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

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’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.