Asia Pacific takes the lead in cell and gene clinical trials


The Asia Pacific region has taken the lead in cell and gene clinical trials, accounting for the largest share of new trials during the first half of 2022, according to the Alliance of Regenerative Medicine’s H1 2022 report that maps the sector’s progress.

The APAC region represents 42 per cent (61) of the 144 new clinical trials started this year and includes a healthy number of trials early in the pipeline (30); 26 in phase 2, and 5 in phase 3. The European pipeline has slowed “to a trickle” with 16 new clinical trials started in H1 2022, however, is the only region that is bottom-heavy.

By region, North America leads with 808 active clinical trials, followed by the Asia Pacific with 640 trials, Europe with 329, and 88 active trials in all other regions.

Cell and gene therapies are a global activity, and there is an exciting opportunity to change the treatment paradigm. This is reflected in the three new therapies approved so far in 2022, including Legend Biotech & Janssen CAR-T therapy (US & EU); BioMarin Pharmaceutical gene therapy (EU); and PTC Therapeutics gene therapy (EU); as well as four therapies approved in new geographies or for new indications – including Novartis’ CAR-T therapy Kymriah, making it the only CAR-T therapy approved for both adult and pediatric settings.

Read more about Kymriah in the case study, published in October 2021 and developed by the AusBiotech-led Regenerative Medicines Consortium Project.

It has already been a record year for the approval of new gene therapies to treat rare diseases, with more yet expected: four further regulatory decisions are anticipated in the final months of 2022 and 16 in 2023, including CSL Behring’s therapy to treat Hemophilia B.

To capitalise further on the opportunities that Australia’s burgeoning cell and gene sector can deliver to the Australian economy and society, AusBiotech, with support from the Australian Medtech Manufacturing Centre, is conducting detailed research to develop a ‘blueprint’ outlining implementation strategies for manufacturing cell and gene therapies medicines locally. This research will build on the work published in Australia’s Regenerative Medicines Manufacturing Capacity and Capability, and will work in harmony with Australia’s Cell and Gene Catalyst.

Building Australia’s sovereign manufacturing capability for complex and advanced cell and gene therapies offers the opportunity for equitable access to cutting-edge treatments for Australian patients and therefore reduce the burden of disease significantly, leading to better health outcomes for Australians and higher participation rates in the workforce, and additionally to create a new highly skilled workforce now and for the future, thereby cumulatively impacting the economy on a large scale. 

Read the Alliance of Regenerative Medicine’s 2022 H1 Report here.