Drugs / AAV2-GDNF
AAV2-GDNF
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT04680065 | Oct 2023 → Aug 2026 overdue | multiple system atrophy | Brain Neurotherapy Bio, Inc. | Active not recruiting | No outcome recorded |
| Phase 1 | NCT04167540 | Apr 2020 → Oct 2023 overdue | Parkinson disease | Brain Neurotherapy Bio, Inc. | Active not recruiting | No outcome recorded |
| Phase 1 | NCT01621581 | Mar 2013 → Feb 2022 | Parkinson disease | National Institute of Neurological Disorders and Stroke (NINDS) | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT06285643 | Jun 2024 → Aug 2028 expected | Parkinson disease | AskBio Inc | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2025-02-19 | Bayer | Regulatory AskBio Receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease investigational gene therapy bayer.com ↗
AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA). |
| 2025-02-19 | AskBio Inc | Regulatory AskBio Receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease investigational gene therapy askbio.com ↗
The FDA determined that AB-1005, an investigational gene therapy intended to slow disease progression and improve motor outcomes in patients with PD, met the criteria for RMAT designation. |
| 2024-07-11 | Bayer | Regulatory AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson’s disease bayer.com ↗
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Fast Track Designation for AB-1005, which is being developed for moderate Parkinson’s disease. |
| 2024-07-11 | AskBio Inc | Regulatory AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson’s disease askbio.com ↗
AB-1005 has also been awarded the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA) innovative medicine designation, for the treatment of Parkinson’s disease. |
| 2024-04-16 | Bayer | Results AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson’s disease bayer.com ↗
The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. |
| 2024-04-16 | AskBio Inc | Results AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson’s disease askbio.com ↗
The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. |
| 2024-01-04 | Bayer | Results AskBio Phase Ib trial of AB-1005 gene therapy in patients with Parkinson’s disease meets primary endpoint bayer.com ↗
No serious adverse events have been attributed to AB-1005, with continued clinical follow-up for up to 5 years post administration ongoing. |
| 2024-01-04 | AskBio Inc | Results AskBio Phase Ib trial of AB-1005 gene therapy in patients with Parkinson’s disease meets primary endpoint askbio.com ↗
The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. |
All press releases naming this drug 11 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAV2-GDNF | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01621581 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “adeno-associated virus, serotype 2 vector (AAV2) containing human GDNF complementary DNA” NCT01621581 ↗ |
| Route | Other | “the vector will be delivered by convection-enhanced delivery (CED) to both putamina” NCT01621581 ↗ |
| Target | GDNF | “adeno-associated virus, serotype 2 vector (AAV2) containing human GDNF complementary DNA” NCT01621581 ↗ |