Gene Therapy is really the hot topic right now in the field of Biotech especially with technologies such as CRISPR and CAR-T therapies. The value of such companies are skyrocketing and many see the field as the next potential blockbuster therapies.

Gene therapy has clearly a huge potential but also lot of challenges, let’s go through them thanks to a panel discussion hosted at Bio-Europe Spring in Paris, including Frédéric Revah, CEO of Genethon, Marina Cavazzana, Head of Department Biotherapy – Necker Hospital of Paris, Alexandra Glucksmann, COO Editas Medicine, and Clark Pan, VP head of discovery therapeutics, Shire.
First question you might ask yourself is why is the field emerging now? The field is now around since 20 years. initially, gene therapy had a very low value because it was unknown. Now, it is less risky and more companies had successes with it as for example Bluebird Bio, Boston-based Biotech with the biggest market cap in the gene therapy field (watch the CEO’s exclusive interview). Every new field takes time to emerge and failure/successes of other before creates valuable knowledge for the whole sector.

CRISPR is clearly the hot topic of the moment in Boston. The main patents are from the Broad Institute, huge research institute formed by the MIT and Harvard. The most-financed company in the field is Editas, where Alexandra Glucksmann is from. She was asked what type of indication she would think CRISPR could cure first? She thinks the first indication to be treated will have clearly identified clinical endpoints and matches with the new technology. The regulations and knowledge from the clinicians side should also be well defined to be able to execute clinical trials much faster.

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As I mentioned, academia contributed a lot to the field wether it is the Broad or Genethon or the Necker Hospital. Now there is lot of industry interest, Marina Cavazzana were asked if this would change anything for their academic strategy. She answered that program won’t change, only the number of programs could be more important with more fundings.

Besides discovery and financing, which may be the biggest challenge in this field is the manufacturing. All industry panelists agreed on that point. The manufacturing process on one product is highly specific and each product can have different manufacturing processes. Genethon see manufacturing as the main challenge, that’s why they opened a bioproduction site south of Paris in Evry to solve this issue. On the other hand, Frederic Revah is talking with lot of VC-backed companies and they are not considering manufacturing as a challenge. They think it is a commodity as it is in monoclonal antibodies production. Alexandra was more optimistic thinking that all the manufacturing knowledge is improving very rapidly and that it should be solved quickly. It is an important opportunities for many companies, as again for example Bluebird bio which has a unique manufacturing process of highly pure viruses for their own therapies, but also for CMO which could differentiate from their competitors. Even VCs could be interested in the field of manufacturing because it could show high return of investment.

Gene Therapy is definitely an exciting field which solve huge unmet medical needs and is a real breakthrough in doing medicine. But it is also facing challenges as manufacturing processes and reimbursement policies. The field should continue to evolve quickly as it did in the last 20 years after it was born and I look forward observing this exciting development.

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