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European Biopharmaceutical Review

Pooling Efforts to Drive Gene Therapies for Rare Diseases

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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Christian K Schneider MD is Head of Biopharma Excellence and Chief Medical Officer (Biopharma) at PharmaLex. He was previously interim Chief Scientific Officer at the UK’s MHRA, where he was also Director of the National Institute for Biological Standards and Control for five years. Christian has also held leading positions at the Danish Medicines Agency and at the Paul-Ehrlich- Institut, Germany’s Federal Agency for Vaccines and Biomedicines. At EMA, he has chaired the Committee for Advanced Therapies, as well as the Biosimilar Medicinal Products Working Party, and served as a member of the Committee for Medicinal Products for Human Use. Christian is one of the key architects of EMA’s advanced therapies and biosimilars framework. As a regulatory scientist, Christian has published over 50 articles in international, peer-reviewed journals.
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christian.schneider@biopharma-excellence.com
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