Boehringer Ingelheim, the UK Respiratory Gene Therapy Consortium (GTC), IP Group and Oxford BioMedica (OXB) have commenced the Phase I/II LENTICLAIR 1 trial of inhaled lentiviral vector-based gene therapy BI 3720931 targeting cystic fibrosis (CF).
CF subjects who are ineligible for current cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatments are mainly targeted in the trial.
Go deeper with GlobalData
This first-in-human trial will assess the gene therapy’s tolerability, efficacy and safety in this particular patient group.
It is structured in two parts, with the Phase I segment focusing on determining the safety and tolerability of different doses to select the optimal ones for the second phase.
The subsequent double-blind, placebo-controlled Phase II trial will evaluate the safety and clinical efficacy of two chosen doses or a placebo.
Participants will engage in a 24-week trial period, after which they will enter a long-term follow-up trial called LENTICLAIR-ON. The anticipated completion time for the trial is early 2027.
OXB’s lentiviral vector manufacturing technology will be leveraged in the trial, which is the culmination of a collaboration that began in August 2018 involving Boehringer Ingelheim and partners.
Boehringer Ingelheim board of managing directors member Dr Paola Casarosa said: “We are very excited about the start of the LENTICLAIR 1 first-in-human trial and how BI 3720931 could potentially improve the lives of people living with CF, and who are unable to benefit from current CFTR modulators.
“The partnership with the GTC and OXB provides an excellent example of the progress that can be made when diverse organisations and people come together with a long-term, shared goal to create a new paradigm of care.”
BI 3720931 is designed to insert the CFTR gene’s functional copy directly into the airway epithelial cells’ DNA.
Thereby, it claims to enhance the functioning of the lungs and decrease exacerbations among CF patients.
CF is a genetic disorder characterised by mutations in the CFTR gene, leading to the production of thick mucus that obstructs the airways and causes persistent lung infections, progressively impairing respiratory function.
Before this development, Boehringer achieved success with its lung disease drug, nerandomilast, in a Phase III trial, setting the stage for the company to seek approval for the drug in an additional therapeutic indication.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
