Drugs / FBX-101
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05739643 | Feb 2023 → Nov 2026 expected | Krabbe disease | Forge Biologics, Inc | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT04693598 | Nov 2021 → Nov 2026 expected | Krabbe disease | Forge Biologics, Inc | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 11
| Date | Issuer | Release |
|---|---|---|
| 2024-04-30 | Forge Biologics, Inc | Results Forge to Present at ASGCT's 27th Annual Meeting forgebiologics.com ↗
We are also especially encouraged by a late-breaking oral presentation that our Chief Medical Officer, Dr. Maria Escolar, will be delivering that provides a clinical update on FBX-101 where all five patients with Krabbe disease receiving Forge’s gene therapy are walking—a remarkable result. |
| 2024-03-19 | Forge Biologics, Inc | Regulatory Novel Gene Therapy is Granted UK’s Innovation Passport Designation forgebiologics.com ↗
Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation |
| 2024-01-29 | Forge Biologics, Inc | Results Forge Provides Clinical Updates and Advocates for Newborn Screening Addition to the RUSP forgebiologics.com ↗
Forge Biologics (Forge), a member of Ajinomoto Bio-Pharma Services and a leading manufacturer of genetic medicines, announced a clinical update today on five patients with Krabbe disease that have received FBX-101, an AAV gene therapy, after hematopoietic stem cell transplantation (HSCT). |
| 2023-04-24 | Forge Biologics, Inc | Regulatory Forge Receives Qualified Person (QP) Declaration to Support European Clinical Programs forgebiologics.com ↗
The QP declaration was received in support of Forge’s internal FBX-101 clinical gene therapy program for the treatment of patients with Krabbe disease. |
| 2023-02-23 | Forge Biologics, Inc | Results Forge Reports Positive Clinical Updates for Patients with Krabbe Disease at WORLDSymposium forgebiologics.com ↗
Data from the RESKUE Phase 1/2 clinical trial continues to demonstrate safety and efficacy post-FBX-101 systemic administration in patients recently receiving hematopoietic stem cell transplantation (HSCT), including normalization of motor function and brain development |
| 2023-01-17 | Forge Biologics, Inc | Regulatory Forge Receives Priority Medicines (PRIME) Designation in Europe forgebiologics.com ↗
Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that the European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge’s lead adeno-associated virus (AAV) drug candidate and novel gene therapy for treating patients with Krabbe disease. |
| 2022-10-11 | Forge Biologics, Inc | Results Forge to Present Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial forgebiologics.com ↗
Subjects treated with FBX-101 have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid (CSF), normal white matter myelination and normalization of motor development in two children 90 days and 9 months post-treatment |
| 2022-08-30 | Forge Biologics, Inc | Results RESKUE Trial Shows Positive Clinical Data on Brain Development and Motor Function forgebiologics.com ↗
FBX-101 significantly increased GALC enzyme activity in leukocytes and patient exhibited improved motor function and normal brain development |
| 2021-09-20 | Forge Biologics, Inc | Regulatory Forge Announces Regulatory Updates from FDA and EMA, Accelerating Manufacturing and Clinical Trial Momentum forgebiologics.com ↗
The EMA has granted orphan designation for the company’s clinical-stage gene therapy FBX-101 for the treatment of patients with Krabbe disease. |
| 2021-02-16 | Forge Biologics, Inc | Regulatory FDA Fast Track, ODD, and RPDD Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease forgebiologics.com ↗ |
| 2021-01-04 | Forge Biologics, Inc | Regulatory FDA Clearance of Investigational New Drug for Patients with Krabbe Disease forgebiologics.com ↗
Forge Biologics Inc., a gene therapy manufacturing and development company, today announced that the company has received FDA clearance of the Investigational New Drug (IND) to initiate a Phase 1/2 clinical trial evaluating its novel, first-in-human AAV gene therapy, FBX-101, for patients with Krabbe disease. |
All press releases naming this drug 11 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | FBX-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04693598 ↗ |
| Known as | AAVrh.10-GALC | “Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)” NCT04693598 ↗1“Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)” NCT05739643 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC” NCT04693598 ↗ |
| Route | Intravenous | “intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT)” NCT04693598 ↗ |