Drugs / AT-GTX-501
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02725580 | May 2016 → Oct 2022 | late infantile neuronal ceroid lipofuscinosis 6 | Emily de los Reyes | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 3
| Date | Issuer | Release |
|---|---|---|
| 2022-10-14 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Results CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress canbridgepharma.com ↗
Endogenous human SMN1 promoter-driven gene replacement improves the efficacy and safety of AAV9-mediated gene therapy for spinal muscular atrophy (SMA) in mice |
| 2022-05-17 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Results CANbridge-UMass Chan Medical School Gene Therapy Research Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting First Study from CANbridge UMass Collaboration Shows Novel Gene Therapy Holds Promise for Spinal Muscular Atrophy Clinical Applications canbridgepharma.com ↗
The novel second-generation gene therapy showed superior efficacy to the benchmark vector in SMA mice along several endpoints, including extended lifespan, restored muscle function and better neuromuscular junction innervation, without the liver toxicity shown in the benchmark-treated animals. |
| 2016-10-07 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Publication of Preclinical Data Supporting Clinical Translation of Juvenile Batten Disease Gene Therapy abeonatherapeutics.com ↗
Preclinical efficacy data demonstrated corrected motor deficits, attenuated neuroinflammation and reduced lysosomal throughout the central nervous system (CNS) and peripheral organs |
All press releases naming this drug 3 releases
| Date | Issuer | Release |
|---|---|---|
| 2022-10-14 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress canbridgepharma.com ↗ |
| 2022-05-17 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | CANbridge-UMass Chan Medical School Gene Therapy Research Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting First Study from CANbridge UMass Collaboration Shows Novel Gene Therapy Holds Promise for Spinal Muscular Atrophy Clinical Applications canbridgepharma.com ↗ |
| 2016-10-07 | Abeona Therapeutics, Inc | Abeona Therapeutics Announces Publication of Preclinical Data Supporting Clinical Translation of Juvenile Batten Disease Gene Therapy abeonatherapeutics.com ↗ |
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AT-GTX-501 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02725580 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “CLN6 Gene delivered by Self-Complementary AAV9” NCT02725580 ↗ |
| Route | Intrathecal | “Participants with diagnosis of vLINCL6 Batten disease will receive a single intrathecal injection of AT-GTX-501 into the lumbar spinal cord region.” NCT02725580 ↗ |