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Kanexthal

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  • Product Information

    Product Information

    Scientific name: KL003
    Brand name: Kanexthal
    RESPONSIBLE: Kanglin Biotechnology

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: N/A
    Code: China: ChiCTR2500100369; USA: NCT06280378
    Phase: 1/2
    Eligible patient diagnosis: TDT patients, aged 3 – 35 years old
    No. of Patients enrolled: 41 [anticipated](Last update: 27/03/2026)
    Study Sites: 2 Sites per country

    Anticipated completion date: 2027
    Scope of the Study / Aim: Toevaluate the safety and efficacy of KL003 cell injection (autologous CD34+ stem cells transduced Ex Vivo with a lentiviral Vector encoding βA-T87Q-Globin)

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • EMA: N/A
    • FDA: N/A
    • MHRA: N/A
Update: 30 June 2026

Data presented at the EHA Annual Congress (11 – 14 June 2026) showed that:

– 44 TDT patients were treated with KL003 between 1 March 2025 – 31 December 2025 in a single centre in China. Median age was 14 years.
– Primary endpoints included transfusion independence defined as maintaining a haemoglobin ≥9g/dL for ≥6 months without RBC transfusions.
– 29/44 patients received busulfan-based conditioning; 15/44 received total marrow irradiation combined with fludarabine. All patients experienced grade ≥ 3 haematologic toxicity due to conditioning.
– Median follow-up (as of 28 Feb 2026 – time of submission to EHA) was 5.6 months.
– 43/44 were transfusion-independent; 15 patients had more than 6 months follow-up, and 100% were transfusion-independent for a median of 7.2 months.
– No unexpected adverse events, replication-competent lentivirus (RCL), insertional oncogenesis, clonal dominance, or new malignancies were detected.

Source: Gene-Modified Autologous Hematopoietic Stem Cell Therapy for Transfusion-Dependent Β-Thalassemia: A Real-World Study on Clinical Efficacy and Safety in 44 Chinese Patients

 

Update: 31 March 2026

Results from the first 17 treated patients in an investigator-initiated trial (IIT) were announced in 2024 (TDT/SCD Lentiviral Therapy Boasts Quick, Well-Tolerated Engraftment in Patients | CGTlive®):

    • 16 out of 17 patients achieved transfusion independence, with a median time of 18 days after treatment (17 out of 17 have maintained transfusion independence as of Dec 2025).
    • By the end of 2025, the longest reported duration of transfusion independence was 5 years.

The developer anticipates that in 2026, a pivotal Phase 2 trial in beta-thalassemia is expected to begin in China.

More information on the product and support programs can be found at:
Kanexthal: Treatment for Beta-Thalassemia | Kanglin Biotec

Source: Correspondence with the company

 

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