Sinobiopharma Initiates First-in-Human Clinical Trial of Innovative In Vivo CAR-T Therapy SNC116 with Successful Enrollment of Initial Patient

Recently, Simnova, a company focused on developing innovative immunocyte therapies, announced that its self-developed CD19-targeted in vivo CAR-T product (SNC116) has officially launched clinical research at the Cancer Institute and Hospital, Chinese Academy of Medical Sciences, and successfully completed the enrollment of the first patient.

Unlike traditional CAR-T therapies that require complex and time-consuming ex vivo preparation processes, Simnova's in vivo technology platform delivers a viral vector carrying the CAR gene in a single infusion, directly "arming" T cells within the patient’s body to express chimeric antigen receptors targeting CD19. This innovative approach significantly simplifies the treatment process and is expected to drive CAR-T therapy from the era of "personalized customization" into a new age of "standardized medication."

Simnova, with its proprietary viral vector platform, has launched a new generation of in vivo CAR-T therapy. This technology employs a T-cell-specific promoter and transfection system, akin to "precision guidance," ensuring efficient and specific expression of the CAR gene in vivo. The next-generation dual-target design aims to enhance the clearance of target cells, pursuing deeper therapeutic effects. Meanwhile, the successful advancement of this therapy is attributed to the company's long-term technical expertise in lentiviral modification and CMC, securing a significant first-mover advantage in process development and scalable production.

Dr. Cao Zhuoxiao, CEO of Simnova, stated: "We are very proud to collaborate with the top team at the Cancer Hospital of the Chinese Academy of Medical Sciences, jointly opening a new clinical chapter for in vivo CAR-T therapy. The successful enrollment of the first patient is a key milestone in advancing this disruptive technology from concept to clinical validation."

The principal investigator (PI) of this study, Dean Li Ning from the Cancer Hospital of the Chinese Academy of Medical Sciences, pointed out: "The in vivo CAR-T technology represents the future direction of development, and it is expected to address the core bottlenecks of accessibility and timeliness in existing CAR-T therapies. The successful enrollment of the first patient is a good start, and we will advance this research with a rigorous scientific attitude."

In addition to its self-developed lentiviral vector-based in vivo CAR-T (SNC116), Simnova is also actively enhancing its technical capabilities and pipeline breadth through international collaborations:

• In January 2023, Simnova reached a cooperation agreement with Orna Therapeutics: Simnova will be responsible for the development and commercialization in China (including Hong Kong, Macao, and Taiwan) of Orna's in vivo cell therapy products developed using its breakthrough circular mRNA technology (oRNA) for the field of oncology. This includes Orna’s most advanced project from its pipeline, the anti-CD19 in situ chimeric antigen receptor T-cell technology (isCAR), named “ORN-101.”

  
  

• In January 2025, to fully leverage the leading advantages of both companies in circular RNA (oRNA®) technology and cell therapy, the two enterprises announced a further expansion of their strategic cooperation, officially designating BCMA (B-cell maturation antigen) as the specified biological target to jointly advance the development and application of in vivo CAR-T therapy. According to the agreement, Simnova will be responsible for the research, development, and commercialization of panCar cell therapy targeting BCMA within China (including Hong Kong, Macao, and Taiwan), while Orna will retain all rights to research, development, and commercialization in the rest of the world.

In recent years, in vivo CAR-T has become a "must-contend territory" in the biopharmaceutical industry because it can fundamentally address the pain points of traditional CAR-T.

Exploration involves many players, but a consensus is gradually forming: safety is the cornerstone of research and development. It is essential to systematically evaluate off-target risks, immunogenicity, and process impurities, and to explore appropriate dosages through rigorous dose escalation. In the preclinical stage, solid data must be used to clarify the product's strengths and weaknesses; the core task in the clinical stage is to verify pre-set hypotheses rather than engage in blind trial and error.

Although different delivery vector pathways still present their own challenges, all must be upgraded from "production tools" to end products that meet pharmaceutical standards. For example, non-viral vectors represented by LNPs are easy to scale up and can be optimized through strategies such as formula optimization and the application of circular RNA. Circular RNA, due to its high stability, simplified production process, and long-lasting in vivo expression, is considered a promising pathway, but further breakthroughs are needed in purity control and expression peak.