Researchers have successfully demonstrated that CRISPR adenine base editors can be utilized to correct a gene defect causing a hereditary liver disease known as argininosuccinate lyase deficiency (ASLD). ASLD is an autosomal recessive metabolic disease caused by a defect in the ASL gene, resulting in the deficiency of the enzyme arginosuccinate lyase. ASLD can manifest in different forms, ranging from a severe neonatal-onset form to a milder late-onset form. Arginosuccinate lyase plays a role in the urea cycle by cleaving argininosuccinic acid to produce arginine and fumarate, necessary for t ..read more

This week: The US Food and Drug Administration (FDA; MD, USA) cleared the first-ever open Investigational New Drug (IND) for prime editing technology and issued ‘safe to proceed’ IND clearance for a cell therapy targeted at relapsed/refractory myeloid malignancies. Plus, a gene therapy for adults with Hemophilia B was also approved by the FDA. The news highlights: First-ever ex vivo prime editing technology granted IND clearance from the FDA FDA approves one-time gene therapy, BEQVEZ™, for adults with Hemophilia B IND clearance issued by the FDA for cell therapy for relapsed/refractory myelo ..read more

A new study details findings that provide an alternative way to study human lung regeneration. A team of researchers from the Boston Medical Center (BMC; MA, USA) and Boston University (BU; MA, USA) have developed a method using pluripotent stem cells (iPSCs) to generate human alveolar epithelial type I cells (AT1s). The model developed in this study will help researchers comprehensively deepen their understanding of human lung regeneration. Pulmonary diseases such as pulmonary fibrosis, acute respiratory distress syndrome and chronic obstructive pulmonary disease (COPD) affect millions world ..read more

We caught up with Molly Tregidgo (right) from the Cell and Gene Therapy Catapult (CGT Catapult; London, UK) during 3D Cell Culture & Organ Modelling 2024 (18-19 March; London, UK) to get an insight into her work in process development. In this interview, Molly delves into the challenges associated with the 2D expansion of iPSCs and explains how utilizing 3D stirred tank reactors allows for the production of hundreds of functional NK cells per seeded iPSC. Molly also discusses the potential impact of process analytical technologies on cell therapy manufacturing in the future. Please i ..read more

Scientists have developed the application of maleic acid-treated bacterial cellulose (MA-BC) enhancing the innovation of bone repair. A recent research collaboration, between scientists in Nanjing, China and the USA, has been able to successfully prepare a controllable bacterial cellulose (BC)-based bio-ink for 3D printing. The scientists modified neat BC with maleic acid (MA) treatment. A MA-BC-based bio-ink application in bioprinting may promote tissue repair using an eco-friendly candidate that has mild reaction conditions. Scaffold fabrication is an essential step in the production of fun ..read more

This week: Multiply Labs (CA, USA) and Stanford Medicine’s Laboratory for Cell and Gene Medicine (LCGM; CA, USA) collaborate to showcase robotic systems to automate cell therapy manufacturing, Dyno Therapeutics (MA, USA) will present breakthrough methods in artificial intelligence for AAV capsid design and Bristol Myers Squibb (NY, USA) and Cellares (CA, USA) announce a US$380 million worldwide capacity reservation and supply agreement for the manufacture of CAR-T cell therapies. The news highlights: Cell therapy robotics collaboration AI-driven capsid engineering CAR-T therapy manufacturing ..read more

Download peer-reviewed content and expert insights in our latest eBook. To conclude our Spotlight on tissue engineering, this eBook will revisit some of our highlights from the feature, including our infographic on the key trends in engineering tissues for regenerative medicine. We will also investigate innovative strategies for improving tissue engineering approaches, such as utilizing smart scaffolds and in situ 3D bioprinting! [ui-tabs] [ui-tab title="Download now"] [/ui-tab] [ui-tab title="Contents"] Contents: [FOREWORD] Welcome to our eBook on tissue engineering [INTERVIEW] Ti ..read more

Researchers at the Children’s Hospital of Philadelphia (CHOP) and Stanford Medicine (both PA, USA) have recently identified FOXO1 protein as a key regulator of CAR-T-cell longevity – an important factor in their ability to fight cancer. Despite the expanding use of CAR-T therapy, over 50% of patients who receive the therapy experience relapse within a year. The therapeutic benefit of CAR-T cells is partially hindered by their limited longevity in vivo. Once infused into the patient, the engineered cells often don’t survive for long enough to entirely eliminate the cancer, rendering patients v ..read more

This week: The first patient has been dosed in a frontotemporal dementia gene therapy study, Kincell Bio (FL, USA) has acquired Imugene’s (New South Wales, Australia) North Carolina cGMP facility as part of a strategic partnership and Ferring Pharmaceuticals (NJ, USA) has partnered with SK pharmteco (PA, USA) to expand its gene therapy manufacturing capacity. The news highlights: First patient treated in frontotemporal dementia gene therapy trial Imugene and Kincell Bio forge strategic partnership Ferring Pharmaceuticals partners with SK pharmteco to expand gene therapy manufacturing ca ..read more

Our sister site Bioanalysis Zone has launched a new podcast series to explore the important role of bioanalysis in cell and gene therapies!  Cell and gene therapies are comprised of multiple elements including living cells or viral vectors and therefore pose additional bioanalytical challenges compared with chemical or biological drugs. The capability of bioanalytical methods to monitor cell and gene therapy safety and efficacy plays a significant role in the therapy’s success. In this six-part series, Ellen Williams, Digital Editor of Bioanalysis Zone, is joined by industry experts expl ..read more