Stem Cells – VJRegenMed

Streamlining cell therapy manufacturing with CliniMACS Prodigy

Simon Ng — Thu, 28 Apr 2022 08:38:41 +0000

Saskia Rösch, PhD, Miltenyi Biotec, Bergisch Gladbach, Germany, gives an overview of CliniMACS Prodigy, a novel next-generation automated cell processing platform that aims to separate and process cell cultures for the manufacturing of cell therapies. The closed, scalable system enables the production of good manufacturing practice (GMP) compliant products of consistent quality in a safe and clean environment. CliniMACS Prodigy additionally is flexible in a centralized or decentralized setting and can be used for various cell types including induced pluripotent stem (iPS) cells, chimeric antigen receptor (CAR) T-cells and natural killer (NK) cells. This interview took place at the International Conference on Lymphocyte Engineering (ICLE) 2022.

Applications of stem cell gene therapy beyond genetic diseases

admin_vjr_user — Fri, 22 Apr 2022 12:21:18 +0000

Luigi Naldini, MD, PhD, San Raffaele Telethon Institute for Gene Therapy, Milano, Italy, comments on the potential application of stem cell gene therapy in oncology. In recent years, growing evidence supporting the efficacy and tolerability of this technology in genetic diseases has suggested it as a promising strategy to target tumors by engineering stem cells to target the tumor or to deliver a therapeutic agent. This interview took place at the Transplantation & Cellular Therapy (TCT) Meetings of ASTCT and CIBMTR® 2022 in Salt Lake City, Utah.

The future of omentum-based autologous tissue engineering

Simon Ng — Thu, 21 Apr 2022 15:01:23 +0000

Advances in autologous tissue regeneration in the form of an omentum-derived hydrogel and induced pluripotent stem cell (iPSC)-derived tissue have led to promising therapies for spinal cord injury and cardiac infarction. Asaf Toker, MD, Matricelf, Ness Ziona, Israel, provides an overview of the future of this novel technology. Whilst currently only being assessed in two indications, this method of autologous tissue engineering can theoretically be applied to any type of damaged tissue. Potential indications include Parkinson’s Disease, age-related macular degeneration (AMD) and other musculoskeletal disorders. This interview was conducted during Meeting on the Mediterranean 2022.

Autologous tissue-engineered solutions for spinal cord injuries

Simon Ng — Wed, 20 Apr 2022 14:43:27 +0000

Asaf Toker, MD, Matricelf, Ness Ziona, Israel, discusses autologous neural implants for patients with spinal cord injuries, which consists of the implant itself and a scaffold. Omental tissue extracted from the patient via a biopsy is decellularized, resulting in the formation of thermo-responsive hydrogel that serves as the scaffold for the tissue-engineered product. Differentiation of induced pluripotent stem cells (iPSCs) derived from the patient’s mature cells within the thermo-responsive hydrogel results in the formation of neural cells that can connect damaged spinal tissue. First-in-man clinical trials for the product are planned in 2025 in patients with spinal cord injuries. This interview was conducted during Meeting on the Mediterranean 2022.

Restoring cardiac tissue with omentum-based hydrogel implants

Simon Ng — Wed, 20 Apr 2022 14:43:26 +0000

Asaf Toker, MD, Matricelf, Ness Ziona, Israel, describes of the development of an autologous omental hydrogel to treat damaged cardiac tissue after a myocardial infarction. In a similar fashion to neural implants for spinal cord injuries also produced by Matricelf, the novel therapy utilizes autologous omental tissue that is decellularized to make a thermo-responsive hydrogel. Induced pluripotent stem cells (iPSCs) derived from the patient’s peripheral blood subsequently differentiates into cardiac tissue when attached to the hydrogel. The implant can either be surgically attached on the injured tissue or can be injected and then solidify once in the body. This interview was conducted during Meeting on the Mediterranean 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.

Overview of the ekko™ acoustic cell processing system

Simon Ng — Thu, 24 Feb 2022 11:16:29 +0000

Nina Bauer, PhD, MBA, Merck KGaA, Darmstadt, Germany, gives an overview of the process and advantages of the ekko acoustic cell processing system, a platform technology for cell and gene therapy manufacturing. In contrast to traditional mechanical or filtration methods that rely on filters with fixed pore sizes and centrifugal forces, the ekko system is based on acoustic waves which allow to gently concentrate and wash cells. The ekko system is a flexible tool that can process very small volumes as well as large aggregates which can be used in multiple cell applications. This interview took place at Advanced Therapies Week 2022.

Adapting cell therapies to solid tumors

Simon Ng — Thu, 24 Feb 2022 11:16:29 +0000

Nina Bauer, PhD, MBA, Merck KGaA, Darmstadt, Germany, outlines the key differences in manufacturing cell therapies for liquid and solid tumors and comments on some of the changes that need to be made to meet the future demand of cell therapies for solid tumors. Dr Bauer explains that cell therapies are still in their infancy and manufacturing therapies for both liquid and solid tumors require further optimization. Nevertheless, chimeric antigen receptor (CAR) T-cell therapy has been significantly more successful in liquid tumors due to easier tumor access and several changes need to be made for widespread adoption in solid tumors. Ongoing research is currently investigating which immune cells are the most efficient at penetrating solid tumors, as well as strategies to produce larger quantities of cells for a wider group of patients. This interview took place at Advanced Therapies Week 2022.

ARU-1801, a novel lentiviral gene therapy for SCD

Simon Ng — Thu, 03 Feb 2022 12:25:15 +0000

Current investigational therapies for sickle cell disease (SCD) require conditioning chemotherapy such as busulfan to administer the genetically-modified stem cells in the patient’s bone marrow. Will Chou, MD, Aruvant, New York, NY, provides an overview of ARU-1801, for patients with SCD. The lentiviral gene therapy uses fewer doses of an alternative chemotherapy, melphalan, which results in fewer side effects such as thrombocytopenia and veno-occlusive liver disease, as well as shorter hospital stays. This interview took place at Advanced Therapies Week 2022.

Addressing issues associated with bringing iSPC-derived MSCs to the clinic

Simon Ng — Thu, 03 Feb 2022 12:25:13 +0000

Tony Ting, PhD, Bone Therapeutics SA, Gosselies, Belgium, discusses challenges associated with developing cell therapies for clinical use. Chemistry, Manufacturing and Controls (CMC) remains a major barrier for many manufacturers, which lead to the creation of genetically engineered induced pluripotent stem cell-(iPSC)-derived mesenchymal stem cells (MSCs). Dr Ting additionally highlights how genetically engineered therapies can overcome regulatory issues relating to potency assays. This interview took place at Advanced Therapies Week 2022.