BI 3720931 / Oxford Biomedica, Boehringer Ingelheim - LARVOL DELTA

A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON) (clinicaltrials.gov) - P1/2 | N=285 | Recruiting | Sponsor: Boehringer Ingelheim | Not yet recruiting ➔ Recruiting

Enrollment open • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON) (clinicaltrials.gov) - P1/2 | N=285 | Not yet recruiting | Sponsor: Boehringer Ingelheim

New P1/2 trial • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Boehringer Ingelheim and partners start clinical development of a first-in-class, inhaled gene therapy for people with cystic fibrosis (GlobeNewswire) - "Boehringer Ingelheim...today announce the start of LENTICLAIR 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, gene therapy aiming to improve disease outcomes in people with cystic fibrosis (CF), regardless of gene mutations that are causing the disease. The trial specifically focuses on adults with CF who genetically cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators...The trial is expected to be completed in early 2027."

Trial completion date • Trial status • Cystic Fibrosis

A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1) (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Boehringer Ingelheim | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Viral vector • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-In-Human Trial. (PubMed, Am J Respir Crit Care Med) - "A third-generation lentiviral vector pseudotyped with Sendai virus F and HN envelope proteins (rSIV.F/HN) has been developed for the treatment of cystic fibrosis. Promising preclinical results support the progression of this vector carrying a full-length CFTR transgene (BI 3720931) into a first-in-human clinical trial expected to begin in 2024."

Gene therapy • Journal • P1 data • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

Comprehensive methods to assess pharmacokinetics and monitor for viral shedding, replication competence, and immunogenicity of BI 3720931 in the first-in-human Lenticlair 1 Phase I/II trial (NACFC 2024) - "Because BI 3720931 is the first-of-its-kind lentiviral gene therapy being investigated in CF, a unique and thorough strategy to monitor its safety and tolerability is required. This panel of novel assays complies with regulatory guidelines outlined for gene therapies and supports the monitoring of safety and tolerability of BI 3720931 in Lenticlair 1 and its extension trial, Lenticlair-ON, in PwCF. CRCs (mean (SD) out of 5)."

P1/2 data • PK/PD data • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Respiratory Diseases • CFTR

A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1) (clinicaltrials.gov) - P1/2 | N=36 | Not yet recruiting | Sponsor: Boehringer Ingelheim | Phase classification: P ➔ P1/2

Gene therapy • Phase classification • Trial completion date • Trial initiation date • Trial primary completion date • Viral vector • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Lenticlair™-ON: An extension trial examining long-term safety and efficacy outcomes associated with an inhaled F/HN-pseudotyped lentiviral vector for CF gene therapy in people with CF (ECFS 2024) - "This trial will investigate long-term safety in pwCF treated with at least one BI 3720931 dose, assess efficacy duration after initial dosing and determine efficacy and safety after re-dosing, as applicable."

Clinical • Gene therapy • Viral vector • Cystic Fibrosis • Gene Therapies • Immunology • Pulmonary Disease • Respiratory Diseases

Lenticlair™ 1: A Phase 1/2 trial evaluating the safety, tolerability and efficacy of an inhaled F/HN-pseudotyped lentiviral vector for CF gene therapy in people with CF ineligible for CFTR modulators (ECFS 2024) - "This trial will evaluate the safety and efficacy of BI 3720931 over 24 weeks in pwCF who are ineligible for CFTRmt. A separate trial will monitor long-term safety for 15 years."

Clinical • Gene therapy • P1/2 data • Viral vector • Cystic Fibrosis • Gene Therapies • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

F/HN pseudotyped lentiviral vector‐mediated transduction of non‐human primates (ESGCT 2023) - "Objectives: We have developed a lentiviral vector pseudotyped with the Sendai virus F and HN envelope proteins (BI 3720931) for cystic fibrosis (CF) gene therapy... This study confirms and extends our previous findings of rSIV.F/HN-based in vivo gene transfer in mice. Using a relatively low vector dose, NHPs demonstrated transduction efficiency in the range of values likely to relate to clinical benefit, without evidence of toxicity. Transduction efficiency and toxicology in animals treated with a higher dose is currently being analysed."

Viral vector • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases

F/HN pseudotyped lentiviral vector-mediated transduction of non-human primates (ECFS 2023) - "This study confirms and extends our previous findings of rSIV.F/HN-based in vivo gene transfer in mice to NHPs. Using a relatively low vector dose, NHPs demonstrated transduction efficiency in the range of values likely to relate to clinical benefit, without evidence of toxicity. Transduction efficiency and toxicology in animals treated with a higher dose is currently being analysed."

Viral vector • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases

Boehringer Ingelheim and Partners to Accelerate Development of First-In-Class Gene Therapy for Patients with Cystic Fibrosis (Businesswire) - "Partners aim to expedite the development of the novel, inhaled cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy BI 3720931 as a long-lasting therapeutic option for patients with cystic fibrosis (CF)....Boehringer Ingelheim...announced today that Boehringer Ingelheim has exercised its options on intellectual property and know-how from the partners to progress and further accelerate the development of a potential, new treatment option for patients with CF. In the partnership, IP Group, acting on behalf of the three GTC host Universities, is granting exclusive global rights to develop, manufacture, register, and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. The GTC is additionally contributing its knowledge in pre-clinical research and clinical gene therapy development."

Licensing / partnership • Cystic Fibrosis

Oxford BioMedica to supply viral vectors to Boehringer Ingelheim (BioProcess International) - "Oxford BioMedica has signed a three-year contract to manufacture and supply viral vectors to support Boehringer Ingelheim’ s cell and gene therapy pipeline...Oxford BioMedica and Boehringer Ingelheim partnered in 2018 to develop a gene therapy treatment for Cystic Fibrosis. The collaboration saw Oxford BioMedica develop and manufacture the candidate alongside material for toxicology studies."

Licensing / partnership • Cystic Fibrosis