BARCELONA – The closing speech honors at the record-setting European Society of Gene & Cell Therapy in Barcelona last week went to Katherine High, MD, the co-founder, president, and CSO of Spark Therapeutics.

Katherine High
Katherine High, MD

As the developer of Luxturna, the first approved gene therapy for a hereditary form of blindness called Leber’s congenital amaurosis, High is well positioned to talk about the commercial success and renaissance of gene therapy. Last week, the Federal Trade Commission recommended that Spark’s $4.3 acquisition by Roche should proceed. Luxturna, developed by University of Pennsylvania researchers Jean Bennett and Al Maguire, has a list price of $425,000 per eye.

“We’re beginning to see more and more products for genetic disease,” High said. Luxturna joins several recent gene therapy approvals, including Glybera (since withdrawn in Europe), Strimvelis, Zolgensma, and Zynteglo. Coming soon are therapies for hemophila, ADA-SCID, X-linked myotubular myopathy, and mucopolysaccharidosis types I and II.

With more than 800 INDs currently under active management at the FDA, High offered a series of future challenges for the gene therapy field. They include: 1) scientific research; 2) manufacturing capacity and capability; 3) regulatory procedures; 4) clinical development; 5) operational integration of big data with trial population selection; and 6) workforce.

On the scientific research front, High said the major issues were achieving global gene delivery to the central nervous system; expanding the cargo of AAV vectors to accommodate larger genes; and immunogenicity.

The importance of scaling up manufacturing was evident in two recent blockbuster acquisitions: Thermo Fisher Scientific’s purchase of Brammer Bio for $1.7 billion, and Catalent’s acquisition of Paragon Bioservices for $1.2 billion. Regulatory frameworks in the United States and Europe needed more agreement and convergence, High said. And in clinical development, there was a need “to validate endpoints in clinical trials for diseases that previously lacked therapies. We must find ways to accept novel endpoints.”

Economics—the most important omics

One topic that High did not address in her remarks was that of drug pricing, especially the exorbitant pricing attached to newly approved therapies such as Zolgensma (more than $2 million per patient). However, the topic came up in the audience questions. High responded by distinguishing gene therapies for diseases where there is an existing therapy with therapies for diseases without a current treatment:

“When you think about diseases like hemophilia, where people already pay something like $300,000 a year for their protein therapeutics or even more, then I think it’s not so difficult to figure out that, in fact, gene therapy may save money… most people in the western world with severe hemophilia have a nearly normal lifespan, so if you think about 65–70 years of a clotting factor concentrate, it is likely that gene therapy will be less expensive, no matter what is charged.

“The question really comes for diseases that lack any therapy right now, so essentially they don’t cost anything. Even for something like thalassemia, our current medical regimen is pretty complex and expensive, and often doesn’t have such a great outcome. But what about things like Luxturna, where there was previously no treatment so no cost to the health system? I think that it would every unfortunate if the conclusion of all that was that gene therapy should only go forward for things where we’re off-setting costs to the current health system. We’re going to have to find a way to pay for them if we want to develop therapies for diseases that right now don’t cost us anything.

“I don’t have the solution.”

High ended her speech by quoting the author Carlota Perez, who wrote in 2002: “[The] full fruits of technology revolutions are only reaped with a time lag. Two or three decades of turbulent adoption and assimilation elapse from the moment when the set of new technologies, products, industries, and infrastructure make the first impact to the beginning of a golden age.”

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