Bespoke Gene Therapy Consortium – cooperation for rare disease therapies

At the Bespoke Gene Therapy Consortium (BGTC, website), a consortium for the treatment of rare monogenetic diseases, they aim to treat neglected diseases with a very small number of patients with a focus solely on adeno-associated virus (AAV) vectors and a standardized translation playbook, believing , that more open collaboration between researchers, clinicians, companies and patients can make these therapies viable and accessible.
Bgtc Pathways
The BGTC consortium was founded in 2021 as part of the Advanced Medicines Partnership (AMP) program, in which leading American experts and organizations participate under the auspices of the US National Institutes of Health (NIH). The consortium has set the following goals: to make adeno-associated virus (AAV) technology more accessible and transferable for use in more diseases, to lower the cost of preclinical and clinical procedures, to enable scientific and regulatory advances for easier translation, and to make gene therapies accessible to all affected patients.
The consortium aims to achieve these four goals in two ways. According to one, they are working to collaboratively find ways to create AAVs cheaper, as well as to achieve better gene expression in the patient’s body. On the other hand, mainly in cooperation with regulatory authorities and experts for the registration of medicines, they prepare and update a regulatory playbook (link), in which they provide a transparent way for the developers of new medicines to go through all the necessary regulatory procedures, including suggestions, examples and explanations.
In CTGCT, we estimate that a similar approach in the European Union could contribute to the same goals. For this purpose, in the future we will be more closely connected with BGTC, establish similar cooperation in the EU, or join initiatives that will pursue similar goals.