- Boehringer Ingelheim will license rights to research around an experimental gene therapy for cystic fibrosis, announcing Tuesday it had exercised an option it secured three years ago from a consortium of universities in the U.K.
- Under the terms of the agreement, Boehringer will pay a small fee to IP Group, a British intellectual property group acting on behalf of the UK Cystic Fibrosis Gene Therapy Consortium, which consists of researchers from three universities. The company will also pay a fee to manufacturer Oxford Biomedica, with additional payments should development, regulatory and sales milestones be met.
- Licensing of the gene therapy marks a step forward in the partners' efforts to develop a cystic fibrosis gene therapy, particularly for those patients who don't benefit from current therapy. The inherited disorder, which damages the functions of the lungs and digestive system, has long been viewed as a candidate for gene-correcting treatments, though progress on that front has been slow.
Boston biotech Vertex is the leading developer of cystic fibrosis medicines. Its four approved drugs can treat most patients. Trikafta, the one most recently approved in the U.S., raised the percentage of people with cystic fibrosis who are eligible for a Vertex product from 50% to 90%, a major achievement.
But the remaining 10%, whose disease is caused by so-called nonsense mutations, don't benefit as much and are left without effective options.
Boehringer's acquisition of the lentiviral gene therapy is aimed at that group, which the company describes as the "immediate target" of its development plans.
The therapy, which is still in preclinical testing, is envisioned as an inhalable treatment, rather than one injected intravenously. Known as BI 3720931, it is designed to selectively introduce a healthy CFTR gene into the relevant cells.
The partnership between Boehringer and its partners in the U.K. was first established in 2018. Tuesday's announcement gives the Germany company exclusive global rights to develop, manufacture, register, and commercialize the gene therapy.
According to a Boehringer executive, the therapy has demonstrated "high gene transfer efficiency in pre-clinical models."