Product Description
AVR-RD-02 is an investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 could slow, halt or potentially reverse symptoms throughout the entire body and brain, such as GBA-related Parkinson’s disease which occurs more frequently in people with Gaucher disease type 1. (Sourced from: https://investors.avrobio.com/news-releases/news-release-details/avrobio-announces-clinical-and-regulatory-progress-gaucher)
Mechanisms of Action: Gene Therapy,CD34
Novel Mechanism: No
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: Orphan Drug - Gaucher Disease *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: AvroBio
Company Location: CAMBRIDGE MA 02139
Company CEO: Geoff MacKay
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: Gaucher Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| NCT06488261 | N/A |
Active, not recruiting |
Gaucher Disease |
2037-06-01 |
2024-07-07 |
Primary Endpoints|Treatments |
|
| AVRO-RD-02-201 | P2 |
Terminated |
Gaucher Disease |
2023-08-21 |
40% |
2024-01-19 |
Primary Endpoints |
| Guard3 | P3 |
Withdrawn |
Gaucher Disease |
2027-12-01 |
4% |
2023-08-10 |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
05/11/2023 |
News Article |
AVROBIO Reports First Quarter 2023 Financial Results and Provides Business Update |
|
03/23/2023 |
News Article |
AVROBIO Reports Fourth Quarter and Fiscal Year 2022 Financial Results and Provides Business Update |
|
03/03/2023 |
News Article |
AVROBIO to Participate in Cowen's 43rd Annual Health Care Conference |
|
02/09/2023 |
News Article |
AVROBIO to Present Clinical and Preclinical Data from Lysosomal Disorder Gene Therapy Pipeline at WORLDSymposium™ 2023 |