A single viral gene therapy platform, originally developed to treat cystic fibrosis, is now being tested against five devastating lung and blood diseases. This matters because each of these conditions currently has limited or no effective treatment. Influenza A kills over 500,000 people annually. Primary Ciliary Dyskinesia affects roughly 1 in 10,000 newborns. Alpha-1 Antitrypsin Deficiency afflicts an estimated 200,000 people worldwide. Haemophilia affects around 400,000 individuals, 85% with Factor VIII deficiency and 15% with Factor IX deficiency. Surfactant Protein B deficiency is so severe that most affected newborns die within months, as lung transplantation is rarely attempted. If the platform succeeds, a single delivery system—the rSIV.F/HN lentiviral vector—could be adapted to produce therapeutic proteins directly in the lungs or bloodstream for multiple diseases. This would replace the need to develop separate gene therapies from scratch for each condition. The approach has already shown it can improve lung function in cystic fibrosis patients, the first demonstration of its kind. Success would transform treatment options for rare genetic lung diseases, reduce global flu mortality, and offer a durable therapy for haemophilia—all from one technological platform.
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Gene therapy aims to treat diseases by delivering genes that produce therapeutic proteins. Over two decades the UK CF Gene Therapy Consortium (GTC) has pooled the resources of three major groups in the UK (Oxford and Edinburgh Universities and Imperial College London), progressing from laboratory studies to the first demonstration that gene therapy can produce improvements in the lungs of CF patients. The key steps forward have been a) to identify methods by which genes can be delivered into the lungs and b) the understanding that the new genes can secrete proteins both into the lungs and blood stream. The GTC's underpinning platform technology for the Portfolio is a viral gene therapy platform based on the rSIV.F/HN lentiviral vector which will be applied to five projects: Influenza A- a major worldwide health risk causing >500,000 deaths each year. Primary Ciliary Dyskinesia (PCD) - a life-threatening recessive condition, it has a worldwide incidence of -1/10000. Alpha 1 Anti Trypsin Deficiency (AATD)- rare genetic disease which is present in almost all ethnic groups. Worldwide there are estimated to be -200,000 patients with AATD. Haemophilia - affects around 400,000 individuals worldwide, of whom 85% have Haemophilia A (Factor VIII deficiency) and 15% Haemophilia B (Factor IX deficiency). Surfactant Protein B (SP-B) deficiency- one of several rare interstitial lung diseases that cause alveolar collapse and respiratory distress syndrome (RDS) in newborn babies. Due to the lack of donor organs, and the unstable state of the disease, lung transplantation is rarely attempted and the majority of individuals affected by SP-B deficiency succumb to the disease within the first few months of life.
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