Krabbe Disease – VJRegenMed

Innovations in treating rare diseases with gene therapies

Simon Ng — Wed, 20 Apr 2022 16:45:46 +0000

John Maslowski, CCO, Forge Biologics, Columbus, OH, comments on the current treatment landscape for rare diseases, especially in the field of gene therapies. With a large number of rare diseases lacking any form of treatment, finding curative solutions for these rare diseases remain an unmet need. Finding a suitable clinical model is essential to develop treatments, especially when the natural history group in a clinical trial is preferable to placebo groups, as in the case of studies targeting Krabbe disease. Introducing closed automated systems in the manufacturing process will additionally enable the production of gene therapies in an economical manner. This interview took place at Advanced Therapies Week 2022.

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.

Overcoming immunogenicity of gene therapies

Simon Ng — Mon, 06 Dec 2021 09:29:02 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, discusses the impact of pre-existing immunogenicity against adeno-associated virus (AAV)-based gene therapies. Patients who have an immune response to recombinant AAV vectors will require immunosuppression to benefit from gene therapies. Various strategies can be employed to overcome this issue by utilizing combination therapies, especially in the case of FBX-101, an AAV-based gene therapy for patients with Krabbe disease. This interview took place at Meeting on the Mesa 2021.

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.

Treating Krabbe disease with AAV-based gene therapies

Simon Ng — Mon, 06 Dec 2021 09:28:59 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, provides an overview of the current treatment landscape for Krabbe disease and the potential for gene therapy as a curative treatment. Krabbe disease is a lysosomal storage disorder that affects the nervous system, and the current standard of care is a bone marrow transplant. Dr Miller additionally describes an adeno-associated virus (AAV)-based gene therapy that when administered in conjunction with the bone marrow transplant, lacks the immunogenicity-related issues associated with AAV vectors. This interview took place at Meeting on the Mesa 2021.