Advanced Therapies Week 2022 – 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.

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.

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.

Human acellular vessels in coronary artery disease

Simon Ng — Thu, 24 Mar 2022 13:14:38 +0000

Coronary artery bypass graft (CABG) surgery represents the standard of care for patients with coronary artery disease, and whilst nearby vessels such as the internal mammary artery are utilized to relive blockages, some patients use the saphenous vein in the leg. Harvesting the saphenous vein is less efficacious and carries a higher risk of infection. Alan Kypson, MD, FACS, UNC REX Hospital, Raleigh, NC, describes the development of off-the-shelf human acellular vessels (HAVs) that are resistant to infection. Studies in primate models demonstrated the efficacy of HAVs, where smooth muscle and endothelial cells repopulated the graft, successfully replicating a blood vessel. This interview took place at Advanced Therapies Week 2022.

Allogeneic therapy highlights at Advanced Therapies Week 2022

Simon Ng — Thu, 24 Mar 2022 13:14:37 +0000

Jeff Liter, MBA, Luminary Therapeutics, Minneapolis, MN, provides his highlights from Advanced Therapies Week 2022, including advances in allogeneic therapy manufacturing. He discusses developments that have led to more consistent donor material and media expansion techniques, as well as the cooperation between various companies to propel progress in the field. This interview took place at Advanced Therapies Week 2022.

The development of human acellular vessels and its advantages

Simon Ng — Thu, 24 Mar 2022 13:14:35 +0000

Alan Kypson, MD, FACS, UNC REX Hospital, Raleigh, NC, provides an overview of the manufacturing process of human acellular vessels (HAVs) and their advantages in the clinical setting. The production of HAVs begin with the application of banked human vascular smooth muscle cells on a tubular mesh scaffold which is subsequently placed in a bioreactor. As the mesh degrades, the extracellular matrix remains and decellularization enables the HAV to be non-immunogenic. Dr Kypson highlights additional advantages including the large-scale manufacturing capabilities of HAVs, as well as their off-the-shelf nature. This interview took place at Advanced Therapies Week 2022.

The future of small-diameter human acellular vessels

Simon Ng — Thu, 24 Mar 2022 13:14:34 +0000

Whilst the field of cardiac bioengineering has predominantly focused on 3D printing and cell and gene therapies, research on human acellular vessels (HAVs) is still in its infancy. Alan Kypson, MD, FACS, UNC REX Hospital, Raleigh, NC, discusses current developments in HAVs and their potential applications. Large-diameter HAVs can be applied in dialysis for patients with end-stage renal disease or in a surgical setting for patients with peripheral arterial disease (PAD). Dr Kypson additionally emphasizes future research on small-diameter HAVs, which can be used in patients with cardiovascular disease, especially in patients who often lack the appropriate vessels for bypass surgery. This interview took place at Advanced Therapies Week 2022.

OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome

Simon Ng — Thu, 03 Mar 2022 17:46:27 +0000

Kent Christopherson, PhD, Orchard Therapeutics, Boston, MA, provides an overview of OTL-103, an investigational autologous gene therapy utilizing a lentiviral gene construct for patients with Wiskott Aldrich syndrome (WAS). OTL-103 aims to insert the corrected version of the WAS gene in patients, who often result in non-functioning leucocytes and platelets. Administration of the functioning cells will result in the cells differentiating into immune cells as well as the progenitor cells to platelets and a clinical trial is currently underway to assess the efficacy of OTL-103. This interview took place at Advanced Therapies Week 2022.

Novel gene therapies for metachromatic leukodystrophy

Simon Ng — Thu, 03 Mar 2022 17:46:25 +0000

Kent Christopherson, PhD, Orchard Therapeutics, Boston, MA, describes the development and mechanism of action of atidarsagene autotemcel, a gene therapy consisting of an autologous CD34⁺ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced using a lentiviral vector. The gene therapy, which has been approved in the EU, aims to treat metachromatic leukodystrophy (MLD) and the vector encodes the human arylsulfatase-A (ARSA) gene, which is faulty in patients with MLD. HSPC cells with the functional gene can subsequently produce the correct protein, which can be trafficked and permanently integrated in the central nervous system (CNS). 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.