All Gene Therapy articles – Page 4
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WebinarSolving the AOC puzzle: Strategies for chemistry, manufacturing and regulatory success
Watch our webinar with Abzena’s experts to explore practical solutions for overcoming chemistry, manufacturing and regulatory challenges in antibody-oligonucleotide conjugate (AOC) development, enabling progress for previously untreatable diseases.
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NewsAWS and ElevateBio join forces to advance gene editing
ElevateBio is teaming up with Amazon Web Services (AWS) to advance CRISPR gene editing using AI and cloud computing. This collaboration aims to accelerate drug discovery for genetic diseases, making next-generation therapies more efficient and accessible.
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NewsRare diseases, big advances: how research is powering a new era of treatments
This Friday, February 28, marks Rare Disease Day 2025, a global initiative raising awareness for the 300 million people living with rare conditions. Dr David Reynolds, CEO of LoQus23 Therapeutics, explores how advancements in rare disease research are driving the development of new treatments.
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NewsNew gene identified for treating malignant cardiac arrhythmias
Researchers have identified a small gene, SCN10a-short, that could enable gene therapy to treat malignant cardiac arrhythmias. This discovery offers the potential for a one-time treatment, reducing the reliance on lifelong medication and invasive procedures.
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NewsDNA repair genes drive Huntington's disease, UCLA study finds
A new UCLA study reveals that DNA mismatch repair genes play a crucial role in Huntington’s disease by driving neuronal damage and motor impairments. Targeting these genes, especially Msh3 and Pms1, could offer promising therapeutic avenues for the disease.
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ArticleEmpowering women in STEM: championing change in genetics
An advocate for genetic medicines and the enormous potential they hold, Leah Sabin at Regeneron Genetic Medicines reveals how passion and curiosity can forge the path to a rewarding career in STEM.
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ArticleExperts explore the future of iPSC-based cell therapies
Discover the transformative potential of iPSC-based therapies in regenerative medicine, alongside their challenges including scalability, safety and targeted delivery.
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NewsTLE6 protein deficiency linked to male infertility
New study reveals that TLE6 protein deficiency causes male infertility in mice. These findings suggest potential genetic causes and future treatment avenues for male infertility.
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NewsRett syndrome discovery could lead to better treatments
Researchers have identified early molecular changes in Rett syndrome that could lead to improved treatments for the condition.
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NewsStudy reveals how neurotransmitters influence brain gene expression
A new study demonstrates how neurotransmitters affect brain gene expression, offering potential therapeutic insights for circadian rhythm disorders.
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NewsNew genetic mechanism could lead to RNA therapies for mental health
Researchers at the Medical University of South Carolina have discovered a key genetic mechanism that could lead to RNA-based therapies for psychiatric disorders triggered by emotional experiences.
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NewsPHC Corporation to present LiCellGrow at Advanced Therapies Week 2025
The LiCellGrow cell expansion system enables real-time metabolic monitoring and automated culture optimisation to accelerate cell and gene therapy manufacturing.
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NewsTessera's gene editing offers hope for sickle cell patients
Find out how Tessera's latest advancements in gene editing bring promising solutions to treat sickle cell disease.
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ArticleIn vivo CAR T: Faster, cheaper, and more effective cancer care
Find out how in vivo cell therapies could transform cancer treatment by providing faster, more affordable, and accessible options for more patients.
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ArticleOvercoming obstacles in personalised medicine
Tune in to this episode to learn how overcoming the key challenges in personalised medicine could transform the future of healthcare.
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ArticleNew insights into the role of viral capsids in gene therapy safety
Viral capsid assembly and quality are key factors in gene therapy success and safety. Here, Dr Chelsea Pratt explores how cutting-edge tools are enabling scientists to tackle these challenges and improve patient outcomes.
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NewsBlood-brain barrier breakthrough for ALS and Alzheimer's disease
A new blood-brain barrier-crossing platform enables the delivery of therapeutic molecules to the brain, offering potential treatments for ALS and Alzheimer’s disease.
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ArticleWomen in STEM with Veerle d’Haenens
We had the privilege of talking to Veerle d’Haenens, General Manager, Global Therapy Innovations at Terumo Blood and Cell Technologies. Her successful career has been driven by her creativity, expertise and passion, and she advises women who are aspiring to STEM roles to always think big.
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ArticleCell 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.
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ArticleScaling 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.