Treating Krabbe disease with FBX-101

Simon Ng — Thu, 17 Feb 2022 12:04:56 +0000

Krabbe disease is a rare genetic disorder caused by a deficiency of the enzyme galactocerebrosidase (GALC) and can result in mortality in infants. John Maslowski, CCO, Forge Biologics, Columbus, OH, provides a brief overview of FBX-101, an adeno-associated virus (AAV)-based gene therapy for patients with Krabbe disease. The therapy is currently being assessed in the Phase I/II RESKUE trial (NCT04693598), where patients also receive a bone marrow transplant. FBX-101 has demonstrated preliminary efficacy and the trial will additionally assess safety as well as mobility in patients. This interview took place at Advanced Therapies Week 2022.

RESKUE: AAV-based gene therapy for Krabbe disease

Simon Ng — Mon, 06 Dec 2021 09:29:01 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, describes the trial design of the Phase I/II RESKUE trial (NCT04693598), assessing FBX-101, an adeno-associated virus (AAV)-based gene therapy for patients with Krabbe disease receiving a bone marrow transplant. The preliminary results of the dose escalation trial will aim to be released by 2022. This interview took place at Meeting on the Mesa 2021.

RESKUE – VJRegenMed

Treating Krabbe disease with FBX-101

RESKUE: AAV-based gene therapy for Krabbe disease