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Turning science fiction into true science

NLSDays 2017 started off with a super session with the title “Gene therapy in regenerative medicine” and both challenges and possibilities were discussed.

Dr Sven Kili, Head of Cell and Gene Therapy Development at GSK, gave the audience a comprehensive overview of the progress and developments within the field of gene therapy, including the facts that today, there are 822+ companies in the world working in this field and 2.45 billion dollars are spent on financing these solutions. It is a rapidly growing area and by mid -year 2017 there were 899 clinical trials,  67 of these in Phase III.  Pharmaceuticals like Zalmoxis, Holoclar, Epicel and Strimvelis have been developed. He also mentioned diseases where gene therapy have had a major impact, for example SCID, Wiskott Aldrich syndrome and metachromatic leukodystrophy. Kili also spoke about the challenges these new technologies are facing.. Two of them are manufacturing and affordability, making a return on them.

Ralf Knöll, Chief Scientist at AstraZeneca, acknowledged important milestones in the technology, including the most recent CRISPR/Cas technique. His company address for example a disease area that is extremely multi complex, heart failure. It is also an area in very much need of better treatments, cardiovascular disease affacts 17.3 million deaths a year. Through precise genome editing and CRISPR/Cas9 they are developing treatments for this complex disease.

Aiming to take gene medicine to the masses

Anthony Cheung from the Canadian company enGene spoke about how he and his colleauges wants to take gene medicine to the masses, and that delivery is an extremely important and challenging issue. His company are developing a solution that targets the gut, uses non viral vectors (less expensive), are orally available and uses a high flexible platform and scalable and low cost manufacturing.

Regulatory challenges

Steffen Thirstrup from the NDA group continued the session with a speech about regulatory challenges for advanced gene therapy. For example he mentioned that it is difficult to live up to the GMP requirements and to continously live up to these requirements. There are several reasons to why ATMP fail, quality, non clinical issues (proper safety data is needed) and risk management for example, but the big stopping block, he said, is efficacy. Another challenge is hospital exemption or special authorization provisions. He spoke about what EMA offers, like SME status, Innovation task force, CAT classification, Certify of quality and non clinical data, Scientific advise and PRIME (PRIority MEdicine). Thirstrup also said that a challenge is actually that the science is ahead of development processes, and it is a challenge to be in the frontline if a field of such fast evolvning science.

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