Big Pharma and Biotech Firms Eye 'In-Vivo CAR-T'... One Step Closer to Legalization

In-Vivo Gene Therapy Passes Plenary Session of Welfare Committee

Major Pharma Invests Billions as Next-Generation Bio Core Technology Emerges

AbClon and Argynomics Accelerate R&D Focused on Therapeutics and Delivery Technologies

The 'In-Vivo CAR-T' therapy, which generates immune cells directly within the body, is emerging as the next-generation technology in the global pharmaceutical and biotech industries, and related regulatory reforms are also gaining momentum in Korea.

According to the political circle on April 1, the National Assembly Health and Welfare Committee held a plenary session on March 13 and passed a committee proposal that merges amendments to the Advanced Regenerative Medicine and Advanced Biopharmaceuticals Act. Some provisions of the bill, which was sponsored by Kim Youngbae, a member of the Democratic Party belonging to the Welfare Committee, were incorporated. These include expanding the definition of human cells to include genetic material, thereby bringing in-vivo gene therapy into the institutional framework. Once the bill passes the National Assembly’s Legislation and Judiciary Committee and the plenary session, completing the legislative process, in-vivo-based advanced regenerative medicine clinical research and therapies will also become possible in Korea.

As discussions on revising the law are making the institutionalization of in-vivo therapies more concrete, the research and development environment is also expected to improve. Industry experts expect that, as the regulatory vacuum that has so far limited in-vivo gene therapy research is filled, the race to develop next-generation therapies, including in-vivo CAR-T, will accelerate further. One industry official stated, "Alongside large-scale investments from global pharmaceutical companies, domestic firms will also become more proactive in securing technological competitiveness."

Expanding Global Investment... Domestic Biotech Companies Also Accelerate In-Vivo Technology Acquisition

Unlike the traditional ex-vivo approach, where a patient's immune cells are manipulated outside the body, in-vivo CAR-T technology involves directly injecting a genetic vector into the body to instantly reprogram immune cells. CAR-T is a cell therapy that equips a patient's immune cells (T cells) with additional genes to recognize and attack specific cancer cells. While conventional CAR-T therapy involves a complicated process of harvesting a patient's blood, cultivating it externally, and reinfusing it, the in-vivo approach allows for administration in the form of an injection, greatly reducing both treatment time and cost.

In-vivo technologies that simplify the treatment process and lower costs have become a key target for major pharmaceutical companies. Last year, AbbVie acquired Capsida Therapeutics for about $2.1 billion (approximately 3.2144 trillion won) to secure related platforms. Bristol Myers Squibb invested about $1.5 billion (approximately 2.2963 trillion won) in Orbital Therapeutics to obtain next-generation RNA-based technologies. Earlier this year, Eli Lilly acquired Orna Therapeutics for $2.4 billion (approximately 3.6741 trillion won), entering the in-vivo CAR-T race.

Korean biotech companies are also actively working to secure leading technologies. AbClon, a company specializing in antibody therapeutics, is seeking to enter the in-vivo CAR-T field based on its experience in developing CAR-T therapies. AbClon currently owns 'AT101,' a CAR-T therapy that is in Phase 2 clinical trials.

According to the company, while conventional CAR-T therapies are tied to patent issues concerning a specific antibody (FMC63), AbClon possesses an independent antibody-based CAR-T, which can reduce commercialization risks during the transition to in-vivo approaches. An AbClon official stated, "As securing CAR-T therapies is a prerequisite for in-vivo CAR-T, owning CAR-T therapies with established clinical data gives us a competitive edge."

Genetic medicine specialist company Argynomics has also achieved significant results in developing core technologies for in-vivo CAR-T. Recently, through joint research with the Inha University research team, they have confirmed the potential for genetic material injected into the body to be effectively delivered to various immune cells.

The research revealed that genetic material can be easily delivered to organs where immune cells are particularly concentrated. The finding that genetic material can be conveniently delivered to organs with abundant immune cells is considered a key factor in realizing in-vivo CAR-T. An Argynomics official stated, "Through research combining Argynomics’ circular RNA and Inha University's polymeric delivery system, we plan to expand the application to in-vivo gene therapy, including CAR-T, going forward."

This content was produced with the assistance of AI translation services.