news
Mechanism that controls reactivation of neural stem cells discovered
New insights into the role of SUMOylation in the brain could lead to new targeted therapies for neurodegenerative conditions.
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Cell & gene therapy
article
Cell and gene therapy development moves into cardiac indications
The success of CAR-Ts in oncology has stoked enthusiasm for developing comparable curative therapies in other disease areas. CellProthera’s Chief Scientific Officer, Ibon Garitaonandia, explains the potential and progress for CGTs in cardiology, where disease-modifying therapies are largely non-existent.
podcasts
Scaling genetic medicine with tRNA
Tune into this episode which explores how engineered tRNAs could treat thousands of rare diseases and cancers that share the same mutation.
article
Overcoming operational challenges in CGT manufacturing with AI
In this Q&A, Dalip Sethi, scientific lead for Terumo Blood and Cell Technologies, Cell Therapy Technologies and Innovation portfolio, discusses the integration of AI into CGT manufacturing processes to enhance operational efficiency and accelerate treatment access. He also elucidates the strategies that are being considered to overcome hurdles when implementing…
article
The rising demand for lentiviral vectors for in vivo gene therapy
Fuelled by advances in rare disease treatments and vaccination efforts, Natalia Elizalde, CBO at VIVEbiotech, discusses how the market demand for lentiviral vectors is evolving, the new therapeutic areas emerging as potential targets for in vivo gene therapy and the latest technological advancements in the development and delivery of in…
news
Prime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
news
Rare types of helper T cells uncovered
Researchers have created a new T cell atlas which could aid the development of novel drug therapies for immune-mediated diseases.
news
Cancer dependencies missed by some CRISPR guides
Scientists discovered that, depending on germline variation, CRISPR-based experiments can result in false negatives.
article
Tessera Therapeutics: addressing Alpha-1 Antitrypsin Deficiency
As the industry looks beyond CRISPR to safely introduce therapeutic genomic changes anywhere in the body, in vivo gene editing holds immense potential to address diseases with a genetic basis. Boston-based biotech Tessera Therapeutics is pioneering the next generation of genetic medicines with its Gene Writing™ platform. At this year’s…
news
New ENaC blocker demonstrates an extended duration of action
The novel drug, ETD001, could provide an improved approach for mucus clearance in cystic fibrosis patients.
news
Macrophage-mediated phagocytosis: treating solid tumours
A new immunotherapy approach combining co-engineered T cells and antibodies enhances phagocytosis of tumour cells.
article
The promise of vectors targeting the LDL receptor
In this Q&A, Pascaline Lécorché, Head of Chemical Biology at Vect-Horus, outlines the potential of RNA interference therapeutics and the challenges of their use in vivo. There are several strategies to overcome these, and why peptide-based vectors targeting the LDL receptor are a promising approach.
news
Novel version of CRISPR gene-editing protein engineered
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
news
New method to predict effectiveness of MSCs for cartilage repair in vitro
Topological defects in mesenchymal stromal cell self-assembly could suggest their potential for cartilage regeneration.
article
Enhancing gene therapy with Circio
In this Q&A, Erik Wiklund, CEO of Circio, explains the key findings of their circVec circular RNA platform technology, why they chose AAV-based gene therapy for AATD as the lead programme, and their plans for the future to enhance the potency and reduce the cost of current gold-standard gene therapy.