Timothy Miller – VJRegenMed

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.

Increasing manufacturing capabilities for gene therapies

Simon Ng — Mon, 06 Dec 2021 09:29:00 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, addresses major hurdles facing gene therapy manufacturers. Commercialization of therapies remains a challenge, due to the accelerated development of novel gene therapies for various conditions including Duchenne muscular dystrophy (DMD) and hemophilia and a lack of suitable bioreactors. Scaling up manufacturing processes and increasing quality control will be necessary to meet increased demand from developers. 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.