The National Institutes for Health (NIH), Food and Drug Administration (FDA) and 15 pharmaceutical and non-profit organisations have joined together to accelerate the development of gene therapies in the USA.
Taken together, rare diseases affect an estimated 30 million Americans. Forming the Bespoke Gene Therapy Consortium (BGTC), the partnership is hoping to overcome obstacles and streamline the gene therapy development process to provide customised treatments for people affected by rare diseases.
'The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivise more companies to invest in this space and bring treatments to patients.' said Dr Joni Rutter, acting director of NIH's National Centre for Advancing Translational Sciences.
There are thought to be at least 7000 rare diseases and since most can be traced to variations or changes in a single gene, gene therapy provides a promising therapeutic approach in many cases. However, gene therapy development for rare disease is highly complex, expensive and time-consuming.
'Because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualised gene therapy treatments for a single disease to market,' noted Dr Rutter.
So how exactly are the consortium planning on overcoming these issues? By using a common gene delivery vehicle known as the adeno-associated virus (AAV) to deliver or activate therapeutic genes within patients' cells.
The consortium will focus on developing a better understanding of how AVV works. The programme then plans to implement between four and six clinical trials for different rare diseases that have no therapies in development, with the aim to reduce the time between testing animal models of the disease and human clinical trials. Expected outcomes include standardised analytical tests for viral vectors to improve efficiency of manufacturing processes and a streamlined regulatory process for the FDA approval of gene therapies to speed up access to these therapies for people who could benefit.
'There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies.' said Dr Francis Collins, NIH director.