS Inserm U964) and IREBS (UMR 7242).

• Transgene is an active member of two French regional competitive clusters that promote industry collaboration, academic excellence in basic science and university advanced training. Alsace BioValley (http://www.alsace-biovalley.com) is dedicated to therapeutic innovations, and Lyonbiopôle (www.lyonbiopole.org) is focused on infectious diseases, cancer, nutrition, metabolic diseases and neurosciences. Two collaborative research projects involving Transgene’s team have been selected and are being funded by BPIfrance:

o NATHEB (completed). The goal of this project was to support the development of new therapeutic approaches to treat chronic hepatitis B infection, including Transgene’s TG1050. Partners on the project were Edelris, Poxel and Inserm Unit 851.

o CELLPAT (ongoing). This project aims at improving the management and control of biomanufacturing processes in the areas of cell culture and molecule and viral particle production in accordance with Process Analytical Technology and Quality by Design initiatives promoted by French health authorities. Partners on the project are Sanofi Pasteur, Merial, The CoSMo Company and LASIR UMR 8516.

NAT B (to be launched in 2015). Transgene, as part of Institut Mérieux, is involved in BIOASTER, the only health-related technology research institute (IRT) in France (www.bioaster.org). This project is focused on the development of biomarkers for therapeutic vaccines to treat chronic hepatitis B infections and involves Transgene, Inserm and HCL (Hospices Civils de Lyon).

International partnerships

IMMUNOCAN (ongoing). Transgene coordinates the IMMUNOCAN FP7-sponsored European program that aims at increasing cooperation in translational research between European countries and China. The program currently focuses on the identification of immunological characteristics as potential prognostic factors in Chinese cancer patients. The Fudan University Shanghai Cancer Centre (FDUSCC)(China), the University of Copenhagen (Denmark), the IRCCS (Instituto Nazionale dei Tumori) Foundation (Italy) and the Medizinische Hochschule of Hannover (Germany) are also part of this program. IMMUNOCAN activities are conducted in the laboratories of the joint institute for cancer research of the FDUSCC and Institut Mérieux (FDUSCC-IM) in Shanghai, where Transgene locally acts as the operational partner. The goal of the program is to build a Euro-Asian reference center for medical and scientific cancer research.

HELAZYME (ongoing). Transgene and New England Biolabs (NEB) have been awarded through a joint innovation program co-organized by Alsace BioValley and the Massachusetts Life Sciences Center (MLSC). This “International Collaborative Industry Program” is supported by grants from MLSC and BPIfrance. This collaboration between Transgene and NEB focuses on the discovery of an innovative approach to improve digestion of DNA impurities in the manufacturing of viral vectors. Transgene is developing a new high-yield production process for viral vectors and NEB brings to the project its know-how in epigenetics and enzymology.

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