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Gene therapies’ potentials, challenges explained

Jeong Sae-im  Published 2019.04.22  11:46  Updated 2019.04.22 11:46

공유
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Can Korea lead the global market of innovative gene therapies?

Healthcare experts discussed the issue sharing various approaches to overcome the limitations of gene therapies, in a forum on the sidelines of BIO KOREA 2019 at COEX, southern Seoul, last Friday.

Professor Kim Jeong-hun of the Department of Ophthalmology at Seoul National University College of Medicine speaks during BIO Korea 2019 at COEX, southern Seoul, on Friday.

Professors who participated in the forum said in a chorus that Korea was ahead of Japan in the gene therapy market, although the nation has been lagging behind the neighboring rival in cell therapies.

Speakers included Seoul National University College of Medicine’s Ophthalmology Department Professor Kim Jeong-hun, Chungnam National University Graduate School of New Drug Discovery and Development Professor Kim Yeon-soo, Hanyang University Chemistry Department Professor Bae Sang-su, and Yonsei University Mechanical Engineering Department Professor Ryu Won-hyoung.

“Korea finds it difficult to catch up with Japan in cell therapies because Japanese companies invest 200 billion won ($175.3 million) every year, Professor Kim at SNU said. “However, Korea has a chance in gene therapies, and local researchers are ahead in gene editing,”

In ophthalmology, Korea was the first to publish a paper trying out a gene editing technique on a common disease, aged macular degeneration, which is not a genetic disorder, he added.

However, gene therapies face many challenges. The biggest hurdle is astronomical prices. For example, Luxturna, gene therapy for the treatment of Leber's congenital amaurosis approved by the U.S. Food and Drug Administration, costs about 900 million won ($790,146) for both eyes. Theoretically, a single treatment should cure the disease, but in reality, a patient reportedly needs the treatment every decade. In other words, it costs about 100 million won a year to get gene therapy.

“I wonder if patients can pay 100 million won every year to get the treatment. Prices of the gene therapies should be lowered after all,” the SNU professor said.

The off-target effect – an unintended genetic modification -- is another problem in gene therapies, the advancement of technologies could resolve the off-target effect issue, Professor Bae said. “There is a tool that detects off-target. Although it is a simple prediction program now, there are more accurate tools being developed,” Bae said.

Opportunities for gene therapies are broadening with the easing of regulations. The shift in the FDA’s review for gene therapies will be a significant opportunity for the market players, observers said.

Early this year, the FDA decided not to apply a general review standard when approving cell and gene therapy. Instead of strict and meticulous inspections being applied to treatments in the other sector, the FDA will allow a quick and easy approval procedure on the condition that it will put more weight in long-term follow-ups.

The decision is based on a logic that it is almost impossible to check all the risks related to gene therapies during clinical trials before a review.

Another major shift in the FDA’s review of gene therapy is that the regulator considers “chemistry, manufacturing and control information” the most important.

The U.S. National Institute of Health also announced that it would abolish preliminary review guidelines for gene therapy products.

“The U.S. is seeking various changes, such as allowing conditional approval for gene therapy on rare and intractable diseases, to help gene therapies quickly arrive in the market,” Professor Kim Yeon-soo said. “Many clinical trials using genetic editing are progressing worldwide, and these technologies will lead gene therapies.”

Professor Ryu said microneedles could be used in gene therapies to boost efficiency. Microneedles are used for drug delivery in the body, often in vaccinations or cosmetic products. However, they can be applied to gene therapies, he added.

“A paper said that in-vivo editing was much less effective than in-vitro editing in an animal experiment. Microneedles will be able to raise efficiency by transmitting mRNA and Cas9,” Ryu said.

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