LONDON (Reuters) - Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones: 1972 - Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans "for the foreseeable future", pending greater understanding of the technology. 1990 - A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States. 1999 - American patient Jesse Gelsinger dies following a gene therapy experiment, setting the field back several years as U.S. regulators put some experiments on hold. 2002-03 - Cases of leukaemia are diagnosed in French children undergoing gene therapy in a further blow to the field. 2003 - The world's first gene therapy is approved in China for the treatment of head and neck cancer. 2007 - Doctors carry out the world's first operation using gene therapy to treat a serious sight disorder caused by a genetic defect. 2012 - Europe approves Glybera, the first gene therapy in a Western market, for an ultra-rare blood disorder. 2016 - Europe approves Strimvelis for a very rare type of immunodeficiency. 2017 or 2018 - The first gene … Continue reading →

Pfizer has chosen a site in Sanford, North Carolina for a gene therapy production plant, just 40 miles from its recent acquisition Bamboo Therapeutics Inc. The US drug firm had been search for a site since March. According to North Carolina Governor Roy Cooper, Pfizer will spend $100m (85m) on the new facility and has also committed $4m to support postdoctoral fellowships in North Carolina universities for training in gene therapy research. The project will create jobs that deliver a total payroll impact of more than $3.9m each year to the community according to the North Carolina Department of Commerce and the Economic Development Partnership. The project will be part funded by a $250,000 grant previously awarded to Wyeth which was acquired by Pfizer in 2009 - by the One North Carolina Fund, which helps local Governments attract economic investment. Bamboo buy The decision follows a little over a year after the US drug manufacturer acquired Bamboo Therapeutics, a North Carolina-based gene therapy developer. The deal included a recombinant Adeno-Associated Virus (rAAV) vector design and production technology, a Phase I candidate for Giant Axonal Neuropathy and a preclinical programme targeting Duchenne Muscular Dystrophy (DMD). Pfizer also gained a 11,000sq ft … Continue reading →

A University of Chicago-based research team has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases. In a study inthe journal Cell Stem Cell, the researchers provide proof-of-concept. They describe gene-therapy administered through skin transplants to treat two related and extremely common human ailments: Type 2 diabetes and obesity. We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems, said study author Xiaoyang Wu, assistant professor in the Ben May Department for Cancer Research at the University of Chicago. We think this platform has the potential to lead to safe and durable gene therapy in mice and, we hope, in humans, using selected and modified cells from skin. Beginning in the 1970s, physicians learned how to harvest skin stem cells from a patient with extensive burn wounds, grow them in the laboratory, then apply the lab-grown tissue to close and protect a patients wounds. This approach is now standard. However, the application of skin transplants is better developed in humans than in mice. The mouse system is less mature, Wu said. It … Continue reading →