Pharma, R&D

Boehringer organizes a coalition in search of first CF gene therapy, working with research, manufacturing partners

Joining hands with several academic and industry partners from the UK, Boehringer Ingelheim is launching a quest to develop a gene therapy for cystic fibrosis — one that, if successful, would shake up a field plagued by some recent failures and dominated by Vertex.

The backbone of the collaboration is 17 years worth of work done by the UK Cystic Fibrosis Gene Therapy Consortium, pulling together researchers from Imperial College London and the Universities of Oxford and Edinburgh. With help from Boehringer, the consortium will lead the development and completion of preclinical studies on a gene therapy that it’s identified, to a point where the product will be ready for human studies.

To facilitate the tech transfer and manufacturing processes, Boehringer has also tapped Imperial Innovations and Oxford BioMedica for a three-way partnership, such that the German drugmaker will have an option to license their viral vector and production technology.

The big idea here is to develop a treatment that can address all the 2,000 known mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR). Whereas existing therapies can only slow the progress of the disease and tend to have varying effects depending on a patient’s mutation status, Boehringer says their novel approach — introducing a healthy copy of the CFTR gene into lung cells using a lentiviral vector via an inhaled formulation — has the potential to be a universal option.

Financial terms of the deal were not disclosed and no timelines were provided.

There’s been considerable attention on cystic fibrosis as Vertex continues to dominate the space and move forward with promising combo studies. A host of rivals have appeared, including Galapagos, but no one has provided any recent data to support one of these other contenders.

In the past weeks, we’ve seen Proteostasis plunge on failed Phase IIb trials, with Celtaxsys joining the club last week clinching shaky data they claimed to be “clinically meaningful.”

Vertex has scored big in the US with its new drugs, but the biotech has been building up some tension with single-payer groups in Europe — which happens to be Boehringer’s home turf.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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