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| home > ebr > winter 2022 > pooling efforts to drive gene therapies for rare diseases |
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European Biopharmaceutical Review
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When multiple organisations pool their efforts, it promotes affordability and return on investment. Gene therapies, which are expensive and complex to develop and study, and whose target populations are very small, are prime candidates for reaping the benefits of collaboration.
To facilitate cooperation, the US-based Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021, designed to encourage the delivery of more gene therapies for rare diseases. The consortium is headed and funded by the FDA and the National Institutes of Health. Other stakeholders range from large and small biotechs and pharma brands to non-profit organisations.
The Potential to Reduce Costs and Accelerate Approvals
The potential benefits of multiple stakeholders banding together could be significant and provide a model for other markets to follow. If a standardised platform can be agreed, with each drug developer sharing findings and building on what has gone before, the potential to reduce costs and accelerate approvals could be significant. As barriers crumble, new momentum might build as more pharma teams are able to see a clearer path to market. The ultimate beneficiaries will be patients, who could find themselves with access to breakthrough treatments to cure their condition, extend their life expectancy, or improve their quality of life.
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