Reforgene Medicine
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Therapeutic Areas

Development Pipeline

We focus on serious diseases that have no cure or still lack truly effective medicines.

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A continuously expanding pipeline

Despite the great achievements in drug development, a large number of patients worldwide still cannot receive effective treatment.

We focus on serious diseases that have no cure or still lack truly effective medicines, including genetic diseases that have long been neglected and major diseases with high prevalence.

Unlike traditional drug technologies, we seek solutions at the most fundamental level of life, the genome, breaking through existing boundaries. Innovative gene and cell therapies will bring patients the hope of returning to health.

Gene editing technology is driving a paradigm shift in drug innovation. We are committed to drug development in genetic diseases and complex diseases.

Our pipeline currently includes gene editing medicines across indications including hematology, cardiovascular and metabolic diseases, infectious diseases and ophthalmology.

Thanks to our powerful TIPBAIG™ digital drug development platform, we are achieving continuous product output, with two gene editing medicine modalities today: ex vivo gene editing and in vivo gene editing. We will update the progress of selected pipeline programs on a regular basis.

Gene editing · Hematology

We operate two gene editing medicine modalities: in vivo and ex vivo.

Ex VivoEx vivo gene editing

Engineers autologous or allogeneic cells before reinfusion into patients.

In VivoIn vivo gene editing

Delivers editing tools or other therapeutic payloads into the body via LNP or AAV.

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Program
Indications
Discovery Preclinical Stage
IND-Enabling
Phase I
Phase II
Phase III*
Approved
Hemoglobinopathies (Ex Vivo)
RM001
Transfusion-Dependent β-Thalassemia
Sickle Cell Disease

Transfusion-Dependent β-Thalassemia

RM001 is an investigational autologous CD34+ hematopoietic stem and progenitor cell (HSPC) therapy for transfusion-dependent β-thalassemia. The cells are edited ex vivo using CRISPR/Cas9 at the HBG1/2 promoters to increase γ-globin expression and fetal hemoglobin (HbF) production.

Sickle Cell Disease

RM001 is an investigational autologous CD34+ hematopoietic stem and progenitor cell (HSPC) therapy for sickle cell disease (SCD). The cells are edited ex vivo using CRISPR/Cas9 at the HBG1/2 promoters to increase γ-globin expression and fetal hemoglobin (HbF) production.

  • EHA 2026 (European Hematology Association) Annual Meeting: DURABLE CLINICAL BENEFITS WITH RM001 (AUTOLOGOUS HBG1/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS) FOR TRANSFUSION-DEPENDENT B-THALASSEMIA Link ↗
  • ASH 2025 (American Society of Hematology) Annual Meeting: All 19 subjects with transfusion-dependent β-thalassemia achieved transfusion independence after treatment with RM001 (autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells) Link ↗
  • EHA 2025 Annual Meeting: Updated Safety and Efficacy Results of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia Link ↗
  • ASH 2024 Annual Meeting: Updated Safety and Efficacy Results of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia Link ↗
  • EHA 2024 Annual Meeting: Safety and Efficacy of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Transfusion-Dependent β-Thalassemia Link ↗
  • Reforgene reported Phase I clinical progress of its thalassemia gene editing medicine RM001, helping open the era of gene editing medicines in China Link ↗
  • ASH 2023 Annual Meeting: Safety and Efficacy of RM001 (Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells) in Patients with Transfusion-Dependent β-Thalassemia Link ↗
  • EHA 2023 Annual Meeting: Safety and Efficacy of RM001 in Patients with Transfusion-Dependent β-Thalassemia: Early Results from the Ongoing Study of Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells Link ↗
  • ASH 2022 Annual Meeting: Preliminary Result of the Safety and Efficacy of Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells (RM001) in Transfusion-Dependent β-Thalassemia Link ↗
  • Clinical research progress of Reforgene’s thalassemia gene editing medicine RM001 was presented at the 64th ASH Annual Meeting in 2022 Link ↗
  • Reforgene’s beta-thalassemia gene editing medicine received IND approval from the NMPA Link ↗
  • EHA 2022 Annual Meeting: Initial Safety and Efficacy Study of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Transfusion-Dependent β-Thalassemia Link ↗
  • Reforgene presented the world’s first novel-target gene editing thalassemia clinical results at the 2022 EHA Congress Link ↗
  • First breakthrough: China’s gene editing technology successfully treats adult patients with severe thalassemia Link ↗
  • The world’s first novel-target treatment for severe beta-thalassemia: Reforgene achieves a new breakthrough Link ↗
RM004
α-Thalassemia (HbH-CS)

α-Thalassemia (HbH-CS)

RM004 is an investigational autologous CD34+ HSPC therapy for transfusion-dependent hemoglobin H–Constant Spring (HbH-CS) disease, a form of α-thalassemia. It uses ex vivo gene editing designed to increase functional α-globin production.

* Not applicable to all programs

Gene editing · In vivo & ex vivo gene editing

Swipe horizontally to view the full table

Program
Indications
Discovery Preclinical Stage
IND-Enabling
Phase I
Phase II
Phase III*
Approved
Cardiovascular & Hepatic (In Vivo)
RM801
Undisclosed

RM801

RM801 is an investigational in vivo gene-editing therapy based on Reforgene's TitanCas12 platform.

RM803
Undisclosed

RM803

RM803 is an investigational in vivo gene-editing therapy based on Reforgene's TitanCas12 platform.

Other System (Ex Vivo)
RM601
Undisclosed

RM601

RM601 is an investigational gene-edited cell therapy that uses Reforgene's TitanCas12 platform to modify cells ex vivo.

Ophthalmology (In Vivo)
RM101
Usher Syndrome

Retinitis Pigmentosa in USH2A-Related Usher Syndrome

RM101 is an investigational adeno-associated virus (AAV)-based RNA editing therapy for retinitis pigmentosa associated with pathogenic USH2A exon 13 mutations in patients with Usher syndrome. It is designed to modulate USH2A RNA splicing to promote exon 13 skipping and restore functional usherin expression in retinal cells.

* Not applicable to all programs