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9 August 2018Americas

Boehringer signs deal to develop cystic fibrosis gene therapy

Boehringer Ingelheim has teamed up with academic institutions and research companies in the UK to develop a long-term gene therapy for cystic fibrosis, according to a statement released on Monday, August 6.

The global collaboration is between German pharmaceutical company Boehringer, technology company Imperial Innovations, biopharmaceutical company Oxford BioMedica, and the UK Cystic Fibrosis Gene Therapy Consortium (GTC).

The GTC is the world’s largest gene therapy group focussed on a single disease. It includes the University of Oxford, the University of Edinburgh, and Imperial College London.

Cystic fibrosis is a genetic disease that causes persistent lung infections and limits the ability to breathe. Existing treatments are only able to slow the progression of cystic fibrosis and they also have varying effects, depending on a patent’s mutation status.

There are more than 2,000 known mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR), many of which result in a person developing CF.

Boehringer said that “there is a critical unmet need for therapies that address all CFTR mutations,” but “gene therapy has the potential to provide such a solution”.

The partnership brings together academic expertise in developing gene therapy for cystic fibrosis, with industry expertise in drug discovery and manufacturing vector-based therapies.

The institutions will work towards a novel approach which uses “a replication-deficient lentiviral vector in an inhaled formulation to introduce a healthy copy of the CFTR gene into the cells of the lung”.

So far, this method has demonstrated high gene transfer efficiency, according to Boehringer. It also offers the possibility of repeated administration, to maintain the therapeutic effect.

Under the terms of the agreement, Boehringer will fund the development of the novel gene therapy. It will then have the option to license the exclusive global rights to develop, manufacture, register, and commercialise it.

Eric Alton, coordinator of the GTC, said: “The GTC believes that this partnership provides cystic fibrosis patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems.”

Financial terms of the deal have not been disclosed.

Boehringer will be represented by Atabak Royaee, Associate Director & Senior Counsel, at the Life Sciences Patent Network North America Fall conference on October 16 in San Francisco. Royaee will be speaking on a panel called “How do we Ensure Effective Protection for Antibodies?”. To find out more, click  here.


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