SEOUL – South Korea's investors have become very interested in the global cell and gene therapy market. Licensing and M&A deals in the field have been active – a good sign for Korean biopharma firms eager to tap in.

However, there are some barriers to development, such as government regulations, negative results of certain clinical trials and the side effects after drug administration. Investors and biopharma execs at the Korea Bio Investment Conference (KBIC) in Seoul last week said they are aware of both the market potential and hurdles.

Cell and gene therapy M&A and licensing deals started to heat up in 2017. A few examples:

  • • Gilead Sciences Inc., of Foster City, Calif., acquired chimeric antigen receptors (CAR T) cell therapy manufacturer Kite Pharma Inc., of Los Angeles, for $11.9 billion.

  • • Janssen Biotech Inc. signed a license agreement with Genscript Biotech Corp. to develop and commercialize CAR T-cell drug candidate targeting the B-cell maturation antigen for the up-front payment of $350 million.

  • • In the first half of 2019, the total deal amount jumped up to $94 billion. F. Hoffmann-La Roche AG based in Basel purchased U.S. gene therapy startup Spark Therapeutics Inc., of Philadelphia, for $4.8 billion.

Wan-sung Ku, analyst at NH Investment & Securities Co. Ltd., of Seoul, said there are key trends in the global cell and gene treatment market that Korean biotechs should focus on.

First, virus vector technology has become very important in gene therapy development. The vectors work as tools to deliver genetic material into cells efficiently, and the technology will be significant to enhance therapeutics.

Ku said that FDA approval of Novartis AG's Zolgensma (onasemnogene neparvovec), a drug for pediatric patients with spinal muscular atrophy, in May was a milestone for virus vector use in gene therapy. It was the first regulatory approval based on novel AAV vector (NAV) technology developed by Regenxbio Inc., based in Rockville, Md.

Use of RNA has expanded to increase pipelines. Moderna Inc., of Cambridge, Mass., is developing its messenger RNA platform with Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J.

Using the platform, Moderna has expanded its pipeline from a prophylactic vaccine to a cancer vaccine and from localized regenerative therapeutics to systemic intracellular therapeutics to treat chikungunya virus and methylmalonic academia (MMA).

However, some research data indicate the cell and gene therapy market will not grow as expected.

According to Inkwood Research, the market size of stem cell therapy is expected to increase from $8.7 billion in 2018 to $19.7 billion in 2027 with a 9.5% compound annual growth rate (CAGR). The CAGR was expected to be 25% when it was estimated in 2015.

Regulations on approval and use of stem cell therapy in each country may slow down the business, Ku said.

"Europe set up the regulations on stem cell treatment, however, only one stem cell therapy has been approved so far. In Japan and China, stem cell therapy is practiced but it is not regarded as medical treatment to cure disease," he said.

Stem cell therapy is not currently allowed in Korea. However, clinical research is expected to develop faster under the new Advanced Regenerative Medicine and Advanced Biopharmaceuticals Safety and Support Act, which passed the country's National Assembly plenary session in August and will take effect in 2020. (See BioWorld, Aug. 14, 2019.)

The new act is designed to encourage stem cell therapy R&D as it aims to introduce a fast track for advanced biopharmaceutical approval, supporting clinical research into regenerative medicine to increase opportunities for rare and obstinate disease treatment.

"The act would accelerate gene and stem cell therapy R&D and shorten the advanced biopharmaceutical products' market launch period by around four years." Ku told BioWorld.

Also, there are some side effects after CAR T administration. Symptoms of cytokine release syndrome (CRS) include fever, headache, rash, rapid heartbeat, low blood pressure and trouble breathing. Ku said more than 90% of patients who were administered CAR T experienced CRS, according to American Society of Clinical Oncology.

"Use of tocilizumab or steroid may lower the possibility of CRS. A technology of switchable CAR T, which can control the drug activation period, would be a solution. Abclone Inc., a Seoul-based biotech that secures switchable CAR T technology, is expected to lead the solution in Korea," he noted.

The conference was hosted by the Korea Economic Daily and the Korea Biotechnology Industry Organization.

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