China's First CAR-T Therapy Nears Approval, Unlocking New Opportunities in Cell and Gene Therapy Market

According to the official website of the National Medical Products Administration (NMPA), the marketing authorization application in China for Fosun Kite's CD19-targeted CAR-T therapy, axicabtagene ciloleucel injection, is currently in the "under review" stage and is expected to receive NMPA approval for commercial launch in the near future. This drug will become the first CAR-T cell therapy to be marketed in China.

Wallstreetcn· Jianzhi Research Institute

According to statistics, as of now, JW Therapeutics' JWCAR029 has been granted the dual regulatory designation of "Priority Review + Breakthrough Therapy" and is currently under regulatory review and approval. Legend Biotech's BCMA CAR-T product is expected to be launched in the United States in H2 2021. Carsgen Therapeutics' CAR-T therapy has received 7 IND (Investigational New Drug) approvals for clinical trials across China, the United States, and Canada.`License`. With the market launch of the first CAR-T product, cell and gene therapy will once again attract the attention of the capital market.

Why Will Cell and Gene Therapy Attract the Attention of Capital Markets?

Cell therapy refers to the utilization of autologous or allogeneic adult cells or stem cells from patients, employing biotechnological...Cheng FangA treatment method in which, after undergoing processing steps such as acquisition, isolation, culture and expansion, and screening, cells are endowed with therapeutic effects including enhancing immune function, eliminating pathogens and tumor cells, and promoting tissue and organ regeneration as well as systemic recovery, followed by the artificial infusion (or implantation) of these cells into the human body.

Gene therapy refers to the introduction of normal genes into human cells to modify abnormal genetic sequences. Primarily targeting rare genetic diseases, it is one of the therapeutic modalities with the greatest potential for achieving a cure.

Wallstreetcn· Jianzhi Research Institute believes that, cell and gene therapy can be regarded as the next generation of therapeutics for cancer and rare diseases, following blockbuster agents such as monoclonal antibodies, bispecific antibodies, and antibody-drug conjugates (ADCs). They bring us a step closer to curing cancer and rare diseases and theoretically offer superior efficacy. For cell therapy, oncology represents its most promising area of development, as these engineered immune cells can undergo robust proliferation and expansion upon encountering tumors, thereby targeting and eradicating cancer cells while maintaining long-term persistence within the patient's body. Consequently, compared with other existing treatment modalities, they are capable of delivering enhanced therapeutic efficacy for certain malignancies.

In the secondary market, top-tier investors have also embarked on a buying spree in cell and gene therapy. Last month, Hillhouse Capital just invested HK$8 billion to acquire a stake inGenScriptBiotech and its subsidiary CAR-T manufacturer Legend Biotech, as well as the cell and gene CDMO company GenScript ProBio.

in the primary market, On March 25 this year, Hillhouse Capital led a 520 million RMB Series B investment in BeiHeng Biotech, and yesterday, BeiHeng Biotech's off-the-shelf CAR-T product CTD401 was granted Orphan Drug Designation by the U.S. FDA, forYu ZhiFor the treatment of T-cell acute lymphoblastic leukemia, this is an anti-CD7 universal CAR-T.

In the industry M&A market, financing and M&A in the cell and gene therapy sector are also in full swing. On June 8, 2021, BeiGene announced a collaboration agreement with Shoreline Biosciences to in-license four allogeneic NK cell therapies targeting four distinct targets, developed using the latter's technology, for an upfront payment of $45 million.

On June 17, 2021, Kite, a Gilead company, entered into a collaboration agreement with Shoreline Biosciences to develop targeted NK cell and macrophage therapies, with a total potential deal value of up to $2.3 billion.

As numerous innovative pharmaceutical companies enter the cell and gene therapy sector, CDMO companies specializing in this field are also poised to experience a period of explosive growth. According to Frost & Sullivan estimates, the global outsourcing market for cell and gene therapy is projected to grow from $1.2 billion in 2017 to $3.6 billion in 2022, representing a compound annual growth rate (CAGR) of 25%, significantly outpacing the industry average.

Relevant companies in China include: WuXi Advanced Therapies, GenScript ProBio, Bosheng Biotechnology, Obio Technology, etc.