FBX-101 / Forge Biologics - LARVOL DELTA

RESKUE: Gene Transfer Clinical Trial for Krabbe Disease (clinicaltrials.gov) - P1/2 | N=6 | Active, not recruiting | Sponsor: Forge Biologics, Inc | Trial completion date: Jul 2024 ➔ Nov 2026 | Trial primary completion date: Jul 2024 ➔ Nov 2026

Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Gene Therapies • Transplantation

REKLAIM: Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT (clinicaltrials.gov) - P1/2 | N=9 | Active, not recruiting | Sponsor: Forge Biologics, Inc | Trial completion date: Jul 2026 ➔ Nov 2026 | Trial primary completion date: Jul 2026 ➔ Nov 2026 | Recruiting ➔ Active, not recruiting

Enrollment closed • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Gene Therapies • Transplantation

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients (clinicaltrials.gov) - P=N/A | N=25 | Active, not recruiting | Sponsor: Forge Biologics, Inc | Enrolling by invitation ➔ Active, not recruiting | Trial completion date: Dec 2029 ➔ Nov 2026 | Trial primary completion date: Dec 2029 ➔ Nov 2026

Enrollment closed • Trial completion date • Trial primary completion date • Gene Therapies

REKLAIM, a Phase I/II clinical trial using a novel immune modulation strategy for systemic administration of FBX-101 (AAVrh10.GALC) after Umbilical Cord Blood Transplantation for the treatment of Infantile Krabbe Disease (ESGCT 2024) - No abstract available

Clinical • P1/2 data • Bone Marrow Transplantation • Immune Modulation • Immunology • Transplantation

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients (clinicaltrials.gov) - P=N/A | N=25 | Enrolling by invitation | Sponsor: Forge Biologics, Inc | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Gene therapy • Gene Therapies

REKLAIM, a novel Phase Ib Clinical Trial of intravenous FBX-101 (AAVrh10.GALC) after UCBT for Infantile Krabbe Disease (EAN 2024) - "It includes a protocol using Rituximab, Serolimus and Prednisolone adjusted to subject needs. FBX-101 leverages the myeloablation and immune suppression after UCBT, resulting in efficient AAV transduction and increased GALC enzyme supporting brain myelination and gross motor development"

Clinical • P1 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • AVEN

REKLAIM ‐ A novel phase 1b gene therapy clinical trial using FBX‐101 (AAVrh10‐hGALC) administered intravenously to patients who received haematopoietic stem cell transplantation for the treatment of Krabbe Disease (ESGCT 2023) - "His ISR also consisted of Rituximab (two doses), Sirolimus and Prednisolone. The subject maintained normal liver function tests and no treatment-related serious adverse events have developed after 30 days of follow-up."

Clinical • Gene therapy • P1 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • Transplantation

REKLAIM, A Phase Ib Clinical Trial Using a Novel Immune Modulation Strategy for Systemic Administration of FBX-101 (AAVrh10.GALC) After Umbilical Cord Blood Transplantation for the Treatment of Infantile Krabbe Disease (ASGCT 2024) - "The protocol includes adjustments with Rituximab, Sirolimus and Prednisolone according to the subject needs. FBX-101 in 5 patients receiving the low dose was well tolerated, with no treatment-related serious adverse events and follow up ranging from 6 to 24 months. FBX-101 in 5 patients receiving the low dose was well tolerated, with no treatment-related serious adverse events and follow up ranging from 6 to 24 months. No antibodies to the transgene developed. In the two subjects treated during myeloablation, there were no antibodies to AAV, plasma and CSF GALC significantly increased, psychosine dropped below the level of detection and subjects achieved normal white matter growth, peripheral nerve conduction velocity and gross motor skills."

Clinical • Late-breaking abstract • P1 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Graft versus Host Disease • Immune Modulation • Immunology • Pain • Transplantation • AVEN

Industry Platforms (ACMG 2024) - "It is a 13-minute presentation followed by a 2-minute Q&A. O34 - Utility of Targeted RNA Analysis in Neurological Disorders O31 - The Landscape of Variant Reclassification: Learnings from over 2 Million Classified Variants in over 3.6 Million Individuals O33- Comprehensive identification of gene-disease relationships across the clinical exome through systematic literature review and parallelized evidence curation O32 - Ensemble Prediction of the Impact of Missense Variants Substantially Decreases VUS Rate in Genetic Testing O30- REKLAIM, a First in Human Phase Ib Clinical Trial of FBX-101 (AAVrh10.GALC) Intravenously administered after UCBT for Infantile Krabbe Disease"

CNS Disorders

REKLAIM, a First in Human Phase Ib Clinical Trial of FBX-101 (AAVrh10.GALC) Intravenously administered after UCBT for Infantile Krabbe Disease (ACMG 2024) - "In summary, FBX-101 after UCBT leverages the myeloablation and immune-suppression after UCBT, resulting in efficient AAV transduction and providing supraphysiological GALC enzyme to support brain myelination and gross motor development. The absence of antibody response in the myeloablated group suggests that redosing once would be possible in the future."

