The United Kingdom Cystic Fibrosis Gene Therapy Consortium (UKCFGTC) recently published the first findings from Phase IIb multi-dose clinical study on cystic fibrosis. In this study, CF patients were treated with aerosolized DNA plasmid expressing Cystic Fibrosis Transmembrane Conductance Receptor (CFTR), developed at Woodlands, Texas-based VGXI‘s cGMP facility, making this study a pioneer in showing gene therapy can produce significant improvement on CF and lung function.
“One hundred and thirty-six patients aged 12 and above received monthly, inhaled doses of the gene therapy or a placebo for 1 year,” said Professor Deborah Gill, from Oxford University. “The results are encouraging and are expected to lead to a further study to assess higher, more frequent doses.”
VGXI, a leading provider of plasmid DNA manufacturing and development services for DNA vaccine and gene therapy research, is determined to maintain its impressive track record of successful plasmid products manufactured under GMP conditions, and is proud to have been an active team player in the UKCFGTC’s translational program for the past 10 years.
“We are delighted that the UKCFGTC has continued to trust in VGXI’s manufacturing capabilities,” said Mr. Young Park, President and CEO of VGXI. “The entire VGXI team is committed to providing the highest quality DNA products and services, and it is rewarding to see these products make a meaningful impact on patients’ quality of life.”
The two companies have been working on CFTR plasmid product development since 2005. Through their collaboration, VGXI’s expert scientists have significantly boosted production yields through plasmid re-design and optimization of the fermentation and recovery process. The company is committed to providing UKCFGTC large-scale manufacturing support as its clinical trials advance to later stages.
In an earlier report on cystic fibrosis, the FDA recently announced the approval of a new therapy that treats Cystic Fibrosis (CF). The drug, called Orkambi, is manufactured by Boston-based Vertex Pharmaceuticals Inc. and treats the underlying cause of the disease in patients who have two copies of a certain mutation.
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