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Pharma BI joins forces against cystic fibrosis

August 7, 2018

Boehringer Ingelheim is upping its game in the fight against cystic fibrosis and jumping into the gene therapy realm.

The pharmaceutical company with U.S. headquarters in Ridgefield announced this week a partnership with the UK Cystic Fibrosis Gene Consortium, Imperial Innovations and Oxford BioMedica. The purpose of the collaboration is to develop first-in-class gene therapy for patients with cystic fibrosis, a progressive, genetic disease that limits a person’s ability to breathe over time.

The Cystic Fibrosis Foundation estimates that 30,000 in the U.S. and 70,000 worldwide have the disease. About 1,000 new cases are diagnosed each year. According to the Cystic Fibrosis Foundation, people with the disease can have persistent coughing, frequent lung infections, shortness of breath and more.

The new collaboration combines BI’s skills in drug discovery and clinical development with the partners’ academic and manufacturing capabilities in gene therapy.

“Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward,” Clive R. Wood, senior corporate vice president of discovery research at BI, said in a release. “Bringing together our existing expertise as a leader for nearly a century in the discovery and development of therapies that have advanced patient care in respiratory diseases with the gene therapy knowledge of our partners, we aim to unlock unprecedented opportunities for patients with this devastating disease, who are desperately waiting for better treatment options.”

Existing treatments only slow the progression of the disease and have varying effects depending on a patient’s mutation status, which is determined by the gene variation that causes their disease. BI officials said there is an unmet need for therapies that address all mutations, and gene therapy has the potential to provide a solution.

BI has received an option to license the exclusive global rights to develop, manufacture, register and commercialize the gene therapy for the treatment of cystic fibrosis. Financial terms for this option were not disclosed.

BI will finance the development as the partners work together during the option period. The collaboration is an initiative of BI’s Research Beyond Borders, or RBB, and Respiratory Therapeutic Area. RBB is one of the pillars of BI’s research and development strategy and explores emerging science, disease areas and technology.

Eric Alton, coordinator of the UK Cystic Fibrosis Gene Therapy Consortium, said his organization has sought for the last 17 years to find out if gene therapy can be a viable option for cystic fibrosis patients.

“Boehringer Ingelheim will provide its multinational industry expertise, including a rich heritage in the respiratory field, to drive the product towards the clinic, (while) Oxford BioMedica is the acknowledged leader in the field of lentiviral vector manufacturing,” Alton said in a release. “We believe that this partnership provides (cystic fibrosis) patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease, as well as treat established problems.”

BI has formed several partnerships over the last few years to research and develop drugs to prevent and treat diseases. Earlier this year, the company committed to spend more than $24 billion by 2025 to develop human pharmaceuticals. The commitment has the potential to deliver 15 new medicines for approval by 2025, company officials said.

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