In the field of cell and gene therapy (CGT), while innovation and exploration are constantly advancing, there are also many challenges, and some experiments are terminated for various reasons. Since January 2025, some experiments in this field have come to an end. The following is a detailed inventory for you.

1. Cargo Therapeutics terminated the second phase study of firi-cel for large B-cell lymphoma.

On January 29th, 2025, Cargo Therapeutics announced that it would terminate the second phase of its experimental CAR-T therapy firi-cel for patients with large B-cell lymphoma (LBCL).

Firi-cel is a CAR-T therapy targeting CD22 protein on B cells. In the FIRCE-1 study, a total of 51 patients were included. Although the overall remission rate and complete remission rate were 77% and 43% respectively, the treatment response was not sustained, and the duration of complete remission was only 18% at three months. In addition, 18% patients had toxic reactions related to CAR-T therapy, that is, immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome, at least one of which led to death. Because the benefit-risk ratio of this therapy is not competitive for patients, Cargo decided to terminate the study.

Affected by this news, Cargo's stock fell by more than 70%. At the same time, Cargo announced that it will implement about 50% layoff plan to prolong the use cycle of funds, and give priority to its tri-specificity CAR-T therapy CRG-023 to the first phase of proof-of-concept data. The therapy targets CD19/CD20/CD22, and the first phase of dose escalation study will be started as planned in the second quarter of 2025. The company is also developing an allogeneic project that is still in the preclinical research stage.

2. AlloVir terminated three phase 3 clinical trials of the core product Posoleucel.

In early January, AlloVir announced the termination of three phase 3 clinical trials of the core product Posoleucel. Posoleucel is an experimental multi-virus specific T-cell therapy. The three clinical trials stopped by AlloVir are related to the prevention of infections or diseases caused by multiple viruses, the treatment of virus-related hemorrhagic cystitis (vHC) and the treatment of adenovirus (AdV).

3. Pfizer terminated its cooperation with Sangamo in gene therapy for hemophilia A.

On December 30th, Pfizer announced the termination of its cooperation with Sangamo Therapeutics in the gene therapy of hemophilia A (Giro-VEC). Although the results of Phase III trial of this therapy are positive and will be submitted to the regulatory authorities soon, Pfizer's overall analysis shows that patients' interest in additional gene therapy options is very limited at present. The cooperation will be terminated on April 21, 2025, just before the gene therapy was submitted in the American and European markets, and it was originally planned to be carried out in early 2025.

The CEO of Sangamo expressed surprise and great disappointment at Pfizer's termination of cooperation on the eve of submission. AFFINE test has shown that giro-vec significantly reduces the annualized total bleeding rate in adults with moderate to severe hemophilia A compared with conventional factor VIII preventive treatment.

4. Roche terminated the clinical development of spk-3006 gene therapy.

The experimental gene therapy spk-3006 for liver targeting AAV developed by Spark Therapeutics, a subsidiary of Roche, is used to treat Pompeii disease. In the pipeline combing of Roche in the first half of 2024, it was decided to eliminate the project and stop its clinical development. The decision to stop the test was not due to security issues, but was made as part of the priority of Spark's internal strategic portfolio.

5. Turnstone Biology terminated the development of TIDAL-01.

In early February, Turnstone Biologics announced the termination of its only clinical stage TIDAL-01 pipeline development. Biotech, a star who had raised more than $220 million and transformed twice, finally fell on the eve of TIL. Turnstone was originally famous for its oncolytic virus technology, but later turned to TIL therapy because of poor progress. The phase I data of TIDAL-01 in the treatment of colorectal cancer showed some positive effects. For example, among the 4 evaluable patients, the overall remission rate (ORR) was 25% and the disease control rate (DCR) was 50%, and one patient showed a lasting complete remission (CR). However, because the production of Selected TIL needs continuous investment in process improvement, the company decided to stop development after considering the capital demand and the current financial market.

The termination of these trials reflects that the CGT field is facing many complex challenges in the process of research and development, such as efficacy, safety, capital and market demand. Despite setbacks, with the progress and innovation of technology, it is expected that more successful therapies will bring hope to patients in the future.

References: public reports

Disclaimer: This article is only for the purpose of knowledge exchange and sharing and popular science, and does not involve commercial propaganda, and is not used as relevant medical guidance or medication advice. If the article is infringing, please contact to delete it.

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