Welcome to the 6th Annual Gene Therapy for Rare Disorders Europe Summit 2022

Whilst a historically low success rate has been the case for progressing from development to market, currently over 70 gene therapy products are in European clinical trials, promising to turn a corner in the treatment of rare disorders. Now more than ever, it is paramount to match the significant recent investment in this space with an understanding of what the regulators are looking for, all to bring safer and more effective gene therapies to the patients who need them.

We’re excited to bring back the first in-person event in the series since 2019 with the added value this format brings to roundtable sessions, interactive workshops, and dedicated networking sessions. You can expect a streamlined one track summit, packed with insight into all aspects of the gene therapy development pathway whilst shining a lens on the key regulatory challenges developers need to understand to progress their products to launch.

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Optimise clinical trial design, successfully defining endpoints and demonstrating drug safety and efficacy to the regulators, with the help of insights from GenSight and uniQure

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Navigate complex manufacturing challenges, with teachings on how to remove productivity bottlenecks and achieving long-term product consistency from the likes of Roche

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Explore new European regulatory submission platforms and leverage the expertise of numerous senior regulatory affairs specialists as well as from the regulatory bodies themselves, including the EMA

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Align clinical development processes with patient needs by understanding their attitudes towards risk-benefit and their work with the regulatory bodies, with insights from the MHRA

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Understand how to develop a comprehensive translational strategy for preclinical programmes to transition successfully into clinical development, with help from Atsena Therapeutics