Product Description
RJK002 is touted as the first clinically approved AAV gene therapy for ALS in China, and also the world's first AAV gene therapy targeting abnormal protein aggregation for ALS. The drug obtained orphan drug designation (ODD) in the US in October 2023, having demonstrated significant neuroprotective and survival prolonging therapeutic effects in preclinical animal models of ALS. It is subject to a multi-center investigator-initiated trial in China. (Sourced from: https://source.gbihealth.com.cn/news/detail?id=2051337&utm_source=official)
Mechanisms of Action: Gene Therapy,CGT
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: Injection
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Rejukon Biopharm Inc.
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count: 2
Highest Development Phases
Phase 1: Amyotrophic Lateral Sclerosis
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| RJK-002-I | P1 |
Not yet recruiting |
Amyotrophic Lateral Sclerosis |
2026-07-01 |
|
| RJK002-0001-R | P1 |
Not yet recruiting |
Amyotrophic Lateral Sclerosis |
2025-11-30 |