New Gene Therapy Trial Aims to Transform Cystic Fibrosis Treatment

Ingelheim, Germany, February 20, 2025 – Boehringer Ingelheim, in collaboration with IP Group, the UK Respiratory Gene Therapy Consortium, and OXB, has initiated LENTICLAIR™ 1, a Phase I/II trial of BI 3720931. This advanced gene therapy is designed to potentially transform the lives of cystic fibrosis (CF) patients, including those currently unable to benefit from CFTR modulators.

Understanding Cystic Fibrosis

Cystic fibrosis is a hereditary, lifelong condition affecting more than 100,000 people worldwide. It is caused by mutations in the CFTR gene, which leads to the production of dysfunctional CFTR proteins. These proteins cause thick, sticky mucus that clogs the airways, leading to frequent lung infections and progressive lung damage.

Over 2,000 mutations of the CFTR gene have been identified, each with different levels of disease severity. While CFTR modulators have brought significant advancements for many patients with specific mutations, these treatments are ineffective for 10-15% of CF patients.

Introducing BI 3720931

BI 3720931 is a first-in-class, inhaled lentiviral vector-based gene therapy. Unlike CFTR modulators, this therapy aims to insert a functional copy of the CFTR gene directly into the airway epithelial cells of CF patients. The objective is to improve lung function, reduce exacerbations, and provide a potential solution for those who cannot benefit from current treatments.

Partnerships Driving Progress

The development of BI 3720931 exemplifies the importance of collaboration in medical research. Since 2018, Boehringer Ingelheim has worked alongside the UK Respiratory Gene Therapy Consortium (GTC) and OXB to bring this groundbreaking therapy to clinical trials.

Dr. Paola Casarosa, Member of the Board of Managing Directors at Boehringer Ingelheim with responsibility for the Innovation Unit, expressed enthusiasm about the trial. “We are excited about the potential of BI 3720931 to improve the lives of people with cystic fibrosis who are not eligible for current treatments,” she stated. “This partnership illustrates the power of diverse organizations coming together with a shared goal.”

UK Respiratory Gene Therapy Consortium’s Role

Professor Eric Alton from Imperial College London, who leads the GTC, echoed the excitement. “The GTC has spent 24 years working towards making gene therapy for cystic fibrosis a clinical reality. We anticipate this novel approach will offer new hope to patients with CF, especially those not suitable for CFTR modulators.”

The GTC has been instrumental in developing the lentiviral vector used in BI 3720931, having conducted multiple proof-of-concept studies and clinical trials.

OXB’s Contribution

OXB, a leading contract development and manufacturing organization specializing in cell and gene therapy, is providing Boehringer Ingelheim with its proprietary lentiviral vector manufacturing technology. Dr. Frank Mathias, CEO of OXB, praised the collaboration. “We are thrilled to be part of this innovative cystic fibrosis gene therapy,” he said.

OXB’s expertise in viral vector technologies, including its fourth-generation Tetravecta™ system, ensures high-quality production for clinical trials.

Future Directions

The LENTICLAIR™ 1 trial is expected to conclude in early 2027. While the efficacy and safety of BI 3720931 have not yet been established, the potential impact on CF patients is substantial. This trial represents a crucial step towards providing a gene therapy solution for all cystic fibrosis patients, regardless of their specific genetic mutation.

For more information about the trial, visit ClinicalTrials.gov.

BI 3720931 is still considered an investigational therapy and has not been approved for use in any country.

About Boehringer Ingelheim

Boehringer Ingelheim is a global biopharmaceutical company dedicated to researching and developing innovative treatments for diseases with unmet medical needs. Founded in 1885, the company is committed to long-term sustainability and ethical practices. With a presence in over 130 markets and more than 53,500 employees, Boehringer Ingelheim remains a leader in human and animal health.

For more details, visit https://www.boehringer-ingelheim.com.

About OXB

OXB, a contract development and manufacturing organization, has specialized in cell and gene therapy since its founding in 1997. With a focus on quality and innovation, OXB collaborates with leading pharmaceutical and biotech firms to develop and produce unique viral vector technologies like the Tetravecta™ system.

For more information, visit www.oxb.com.

About the UK Respiratory Gene Therapy Consortium

The UK Respiratory Gene Therapy Consortium (GTC) is a collaboration of researchers from Imperial College London, the University of Oxford, and the University of Edinburgh. Since 2001, the GTC has conducted numerous studies and clinical trials aimed at making gene therapy for cystic fibrosis a reality.

For updates and further details, visit www.respiratorygenetherapy.org.uk.

Supporting Organizations

The development of gene therapies for cystic fibrosis is made possible by the support of various organizations and funders, including the Cystic Fibrosis Trust, the Health Innovation Challenge Fund, and Wellcome. Contributions from these organizations have facilitated crucial research and clinical testing.

The Cystic Fibrosis Trust supports patients and families affected by CF, funding research and providing essential resources. For more information, visit https://www.cysticfibrosis.org.uk.

About the Cystic Fibrosis Trust

The Cystic Fibrosis Trust is dedicated to improving the lives of everyone affected by cystic fibrosis through research, support, and innovative care. By funding cutting-edge research and providing confidential advice and resources, the Trust aims to accelerate the development of new treatments and improve patient lives.

To learn more or to make a donation, visit https://www.cysticfibrosis.org.uk.

Conclusion

The initiation of the LENTICLAIR™ 1 trial marks a critical step in the ongoing quest to find a cure for cystic fibrosis. By targeting the underlying genetic defect with a novel gene therapy, researchers and pharmaceutical partners aim to provide hope to patients who have not benefited from current treatments. As this groundbreaking study moves forward, it underscores the importance of collaboration and perseverance in advancing medical science.

Share your thoughts on this exciting development in cystic fibrosis treatment or ask questions in the comments below. Join our community to stay informed about the latest advancements in gene therapy and medical research.