-
Product Information
Scientific name: KL003
Brand name: Kanexthal
RESPONSIBLE: Kanglin Biotechnology -
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 countryAnticipated 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
Status: Not Authorised
Additional notable points:
- EMA: N/A
- FDA: N/A
- MHRA: N/A
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
