All Rare & Genetic Disorders articles – Page 4
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NewsGlox Therapeutics secures £500K to develop antibiotics for cystic fibrosis
Glox Therapeutics has received a portion of a £3 million fund to combat antimicrobial resistance in cystic fibrosis patients.
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NewsFirst patient enrolled in fostamatinib trial for sickle cell disease
Rigel Pharmaceuticals has announced the enrolment of the first patient in a Phase I trial evaluating fostamatinib for sickle cell disease.
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ReportBeyond the Lab: Stem cell research
Download our latest report to discover how stem cell research is revolutionising treatments for chronic diseases, cancer and regenerative medicine.
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NewsNew painless vaccine technology targets viruses
Learn how Kindeva and Emervax are advancing vaccine delivery with painless, needle-free technology designed to improve accessibility and uptake worldwide.
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NewsPhage therapy: A step forward in the fight against antibiotic resistance
A new study shows how phages, viruses that kill bacteria, can coexist on a single bacterial strain. This offers valuable insights for developing more effective therapies against antibiotic-resistant infections.
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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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NewsNew data shows benefits of automated blood exchange for sickle cell disease
New studies highlight the potential of automated blood exchange to improve outcomes for sickle cell disease patients.
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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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ArticleOncology advances through the lens of women in STEM
Dr Pooja Hingorani opens up about her journey in oncology research, the obstacles she’s overcome as a woman in STEM, and her mission to transform cancer care.
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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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NewsBreakthrough trial offers new hope for cystic fibrosis patients
A promising new inhaled therapy is progressing in Phase 2 trials, presenting new opportunities for improving cystic fibrosis treatment.
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NewsAI for drug discovery in advanced pain management
A deep-learning model found metabolites and drugs that could be repurposed for non-addictive and non-opioid options to treat chronic pain.
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NewsCrohn’s disease: discovery of two distinct molecular subtypes
By studying organoids derived from adult stem cells, researchers made a discovery that could improve the development of personalised therapeutics.
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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.
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NewsDamaged small intestine repaired with hPSC-derived organoids
Cincinnati Children’s have repaired damaged intestines in a rodent model, moving research efforts closer to clinical trials.
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NewsmRNA vaccine provides enhanced protection against mpox
The mRNA vaccine candidate more effectively limited symptoms and disease duration in non-human primates.
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ArticleExploring the potential of ADCs beyond oncology
Antibody Drug Conjugates (ADCs) are already deployed as part of the roster of oncology treatments, but here, Mike Ward, Head of Thought Leadership for Life Sciences & Healthcare, Clarivate, looks into their potential to help treat a whole range of other diseases and conditions. He discusses the barriers and enablers ...
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NewsSpinal muscular atrophy: searching for a cure
Organoids with SMA-pathology uncovered key findings about the disease, which could be utilised to develop new therapeutic options.
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NewsPrime 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.
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ArticleSmall molecules for regenerative medicine
In this episode, we discuss how new insights into stem cell biology are leading to novel therapeutics in regenerative medicine beyond cell therapy.