Hemophilia – VJRegenMed

Overcoming manufacturing issues in gene therapies for rare diseases

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

John Maslowski, CCO, Forge Biologics, Columbus, OH, discusses strategies to optimize the manufacturing of gene therapies for rare diseases. In addition to regulatory barriers, maintaining the consistency of the product is vital, especially when manufacturing occurs in large-scale bioreactors. To ensure the cost of production remains low and the patients’ needs are successfully met, he emphasizes the need to use automated analytical tools in a closed system. Assessing product quality via high throughput systems can expedite production times on a larger scale. This interview took place at Advanced Therapies Week 2022.

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.

Synthetic AAV vectors for gene therapy of hemophilia in children

admin_vjr_user — Fri, 21 May 2021 17:21:44 +0000

Arun Srivastava, PhD, University of Florida, Gainesville, FL, explains that a key challenge associated with the use of adeno-associated virus (AAV) vectors for gene therapy for hemophilia in children is the fact that up until age 10-12, the liver is still growing and dividing, meaning traditional AAV gene therapies are diluted out with every cell division, given their episomal nature. Synthetic AAV vectors containing a no-end (NE) AAV DNA encapsulated in liver-specific synthetic liposomes are currently being investigated. It is hoped that this strategy may allow repeated dosing and specific targeting of the liver, without the induction of a host immune response. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.