Cell and gene therapy development: four Asian markets to look out for

China is a rising thought leader in gene therapy

Fourteen years before the FDA approved its first gene therapy product, China had approved Gendicine for the treatment of head-and-neck squamous cell carcinoma by replacing mutated copies of the p53 tumor suppressor gene with normal copies.

This development took the Western world by surprise where a series of failed clinical trials had dimmed public interest. Despite criticisms throughout the 2000s, a meta-study report published 12 years later for its Phase IV post-marketing surveillance trials did show good longitudinal data for its claims. Around 160,000 vials of the drug had been manufactured and opened up, for the first time, wide-spread medical tourism of Western patients towards China.

Key developments in gene therapy are now focused on the CRISPR-Cas9 genome editing technology. The technique enables the targeting, cleavage, replacement and repair of DNA in-vivo, which has profound implications in the way we deliver gene therapy.

Cell and gene therapy development: four Asia markets to look out for

Enthusiastic capital investors in the Japan gene therapy market

Japan is home to the world’s second-largest drug market as well as enthusiastic capital investors that are willing to invest in gene therapy. Fujifilm’s USD 120 million investment into its gene therapy CDMO division, and the USD 3 billion blockbuster acquisition of Audentes Therapeutics by Japanese giant Astellas Pharma, are just two of many high profile cases that have materialized within the past three months.

In early 2019, Japan opened up its gene therapy market floodgates using an expedited regulatory review process called the Sakigake designation, it is the equivalent of the FDA breakthrough therapy designation and the EMA Priority Medicine (PRIME) designation. These are also known as conditional, time-limited approval processes. The Japan Pharmaceutical Medical Device Agency (PMDA) approved two gene therapy products simultaneously under the Sakigake designation and expects to pass at least one therapy annually.

For those interested in entering the Japanese market, you should save the date of July 13, 2020, when the highly influential Japan Society of Gene and Cell Therapy will convene for the first time since PMDA approvals.

Taiwan certifying more hospitals for cell therapy treatments

Taiwan’s Food and Drugs Administration typically defers to the US FDA and Japan’s PMDA when adopting new drugs and therapies. This has been the case for gene and cell therapy too. Though it has not officially approved gene therapy, Taiwan became the ninth regulatory agency in the world to approve clinical cell therapy treatments in 2018.

This bill introduced six autologous cell therapy treatments: two in oncology, two in skin cell transplantation and two in bone and cartilage regeneration. As of January 2020, four hospitals in Taiwan are licensed to provide these treatments and 26 more have applied for the license to provide cell therapy treatments.

Taiwan is no stranger to gene therapy clinical trials. Hospitals in Taiwan are actively participating in eight gene therapy clinical trials. Of note is the trial “A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC,” which is the first trial sponsored and conducted entirely within Taiwan.

In 2020, Taipei is expected to pass the “Regenerative Medicine Regulation Draft Bill” that will enable hospitals delivering cell therapy treatments to begin providing approved gene therapy treatments as well. As such, we can expect to see significant movement in Taiwan for gene therapy research and treatment.

Largest approved cell therapy product portfolio paves way for South Korea in gene therapy

As a global leader in plastic surgeries and regenerative medicine, the South Korean market has enjoyed success in cell therapies. Having founded the second earliest approved cell therapy product, Chondron, for treatment of articular cartilage injury of the knee joint, South Korea also has the largest approved cell therapy product portfolio with a total of 18 products in the world.

In 2017, South Korea’s Ministry of Food and Drug Safety approved Invossa, a gene therapy treatment for knee osteoarthritis. Invossa has since treated over five million people mostly in South Korea, Hong Kong and Macau. It is now turning to VM202 by Helixmith for its next success, which has potential uses in cardiovascular and neurological indications like coronary artery disease and Lou Gehrig’s disease.

The South Korean administration has taken an involved approach to push gene and cell therapy. The “Act on the Safety and Support of Advanced Regenerative Medical and Advanced Biopharmaceuticals” was passed in 2019. This act allows the conditional approval of drugs that have completed phase two clinical trials for commercial sale. Industry experts expect this legislation to shorten time-to-market by up to four years.

A second proposed legislation will remove significant hurdles to gene therapy and gene editing research by amending the Bioethics Act, which currently limits research to specific and designated diseases, bringing the Act more in line with global norms. The bill is expected to pass in 2020.

About the author

Gabriela is based in Zurich and has been with DKSH for seven years helping the company to expand their business in Asia. She has previously worked with several international brands in the life sciences sector and has vast experience developing strong relationships with customers and business partners worldwide.

About the author

James Hsu joined DKSH in 2019 and is part of the Business Development, Business Unit Technology team in Taiwan. In this role, he is responsible for growing the life sciences and scientific instrumentations business. His previous experience was accumulated in the bustling Asian genomics and proteomics sector, where he worked on bringing a digital PCR startup to market. James graduated from the University of California, San Diego.