ET-01 / EdiGene - LARVOL DELTA

A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia. (clinicaltrials.gov) - P=N/A | N=2 | Terminated | Sponsor: EdiGene (GuangZhou) Inc. | Trial completion date: Aug 2025 ➔ Mar 2025 | Active, not recruiting ➔ Terminated; Development plan change

Trial completion date • Trial termination • Beta-Thalassemia • Genetic Disorders • Transplantation

A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia (clinicaltrials.gov) - P1 | N=8 | Terminated | Sponsor: EdiGene (GuangZhou) Inc. | Trial completion date: Jun 2024 ➔ Mar 2025 | Active, not recruiting ➔ Terminated; Development plan change

Trial completion date • Trial termination • Beta-Thalassemia

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia (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 • Transplantation

A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia. (clinicaltrials.gov) - P=N/A | N=2 | Active, not recruiting | Sponsor: EdiGene (GuangZhou) Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Beta-Thalassemia • Genetic Disorders • Transplantation

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P=N/A | N=6 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital | Active, not recruiting ➔ Recruiting | Trial completion date: Oct 2023 ➔ Aug 2025 | Trial primary completion date: Apr 2023 ➔ Jun 2025

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

A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia. (clinicaltrials.gov) - P=N/A | N=3 | Recruiting | Sponsor: EdiGene (GuangZhou) Inc.

New trial • Beta-Thalassemia • Genetic Disorders • Transplantation

A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P1 | N=8 | Active, not recruiting | Sponsor: EdiGene (GuangZhou) Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Beta-Thalassemia

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P=N/A; N=1; Active, not recruiting; Sponsor: Institute of Hematology & Blood Diseases Hospital; Trial primary completion date: Oct 2021 ➔ Apr 2023

Clinical • Trial primary completion date • Beta-Thalassemia • Genetic Disorders • Transplantation

A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P1; N=8; Recruiting; Sponsor: EdiGene (GuangZhou) Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Beta-Thalassemia

A Safety and Efficacy Study Evaluating ET-01 in Participants With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P1; N=8; Not yet recruiting; Sponsor: EdiGene (GuangZhou) Inc.

New P1 trial • Beta-Thalassemia

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P=N/A; N=1; Active, not recruiting; Sponsor: Institute of Hematology & Blood Diseases Hospital; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Beta-Thalassemia • Genetic Disorders • Transplantation

EdiGene Announces Approval of its IND Application for its CRISPR/Cas 9 Gene-editing Hematopoietic Stem Cell Therapy ET-01 in β-thalassemia by China National Medical Products Administration (Businesswire) - "EdiGene, Inc...announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has approved the Company’s Investigational New Drug (IND) application for ET-01...for patients with transfusion dependent β-thalassemia...'We are delighted that our IND application for ET-01 is approved by the regulatory authority and we’ll roll out phase I clinical trial soon'..."

New P1 trial • Non-US regulatory • Beta-Thalassemia

EdiGene Announces IND Application for its CRISPR/Cas 9 Gene-editing Hematopoietic Stem Cell Therapy ET-01 in β-thalassemia Accepted for Review by China National Medical Products Administration (Businesswire) - "EdiGene...announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has accepted for review the Company’s Investigational New Drug (IND) application for ET-01, autologous CD34+ hematopoietic stem/progenitor cells with the erythroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9, an investigational gene-editing therapy for patients with transfusion dependent β-thalassemia...'We look forward to receiving approval from NMPA and initiating ET-01 clinical studies in the near future'."

Non-US regulatory • Beta-Thalassemia • Hematological Disorders

EdiGene Gets $67 Million Infusion to Bring Gene Editing to the Clinic (BioSpace) - "EdiGene raked in approximately $67 million in a series B financing round to further their genome-editing pipeline and expand the team....Right now, its fastest progressing projects are ET-01 for severe β-thalassemia, a hereditary blood disease, and U Car-T ET-02 for cancer. CEO Dong Wei said this round will help them transform their pipeline into clinical stage, drawing them closer to their goal of bringing innovative and high-quality gene-editing therapies to patients in need."

Financing • Beta-Thalassemia

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia (clinicaltrials.gov) - P=N/A; N=3; Recruiting; Sponsor: Institute of Hematology & Blood Diseases Hospital; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Gene Therapies • Genetic Disorders • Transplantation

Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34+ Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major (ASH 2019) - "In vitro erythroid differentiation efficiency, HbF+ cells and γ-globin mRNA levels were similar to those observed at research scale. IND-enabling preclinical studies, including GLP studies using ET-01 manufactured at the clinical scale, are ongoing."

CD34