All Cell & Gene Therapy articles – Page 6
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NewsProgramming immunity from within: in-body generation of CAR T cells
Researchers have developed a new method to generate CAR T cells directly inside the body using targeted lipid nanoparticles that deliver mRNA to T cells - offering a safer, faster and more accessible alternative to traditional cell therapies for cancer and autoimmune diseases.
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ArticleInside AACR: DARPins take aim at hard-to-treat tumours
Amelie Croset of Molecular Partners breaks down the DARPin developments featured at AACR 2025 – from targeted radiopharmaceuticals to precision-engineered immune cell engagers.
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$250K grant fuels development of new type 1 diabetes therapy
Researchers at Washington University in St. Louis have received a $250,000 grant from the Critical Path Institute’s Translational Therapeutics Accelerator (TRxA) to develop a novel CD22 bidentate therapeutic for type 1 diabetes to support formulation and new preclinical studies.
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New discovery shows how viral vectors release genes in therapy
Researchers at The University of Osaka have found how structural changes in the VP1 protein of AAV vectors trigger genome release - offering insights for improving the safety, efficiency and stability of gene therapy delivery systems.
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News‘Delete-to-recruit’: new gene-editing method targets sickle cell
Researchers have developed a novel gene therapy approach that reactivates dormant genes by repositioning them closer to genetic switches called enhancers - showing promise for treating blood disorders like sickle cell disease.
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NewsTurning AI into a biological design engine
DenovAI has unveiled a powerful AI-driven protein design platform capable of creating new, functional synthetic proteins from scratch - marking a big step forward for drug discovery.
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NewsNew therapy for incurable brain disease heads to clinical trials
SynaptixBio has selected its lead drug candidate, SB H-19642, for clinical trials to treat H-ABC - a rare, fatal neurodegenerative disease with no current cure.
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Stanford grows vascularised mini-organs
Stanford scientists have successfully grown heart and liver organoids that include functioning blood vessels. This breakthrough overcomes a major size and maturity barrier, which could advance disease modelling and regenerative therapies in the future.
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NewsSB000: a safer path to anti-aging therapies
Shift Bioscience has announced new aging research, highlighting the discovery of SB000. This novel single-gene target reverses cellular aging without activating dangerous pluripotency pathways.
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ArticleNew tRNA tech aims to rewrite rare disease treatment
Alltrna is redefining the future of rare disease treatment with engineered tRNA therapeutics that target genetic mutations - not just individual diseases. CEO Michelle Werner shares how this bold, mutation-driven approach could unlock universal treatments for thousands of patients long overlooked by traditional medicine.
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ArticleAutomated red blood cell exchange: bridging treatment gaps in sickle cell disease care
Despite the promise of gene therapies, automated red blood cell exchange (aRBCX) remains an underutilised therapy in the management of sickle cell disease (SCD). In this article, Dr Aaron Haubner and Carly Newton of Terumo Blood and Cell Technologies, highlight the urgent need for partnerships and equitable access to this ...
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NewsHerpes virus protein boosts T cell power against cancer
Researchers at the University of Michigan have engineered a herpes virus protein to enhance T cell survival and function, offering a new strategy to strengthen cancer immunotherapy.
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NewsAstrocyte protein RTP801 linked to Alzheimer’s cognitive decline
A new study from the University of Barcelona’s Institute of Neurosciences has discovered a crucial role for the RTP801 protein in astrocytes, potentially making way for future therapies aimed at slowing or reversing cognitive decline.
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NewsNew UCD centre targets the biggest bottleneck in rare research
The world’s first translational research center dedicated to urea cycle disorders (UCDs) has been launched in Zürich, marking a significant moment in rare disease innovation.
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NewsVascularised stem cell organoids advance diabetes therapy
A team of researchers have developed the first vascularised organoid model of human pancreatic islets, which could lead to further development of advanced cell therapies for diabetes.
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NewsNasal spray gene therapy shows potential to treat lung diseases
Researchers at Mass General Brigham have developed a new gene therapy delivery system, AAV.CPP.16, that targets the lungs via nasal spray, which could offer a potential breakthrough in respiratory gene therapy.
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ArticleTackling complex manufacturing challenges in modern drug development
Chief Scientific Officer at Solvias reveals how CROs are overcoming manufacturing challenges and harnessing AI to fast-track the development of groundbreaking cell and gene therapies.
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ArticlePOLB 001: tackling cytokine storms before they start
Cytokine release syndrome (CRS) is a major barrier in cancer immunotherapy - but what if we could prevent it before it begins? Dr Liam Tremble, Principal Scientist at Poolbeg Pharma, discusses how POLB 001, an oral anti-inflammatory candidate, could offer a new way forward.
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ArticleTransforming biotherapeutics delivery for local communities
Vitalant is transforming the path from preclinical drug discovery to patient care by enabling access to cutting-edge cell and gene therapies. With decades of expertise and a nationwide network, find out how they bridge the gap between innovative treatments and local communities, ensuring life-changing therapies reach patients efficiently.
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ArticleCracking the code to scalable cell and gene therapy
Cell and gene therapy is rapidly transforming the treatment of complex diseases, yet scaling production efficiently remains a challenge. Dr Tia Harmon from PHC Corporation of North America explores the innovations that are optimising cell expansion to enhance the discovery of advanced therapies.