Clinical • P1 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • Transplantation • AVEN

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients (clinicaltrials.gov) - P=N/A | N=25 | Not yet recruiting | Sponsor: Forge Biologics, Inc

Gene therapy • New trial • Gene Therapies

REKLAIM, A NOVEL PHASE IB CLINICAL TRIAL OF FBX-101 (AAVRH10.GALC) INTRAVENOUSLY ADMINISTERED AFTER UCBT FOR THE TREATMENT OF INFANTILE AND LATE INFANTILE KRABBE DISEASE (EBMT 2024) - P1/2 | "A Rituximab, Serolimus and Prednisolone based regime is used to adjust immune suppression. In summary, FBX-101 after UCBT leverages the myeloablation and immune suppression after UCBT, resulting in efficient AAV transduction providing increased GALC enzyme that supports normal brain myelination and gross motor development"

Clinical • P1 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • Transplantation • AVEN

RESKUE: Gene Transfer Clinical Trial for Krabbe Disease (clinicaltrials.gov) - P1/2 | N=6 | Active, not recruiting | Sponsor: Forge Biologics, Inc | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2024 ➔ Jul 2024 | Trial primary completion date: Dec 2024 ➔ Jul 2024

Enrollment closed • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Gene Therapies • Transplantation

REKLAIM: Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT (clinicaltrials.gov) - P1/2 | N=9 | Recruiting | Sponsor: Forge Biologics, Inc | Phase classification: P1b ➔ P1/2 | Trial completion date: Dec 2025 ➔ Jul 2026 | Trial primary completion date: Dec 2025 ➔ Jul 2026

Phase classification • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Gene Therapies • Transplantation

RESKUE, a First in Human Phase I/II Clinical Trial of FBX-101 (AAVrh10.GALC) Intravenously administered after UCBT for the treatment of Patients with Infantile Krabbe Disease (ESGCT 2023) - No abstract available

Clinical • P1/2 data

A novel approach for the treatment of inborn errors of metabolism that benefit from HSCT: development of FBX 101 for the treatment of Krabbe disease (ESGCT 2023) - No abstract available

Bone Marrow Transplantation • Metabolic Disorders

Updates on the FBX-101 REKLAIM Phase Ib Gene Therapy Clinical Trials for Patients with Infantile and Late-Infantile Krabbe Disease (SSIEM 2023) - "They receive an individualized immune suppression regimen using Rituximab, Sirolimus and Prednisone according to immune status. Administration of FBX-101 after HSCT represents a novel gene therapy strategy after UCBT. FBX101 in REKLAIM has demonstrated a good safety profile when using immune suppression with Rituximab, Sirolimus and Methylprednisolone/Prednisolone."

Clinical • Gene therapy • P1 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • Transplantation

Updates on the FBX-101 RESKUE Phase I/II Gene Therapy Clinical Trials for Patients with Infantile Krabbe Disease (SSIEM 2023) - "FBX-101 after UCBT leverages the immune/ myeloablation after UCBT, resulting in efficient AAV transduction that prevents antibodies against the transgene and capsid and ensures that sufficient GALC enzyme supports brain and peripheral nerve development."

Clinical • Gene therapy • P1/2 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • Transplantation

Updates on the FBX-101 RESKUE Phase I/II Gene Therapy Clinical Trials for Patients with Infantile Krabbe Disease (SSIEM 2023) - No abstract available

Clinical • Gene therapy • P1/2 data • Gene Therapies

Updates on the FBX-101 RESKUE Phase I/II Gene Therapy Clinical Trials for Patients with Infantile Krabbe Disease (SSIEM 2023) - No abstract available

Clinical • Gene therapy • P1/2 data • Gene Therapies

Updates on the FBX-101 RESKUE Phase I/II Gene Therapy Clinical Trials for Patients with Infantile Krabbe Disease (SSIEM 2023) - No abstract available

Clinical • Gene therapy • P1/2 data • Gene Therapies

Updates on the FBX-101 REKLAIM Phase Ib Gene Therapy Clinical Trials for Patients with Infantile and Late-Infantile Krabbe Disease (SSIEM 2023) - No abstract available

Clinical • Gene therapy • P1 data • Gene Therapies

Updates on the FBX-101 REKLAIM Phase Ib Gene Therapy Clinical Trials for Patients with Infantile and Late-Infantile Krabbe Disease (SSIEM 2023) - No abstract available

Clinical • Gene therapy • P1 data • Gene Therapies

Updates on the FBX-101 REKLAIM Phase Ib Gene Therapy Clinical Trials for Patients with Infantile and Late-Infantile Krabbe Disease (SSIEM 2023) - No abstract available

Clinical • Gene therapy • P1 data • Gene Therapies

Novel gene therapy approach corrects manifestations of Infantile Krabbe Disease. FBX-101 is a Phase I/II Intravenous AAV Gene Replacement Therapy after infusion of transplanted Umbilical Cord Blood (EPNS 2023) - "Administration of FBX-101 during UCBT represents a novel gene therapy strategy that is well tolerated with no adverse events. FBX-101 leverages the myelo/immune-ablated environment of UCBT, resulting in efficient AAV transduction, increasing GALC activity and supporting brain and motor development."

Gene therapy • P1/2 data • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Pain • Transplantation