KL003 / Kanglin Biotech - LARVOL DELTA

Gene Therapy of Transfusion-Dependent β-Thalassemia Patients with Quick Engraftment of Reinfused Hematopoietic Stem Cells: An Investigator-Initiated Trial of KL003 (ASH 2023) - P=N/A | "The safety profile was consistent with that typical of busulfan-based myeloablation conditioning. This has led to significantly faster engraftment and better efficacy. Thus, KL003 could be a potential functional cure for β-thalassemia major patients."

Clinical • Gene therapy • Anemia • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders

Deciphering Hematopoietic Reconstitution Dynamics of Lentiviral Vector HBBT87Q-Engineered Hematopoietic Stem Cells Gene Therapy for β-Thalassemia Patients (ASH 2024) - P=N/A | "This study focuses on three β-TDT patients enrolled in the IIT clinical trial (NCT05860595), who received the autologous lentiviral vector gene therapy product KL003 carrying the codon-optimized HBBT87Q gene...This work showed for the first time that in vivo dynamics and lineage commitment of LVV-HBBT87Q HSPCs clusters in β-TDT, highlighting the enhanced fitness of LVV-HBBT87Q HSPCs to compensate for demands in specific clinical temporal. Future studies should leverage these mechanistic insights to further improve lentiviral gene therapy."

Clinical • Gene therapy • Viral vector • Beta-Thalassemia • Gene Therapies • Genetic Disorders • CD34 • GYPA • TFRC

Safety and efficacy study of KL003 cell injection in the treatment of transfusion dependent β - thalassemia pretreated with a novel myeloablative conditioning. (ChiCTR) - P=N/A | N=3 | Recruiting | Sponsor: Guabgzhou First People‘s Hospital; The First People's Hospital of Guangzhou

New trial • Genetic Disorders

Β-Thalassemia Treatment with KL003 Cell Injection (clinicaltrials.gov) - P1/2 | N=41 | Recruiting | Sponsor: Kanglin Biotechnology (Hangzhou) Co., Ltd. | Not yet recruiting ➔ Recruiting | Trial primary completion date: May 2025 ➔ Oct 2025

Enrollment open • Trial primary completion date • Beta-Thalassemia • Genetic Disorders

Kanglin Biotechnology Announces $20 Million Series A Financing to Advance the Development of Gene Therapies (GlobeNewswire) - "Kanglin Biotechnology (Hangzhou) Co., Ltd...today announced the successful completion of a Series A financing of $20 million. Proceeds from the financing will be used to support the clinical development for the company’s lead asset, KL003, for the treatment of beta thalassemia and sickle cell disease. The company will use additional funds from the financing to evaluate the feasibility of additional therapeutic indications in their pipeline...We believe that KL003’s clinical profile sets it apart from others in the class and look forward to beginning our Phase 2 pivotal study next year."

Financing • New P2 trial • Beta-Thalassemia • Hematological Disorders

A domestic gene therapy company completed a RMB 150 million Series A financing [Google translation] (bydrug.pharmcube.com) - "On November 8, Kanglin Biotech (Hangzhou) Co., Ltd...announced that the company has recently successfully completed its Series A financing. This round of financing was jointly invested by Zhejiang Province's '4+1' Biomedicine and High-end Equipment Industry Fund and old shareholder Zhejiang Miyin Investment Management Co., Ltd. , with a total financing amount of 150 million yuan...This round of financing will help accelerate the advancement of the company's various pipelines under development. One of the core products, KL003 cell injection..."

Financing • Beta-Thalassemia • Hematological Disorders

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. (clinicaltrials.gov) - P=N/A | N=3 | Active, not recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Recruiting ➔ Active, not recruiting

Enrollment closed • Beta-Thalassemia • Genetic Disorders • CD34

Clinical Efficacy and Safety of Gene Therapy for Transfusion-Dependent β-Thalassemia Patients with Quick Engraftment of Genetically Modified Hematopoietic Stem Cells (ASGCT 2024) - "The safety profile was consistent with that typical of busulfan-based myeloablation conditioning. The median engraftment time for 11 patients with <5,000 μg/L ferritin at enrollment was 13 days for neutrophils (range: 8-15 days) and 19 days (range: 13-28 days) for platelets, respectively. This is a significant improvement compared with much longer engraftment time (i.e. approximately 4 weeks for neutrophils and 6 weeks for platelets) previously reported for autologous HSC-based gene therapies."

Clinical • Gene therapy • Beta-Thalassemia • Gene Therapies • Genetic Disorders

A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants (clinicaltrials.gov) - P1/2 | N=41 | Not yet recruiting | Sponsor: Kanglin Biotechnology (Hangzhou) Co., Ltd.

New P1/2 trial • Beta-Thalassemia • Genetic Disorders

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients (clinicaltrials.gov) - P=N/A | N=3 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Gene therapy • New trial • Viral vector • Beta-Thalassemia • Gene Therapies • Genetic Disorders

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. (clinicaltrials.gov) - P=N/A | N=3 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Beta-Thalassemia • Genetic Disorders • CD34

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. (clinicaltrials.gov) - P=N/A | N=3 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital

New trial • Beta-Thalassemia • Genetic Disorders • CD34