Managed by the Foundation for the National Institutes of Health (FNIH), the consortium aims to accelerate the process of gene therapy development and manufacturing for rare diseases
Genetic medicines manufacturer Forge Biologics has joined the Bespoke Gene Therapy Consortium (BGTC) to expedite the development and manufacturing of Adeno-associated virus (AAV) gene therapies for patients with rare diseases.
BGTC collaboration is part of the Accelerating Medicines Partnership (AMP) programme, a public–private partnership between the National Institutes of Health (NIH), and the US Food and Drug Administration (FDA).
The partnership also includes multiple pharmaceutical and life sciences companies and non-profits and other organisations.
Managed by the Foundation for the National Institutes of Health (FNIH), the consortium aims to speed up the process of gene therapy development and manufacturing for rare diseases.
BGTC was established to fill the gap in the current drug development model, which makes it challenging for businesses to recover the expenditures associated with developing gene therapies for patients with rare diseases.
The consortium is expected to eliminate development hurdles and enable significant progress against several rare diseases by developing a standardised, reusable method that lowers upfront expenses.
Forge Biologics regulatory affairs and quality senior vice president Christopher Shilling said: “The tools and resources for clinical development, particularly large-scale platform cGMP manufacturing and regulatory evaluation of AAV therapies will provide a much-needed standardised approach, enabling meaningful progress for rare disease gene therapy development.
“Forge is committed to supporting this collaboration with the NIH, the FDA, and like-minded partners on the BGTC, to provide a critical development platform that will enable the future of AAV therapies for patients.”
The BGTC is developing platforms and standards to speed the creation and distribution of specialised or bespoke gene treatments for patients with rare diseases.
It is the sixth AMP initiative overall and the first AMP effort with a rare disease component. Additionally, it is the first to focus on a therapeutic platform as well.
BGTC programme lead Courtney Silverthorn said: “As one of the largest AAV manufacturers in the world, Forge Biologics has the necessary industry expertise and manufacturing capacity to be a meaningful partner to the BGTC.
“We are certain their knowledge and capabilities will help the BGTC realise its goal of streamlining the drug development process to reduce costs and enable companies to bring more gene therapies to patients.”