PCI 7 November 2023, 15:22
Thermofisher: Thu 29 February 2024, 11:15
BMG Labtech: Wed 18 September 2024, 11:55
Owen Mumford 12 January 2022, 16:46

Current Edition

Cell and Gene Therapy

Upcoming Events

Biotechnology Show 2025: 20th January 2025
Anglonordic: 16th January 2025
AI in Drug Discovery – SAE media – January 14th 2025

Advertisement

Fujifilm rectangle: Fri 22 November 2024, 14:23
Roald Dahl Charity: Fri 15 November 2024, 12:57
A&M STABTEST: Fri 21 June 2024, 11:43
CDD Vault: Wed 17 July 2024, 11:46
HTI Automation: Tue 12 November 2024, 14:28
Discovery Park: Tue 17 September 2024, 10:01

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

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.”

Newcells 3 June 2024, 15:12
Novonordisk: Wed 17 July 2024, 11:22
FujiFilm 30 October 2023, 16:23
Eclateral 30 October 2023, 15:34
Autoscribe Mon 26 June 2023, 15:15
Aldevron: 16th January 2025
Richter: Wed 23 October 2024, 09:03
GenXPro: Mon 16 September 2024, 10:40
Biosynth: Tue 1 October 2024, 13:25