AAV Vectors – VJRegenMed

Meeting the growing demand for gene therapy manufacturing

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

John Maslowski, CCO, Forge Biologics, Columbus, OH, gives an overview of Forge Biologics’ plans to meet the increasing demand for gene therapies. The recent $80 million expansion of the manufacturing facilities at Forge Biologics with automated components and clean rooms will enable global expansion. Additional recruitment of personal and inhouse analytical services will also enhance current adeno-associated virus (AAV) vector production capabilities. This interview took place at Advanced Therapies Week 2022.

Tangential Flow Depth Filtration in viral vector manufacturing

Simon Ng — Thu, 03 Mar 2022 17:46:23 +0000

Rachel Legmann, PhD, Repligen, Waltham, MA, discusses the Tangential Flow Depth Filtration (TFDF) platform and its benefits. TFDF combines tangential flow and depth filtration in a single closed system, enabling high cell density cultures for the production of various viral vectors such as adeno-associated viruses (AAV) and lentiviruses in gene therapies. A high cell density results in higher viral vector yields and a perfusion system allows the final product to be continuously collected. The platform additionally requires a lower surface area for defiltration, meaning that viral vectors can be manufactured in a shorter timeframe. This interview took place at Advanced Therapies Week 2022.

Challenges in manufacturing viral vectors for the clinic

Simon Ng — Mon, 28 Feb 2022 17:56:23 +0000

Nathalie Clement, PhD, Unicorn Consultations, Gainesville, FL, describes challenges in the development and production of clinically effective viral vectors. Manufacturing larger doses on a bigger scale has been a barrier for companies, and difficulties in purification has led to issues in potency and safety. Finding a standard method for purifying viral vectors is especially difficult when there are numerous varieties of viral vectors including adeno-associated viruses and lentiviruses. Whilst the demand for viral vector gene therapies have increased in certain disease areas such as Duchenne muscular dystrophy, safety concerns possibly relating to impurities in the product must be addressed. This interview took place at Advanced Therapies Week 2022.

Meeting the increasing demand for viral vectors

Simon Ng — Fri, 18 Feb 2022 15:46:07 +0000

Rachel Legmann, PhD, Repligen, Waltham, MA, describes issues in producing viral vectors and approaches to improve manufacturing capabilities. Low yield and constraints in bioreactor capacity affects a wide range of viral vectors including adeno-associated viral (AAV) vectors, lentiviral vectors, and oncolytic vectors. Additional barriers include the process of replicating viral vectors, where transfection and lysis are stages in the manufacturing process that are difficult to scale up. Viral vectors are also more difficult to produce cost effectively due to the stricter safety regulations needed. 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.

The past, present and future of AAV vectors

Simon Ng — Tue, 15 Feb 2022 12:24:08 +0000

Nathalie Clement, PhD, Unicorn Consultations, Gainesville, FL, provides an overview of her experiences working with adeno-associated viral (AAV) vectors, from its infancy of the field to the present day, where clinical trials are being conducted to assess AAV-based therapies. Dr Clement additionally highlights the growth of the AAV vector field and the increased demand for treatments, as well as the history of AAV vector research. This interview took place at Advanced Therapies Week 2022.

Innovations in viral vector development

Simon Ng — Tue, 15 Feb 2022 12:24:07 +0000

Nathalie Clement, PhD, Unicorn Consultations, Gainesville, FL, discusses strategies for safe and effective viral vector development. Whilst there are a variety of viral vectors including adeno-associated viral (AAV) vectors, there are common safety concerns. However, collaboration between stakeholders can drive innovation, especially in the case of developing an AAV-based COVID-19 vaccine, which required quick adaptation of existing technology for vaccine manufacturing. Dr Clement additionally describes research to improve vector yields as well as efforts to discover more efficient capsids. This interview took place at Advanced Therapies Week 2022.

Optimizing AAV vectors with AI

Simon Ng — Mon, 07 Feb 2022 17:06:03 +0000

Christopher Reardon, Dyno Therapeutics, Cambridge, MA, discusses using artificial intelligence (AI) to enhance the properties of adeno-associated virus (AAV) vectors. Whilst a vast number of changes to the capsid are possible, the advent of AI technology can refine the process to make productive alterations. The CapsidMap platform has enabled the optimization of AAV capsids, with the intention of using personalized AAV vectors for therapies instead of the standard AAV9 or AAV2 vectors. This interview took place at Advanced Therapies Week 2022.

Clinical challenges in developing AAV-based gene therapies

Simon Ng — Tue, 01 Feb 2022 18:03:38 +0000

Adeno-associated virus (AAV) vectors have become pivotal in the clinical development of gene therapies. Christopher Reardon, Dyno Therapeutics, Cambridge, MA, describes challenges associated with using AAV vectors, including regulatory barriers. Despite a small number of gene therapies successfully being approved, the pathway for regulating this novel technology requires consolidation and optimization. This interview took place at Advanced Therapies Week 2022.

The need for standardization in cell and gene therapy manufacturing

Simon Ng — Fri, 28 Jan 2022 09:54:52 +0000

Michael Lehmicke, Senior Director of Science & Industry Affairs, Alliance for Regenerative Medicine, Washington, D.C., discusses the importance of generating and adhering to best practices and standards in cell and gene therapy (CGT) manufacturing. Mr Lehmicke explains that a major issue in the CGT industry is disagreement on best manufacturing practices. He outlines some of the efforts being made by ARM to tackle this problem, highlighting the A-Gene and A-Cell projects which aim to describe the application of quality by design to the development of CGT products. This interview took place at Advanced Therapies Week 2022.