Executives from Frost & Sullivan attend CGT Asia 2021, an event on Asian cell and gene therapy innovation

At the meeting, Mao Hua delivered a keynote speech titled 'Market Opportunities and Challenges in Cell and Gene Therapy Industries'. He pointed out that the global cell and gene therapy (CGT) market began in the 1960s and has gone through four development stages. The foundation of CGT clinical research is becoming increasingly mature. Currently, China ranks second globally in terms of the total number of CGT clinical studies, and in categories such as CAR-T, China is the largest clinical research initiator in the world. In recent years, continuous technological breakthroughs have brought about another boom in CGT, subverting the entire pharmaceutical ecosystem and also changing the way humans treat genetic diseases and complex conditions.

He further explained: 'Rare diseases affect the health of nearly 350 million people worldwide. Most causes are due to gene mutations or inheritance, including single-gene genetic diseases such as hemophilia, as well as polygenic diseases like cancer, neurodegenerative diseases, eye diseases, and cardiovascular diseases. More than 95% of these genetic diseases have no effective treatment methods. The use of traditional drug therapies such as small molecules or enzymes can alleviate patients' pain to a certain extent, but this treatment requires continuous medication, which is extremely costly for most patients, increasing their economic and socio-psychological burden. At the same time, drugs not only fail to cure genetic diseases fundamentally but also cause serious complications.'

CGT therapy has become the ultimate weapon on the path to conquering rare genetic diseases, bringing survival possibilities and hope to patients. He cited an example: in the case of typical rare disease hemophilia A, gene therapy offers greater clinical benefits compared to other treatment methods. CAR-T therapy also shows significant therapeutic advantages for advanced or relapsed B-cell leukemia and lymphoma that are otherwise untreatable. "Currently, most of the marketed products for gene therapy are for treating rare diseases, with original research manufacturers being mostly innovative companies focusing on cell and gene therapies. However, over decades of gene therapy research and development, several barriers have been introduced, including R&D technology, cost, production, safety, commercialization, and legal regulation, which have restricted the rapid approval process for products."

Currently, multinational biotech companies are accelerating their cooperation with local firms through licensing and joint ventures to ensure the registration speed of CGT products and acquire localized production and commercial capabilities. Local biotech companies actively cooperate with industry associations to jointly formulate product quality and production process standards to guide industry development. At the same time, local governments have established alliances with industry leaders to jointly promote regional infrastructure construction. With the first wave of commercialization of cell therapy approaching, reagent and equipment suppliers such as Thermo Fisher and General Electric are closely collaborating with CGT companies to advance the automation and industrialization of cell therapy processes.