Drugs / AB-1003
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05230459 | May 2023 → Dec 2028 expected | autosomal recessive limb-girdle muscular dystrophy type 2I | AskBio Inc | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2025-10-10 | Bayer | Results AskBio Presents Interim Safety Results from Phase 1/Phase 2 LION-CS101 Clinical Trial of AB-1003 in Participants with Limb-Girdle Muscular Dystrophy 2I/R9 bayer.com ↗
There were no dose-limiting toxicities or serious adverse events reported up to 52 weeks post-treatment. |
| 2025-10-10 | AskBio Inc | Results AskBio Presents Interim Safety Results from Phase 1/Phase 2 LION-CS101 Clinical Trial of AB-1003 in Participants with Limb-Girdle Muscular Dystrophy 2I/R9 askbio.com ↗ |
| 2024-11-07 | Bayer | Regulatory AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 bayer.com ↗
today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). |
| 2024-11-07 | AskBio Inc | Regulatory AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 askbio.com ↗
Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). |
| 2023-02-27 | Bayer | Regulatory AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular bayer.com ↗
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). |
| 2023-02-27 | AskBio Inc | Regulatory AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD) askbio.com ↗
Asklepios BioPharmaceutical , Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). |
All press releases naming this drug 12 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AB-1003 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05230459 ↗ |
| Known as | LION-101 | “A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)” NCT05230459 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “AB-1003 gene therapy” NCT05230459 ↗ |
| Route | Intravenous | “Single intravenous infusion of AB-1003 gene therapy at dose level 1” NCT05230459 ↗ |