The cell and gene therapy capabilities journey for local players in the Asia-Pacific region

There are five key milestones that are crucial for the success of players in the CGT arena. The graphic below maps some of the local players against these milestones.

1. Developing CGT technology

The development of CGT technology entails developing the product itself, often with the involvement of academic institutions or academic medical centers. Similar to global products such as Kymriah and Zolgensma, which had their origins in academic labs, many of the promising pipeline therapies in the region are also arising out of academic collaborations.

Important considerations for companies at this stage include raising funding to support proof of concept, and decisions around in-house development versus in-licensing the intellectual property for CGT technology, such as biomaterials and vectors. Across the APAC markets, most innovators are currently focused on this stage as they look to optimize the therapy design and delivery.

 

2. Scaling up CGT capabilities

Once the technology has been developed, scalability becomes a key consideration. Companies that develop CGTs in emerging APAC markets aim to keep overall research, development, and manufacturing costs low to meet local and regional willingness- and ability-to-pay thresholds in resource-limited settings.

A recent example of a biotech currently at this milestone is ImmunoACT. During the technology development phase, the company kept costs low by leveraging frugal research and development costs in local academic settings. And as it now scales up, the company is using a locally trained workforce and limiting intellectual property costs by building its own value chain. It also plans to decentralize the manufacturing process by building more good manufacturing practice (GMP)-certified facilities to meet demand. Projected production costs with a regional decentralized manufacturing model, at a scale of 300 patients per year, are approximately US$15,000 per patient. ImmunoACT estimates that it may be able to provide the chimeric antigen receptor T-cell (CAR-T) at US$36,000 to US$48,000 once fully scaled up.

Another company of interest is Curocell in South Korea, which is about to go to market with its CAR-T, representing the first APAC company outside of China and India to do so.

 

In-market CDMO support

Companies may also rely on the services of Contract Development and Manufacturing Organizations (CDMOs) to tap into established local manufacturing and supply-chain ecosystems. This strategy may also offer an opportunity to develop self-sufficient CGT value chains in the region. Local GMP-certified CGT manufacturing capabilities are already established in most major APAC markets, such as ACTRIS in Singapore, Samsung Biologics in South Korea, and Wuxi AppTec in China.

 

3. Commercializing the CGT

For local biotechs, commercialization means focusing on successful local market penetration but could also involve expanding horizons by exporting innovations internationally. Companies such as Belief BioMed, with a hemophilia B gene therapy, and Immuneel, with another CAR-T, have stated explicit goals of making these therapies more accessible in their home markets of India and China. Other manufacturers, such as Legend and ImmunoACT, are expanding their use of out-licensing or joint partnership collaborations to enable access to innovative therapies across emerging markets or even globally.

 

Exporting the CGT to other markets

The ability of local manufacturers to export CGTs outside their home countries is often limited by a lack of long-term evidence, resulting in evidence packages that may not meet the requirements for approval in the USA or EU. However, these products could find willing takers in the emerging markets of Southeast Asia, Africa, or Latin America, where they would address an almost completely unmet medical need, and where the affordability challenges make these new, potentially low-cost CGTs more attractive.

 

4. Basic financing for the CGT

As products are commercialized, more companies are seeking to enable access for populations that are unable to tap into self-pay channels for high-cost therapies.

 

5. Comprehensive CGT financing

While research and development is sufficiently advanced in APAC, companies have yet to bridge the financing gaps for these therapies in the region. In the future, CGT manufacturers may borrow the innovative financing mechanisms that have been tested with other high-cost therapies. For example, in China, city commercial health insurance (city-CHI) plans with affordable annual premiums offer coverage for diseases in addition to basic statutory insurance, and are often used to reduce out-of-pocket payments after public reimbursement. When Carteyva, a CAR-T product manufactured by JW Therapeutics, did not qualify for public reimbursement in China, the company started exploring the city-CHI route. As a result, 25%-40% of the Carteyva RMB 1.2 million per injection cost is covered by over 20 city-level CHI plans today, helping to address affordability issues.

 

 

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