Drugs / ABO-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06839235 | Jun 2025 → Mar 2029 expected | primary hyperoxaluria type 1 | Arbor Biotechnologies | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2026-06-25 | Arbor Pharmaceuticals, Inc. | Regulatory Chiesi Group and Arbor Biotechnologies Announce ABO-101 Granted Orphan Drug Designation by European Commission for Primary Hyperoxaluria arbor.bio ↗
The European Commission (EC) has granted ODD to ABO-101 for the treatment of PH, a group of rare genetic disorders characterized by excessive oxalate production. |
| 2025-05-13 | Arbor Pharmaceuticals, Inc. | Results Arbor Biotechnologies to Present Preclinical Data for ABO-101 in PH1 at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting arbor.bio ↗
Preclinical data demonstrate highly specific and durable editing of HAO1 with long-lasting reduction of urinary oxalate levels out to one-year post-single dose administration of ABO-101 |
| 2025-02-04 | Arbor Pharmaceuticals, Inc. | Regulatory Arbor Biotechnologies Announces FDA Orphan Drug and Rare Pediatric Disease Designations Granted to ABO-101 for the Treatment of Primary Hyperoxaluria Type 1 (PH1) and Upcoming Presentations at the 20th Congress of the International Pediatric Nephrology Association (IPNA) arbor.bio ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to ABO-101 for the treatment of primary hyperoxaluria type 1 (PH1). |
| 2024-12-19 | Arbor Pharmaceuticals, Inc. | Regulatory Arbor Biotechnologies Announces FDA Acceptance of IND Application for ABO-101 for the Treatment of Primary Hyperoxaluria Type 1 arbor.bio ↗
Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). |
| 2024-05-09 | Arbor Pharmaceuticals, Inc. | Results Arbor Biotechnologies Presents Data Supporting Clinical Development of ABO-101 and Robust Potential of Platform to Enable Therapeutic Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting arbor.bio ↗
Presented data which, along with ongoing IND-enabling studies, demonstrate efficacy and pharmacology of ABO-101 in non-human primates (NHPs), supporting continued development as a potential novel gene editing therapy for patients with primary hyperoxaluria type 1 (PH1) |
| 2021-02-12 | Abeona Therapeutics, Inc | Results New Positive Phase 1/2 Interim Data Presented at WORLDSymposium™ Shows Neurocognitive Development of Young MPS IIIA Patients Preserved up to Three Years Following Treatment with Abeona’s ABO-102 Gene Therapy abeonatherapeutics.com ↗ |
| 2020-02-12 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Positive Interim Data from MPS III Gene Therapy Programs Presented at WORLDSymposium™ abeonatherapeutics.com ↗
Results from the Transpher B study showed that ABO-101 also improved multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB. |
| 2019-04-04 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB) abeonatherapeutics.com ↗
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy development, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ABO-101, the Company’s novel one-time gene therapy for Sanfilippo syndrome type B (MPS IIIB). |
| 2018-09-12 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces Authorization to Commence Phase 1/2 Gene Therapy Clinical Study for Patients with MPS IIIB in Spain abeonatherapeutics.com ↗
The clinical study was approved by the Agencia Espanola de Medicamentos y Productos Sanitarios and is being conducted at Hospital Clinico Universitario of Santiago de Compostela, Spain. |
| 2018-02-07 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Reports on Initial Safety and Biopotency Signals in MPS IIIB Gene Therapy Clinical Trial abeonatherapeutics.com ↗
The first patient treated with ABO-101 has demonstrated that the systemic AAV gene transfer is well-tolerated, and the preliminary evidence of biopotency in the CNS and periphery is very encouraging, |
| 2017-01-19 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B abeonatherapeutics.com ↗
the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation (EMA/OD/226/16) for Abeona’s gene therapy program ABO-101 |
| 2016-05-24 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces FDA Allowance of Investigational New Drug (IND) for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB) abeonatherapeutics.com ↗ |
| 2016-01-11 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces Initial European Regulatory Approvals for Phase 1/2 Gene Therapy Clinical Studies for Patients With Sanfilippo Syndromes Type A (MPS IIIA) and Type B (MPS IIIB) abeonatherapeutics.com ↗
the Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) |
All press releases naming this drug 17 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ABO-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06839235 ↗ |
| Route | Intravenous | “Intravenous (IV) infusion” NCT06839235 ↗ |