Gene therapy gathers pace

Gene and cell therapy concept image

Source: © Shutterstock

Sarah Houlton charts the evolution, challenges and opportunities of cell and gene therapy

Since the first human gene therapy trial was carried out nearly 30 years ago, on children with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID, also known as bubble baby syndrome), there have been several product approvals – and a lot of optimism, despite the challenges. According to Research and Markets, more than 2200 clinical trials are under way, and the market is predicted reach $363 million (£271 million) by 2020. Gene therapy now promises potentially transformative therapies for many forms of cancer, plus conditions such as haemophilia and a multiplicity of rare diseases.