Boehringer Acquires Rights to Potential CF Inhalation Gene Therapy

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by Vanda Pinto, PhD |

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Boehringer Ingelheim has taken legal steps giving it the lead role in further development of a long-lasting, inhalation gene therapy for cystic fibrosis (CF), called BI 3720931, that stems from a public-private research effort begun in 2018.

The 2018 agreement brought together Boehringer, the U.K. Cystic Fibrosis Gene Therapy Consortium (GTC; composed of university scientists), and Oxford Biomedica in work to develop and conduct early tests on a potential first-in-class gene therapy for all with CF.

Now, Boehringer is exercising license options with these partners to acquire exclusive global rights to manufacture, register, and potentially market the therapy. Also involved is the IP Group, acting on behalf of the three U.K. universities in the GTC, Boehringer announced in a press release.

GTC will continue to contribute to research and testing, and Oxford Biomedica to the refinement and manufacturing of lentiviral vectors stemming from its lentiviral vector delivery platform.

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“Since 2018, Boehringer Ingelheim has sponsored research and development activities with the GTC and [Oxford Biomedica]. The shared success achieved with our partners in this potentially revolutionary project makes us confident that we can now further accelerate this highly innovative therapeutic approach,” Clive R. Wood, PhD, corporate senior vice president and global head of discovery research at Boehringer Ingelheim.

“With our leadership in the discovery and development of therapies in respiratory diseases combined with the gene therapy and manufacturing knowledge of our partners, we aim to bring the next breakthrough to patients suffering from CF, who are desperately waiting for better options,” Wood added.

Delivered through a modified lentiviral vector (a virus that cannot replicate), BI 3720931 aims to carry a healthy copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is mutated in CF patients, to target cells. This could allow for greater production of the CFTR protein that is deficient in these people.

The therapy is being developed as an inhaled formulation that could make repeated administration possible. It also is designed  to treat all CF patients, regardless of their specific CFTR mutations.

“The novel lung-targeting technology we have developed has demonstrated high gene transfer efficiency in pre-clinical models and offers the possibility of repeated administration to maintain a therapeutic effect, a benefit that other viral-based gene therapies may not be able to provide,” said Eric Alton, MD, coordinator of the GTC.

“Our novel therapy has the potential to improve CFTR function and modify disease in all CF patients, independent of the more than 2,000 different known gene mutations,” Alton added. “The immediate target is those patients who are not eligible for CFTR modulators. … The GTC is very excited to have reached this milestone after 21 years of focused effort.”

Under the terms of the agreement, Boehringer will pay IP Group — the intellectual property company working on behalf of the researchers with Imperial College London and the universities of Oxford and Edinburgh — an undisclosed option exercise fee, plus future milestone payments and royalties based on potential sales. This option includes giving Boehringer rights to the lentiviral vector developed by GTC, the company reported in a related release.

Oxford Biomedica will receive an option exercise fee of £3.5 million (about $5 million), with additional payments of up to £27.5 million if various development, regulatory, and sales milestones are met.

“We have enjoyed working with Boehringer Ingelheim, IP Group, and the GTC since 2018,” said John Dawson, CEO of Oxford Biomedica. “Building on the great progress made to date, we are delighted that Boehringer Ingelheim, one of the world’s leading respiratory medicine organizations, has chosen to exercise the option to license [Oxford Biomedica’s] lentiviral vector manufacturing technology for this highly innovative inhaled cystic fibrosis gene therapy formulation developed by the GTC.”

The partnership’s past and continuing work, he added, “has the potential to provide a new therapeutic option for many cystic fibrosis patients globally.”