Boehringer, Oxford BioMedica join on CF gene therapy project
- Gene and cell therapy group Oxford BioMedica has signed up to a collaboration with the U.K. Cystic Fibrosis Gene Therapy Consortium (GTC) and Boehringer Ingelheim to develop an inhaled gene therapy for cystic fibrosis using a lentiviral vector.
- Per the process development deal with GTC and its partner Imperial Innovations, Oxford BioMedica will be responsible for process and analytical development, scale-up of manufacture and generation of material for toxicology studies. The partners may also establish a clinical supply agreement in the future.
- Oxford BioMedica has given Boehringer an exclusive option to global rights for the manufacture and commercialization of a CF gene therapy based on its lentiviral vector technology. Financial terms were not disclosed.
Since the CTFR gene was identified in 1989, gene therapy for cystic fibrosis has been a sought-after goal for researchers. The hurdles have been perhaps steeper than expected, at least in part because the lung has evolved barriers to protect against bacteria, viruses and other foreign bodies. These include mucus and sputum, which are particularly hostile in CF patients, as well as the cilia lining the lung.
The deals signed on Monday, creating a collaboration between academia, industry and manufacturing, are another effort toward that objective.
Each partner has a specific role. As the big pharma, Boehringer Ingelheim can provide the funding and has an existing respiratory portfolio with a number of drugs on the market and in development. However, this is the company's first step into the cystic fibrosis arena, and its first gene therapy.
The novel viral vector-based product comes from the British GTC, which has years of experience in gene therapy and in cystic fibrosis. The GTC has carried out six non-viral proof-of-concept studies and the first Phase 2b gene therapy trial for CF.
"The UK CF Gene Therapy Consortium has, for the last 17 years, vigorously sought to establish whether gene therapy can become a clinically viable option for patients with CF," said Deborah Gill of the Department of Medicine of Oxford University in a statement. "The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice."
Oxford BioMedica brings its sector-leading lentiviral vector delivery platform, which it has used to develop in vivo and ex vivo gene therapy products both in-house and with partners. The collaboration may also involve development of stable producer cell lines for large scale production of the lentiviral vector.
Oxford BioMedica's involvement at this early stage is notable, indicative of the manufacturing challenges inherent with any gene therapy development.
Securing a ready supply of lentiviral vector production early on could be prudent planning. A report from The New York Times last fall noted that companies developing lentiviral-based gene therapies face long delays due to limited supply of the vectors, which are needed to insert corrected or replacement genes.
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