Category GENE THERAPY RARE DISEASES

UK accounts for over 12% of global cell and gene therapy clinical trials. CGT Catapult new report

The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials.

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bluebird bio Announces Launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

First agreements with statutory health insurances utilize bluebird’s innovative value-based payment model and provide coverage for ZYNTEGLO for up to 50% of patients in Germany First qualified treatment center established at University Hospital of Heidelberg to provide ZYNTEGLO to patients  bluebird bio, Inc. announced the launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a

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The Alliance for Regenerative Medicine Demonstrates Potential for Cell and Gene Therapies to Provide 10-Year Cost Savings to the Healthcare System

ARM today announced the release of a report, “A Transformative Therapy Value Model for Rare Blood Diseases.” The report uses a refined value model to assess the impact of durable and potentially curative cell and gene therapies on health system costs.   Produced in collaboration with the Marwood Institute, this report calculates the potential cost savings that

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Nanoparticles deliver ‘suicide gene’ therapy to pediatric brain tumors growing in mice

Johns Hopkins researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a “suicide gene” to pediatric brain tumor cells implanted in the brains of mice. The poly(beta-amino ester) nanoparticles, known as PBAEs, were part of a treatment that also used a drug to kill the cells and prolong the test

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Gene therapy for chronic pain. Preclinical studies on mice.

Scientists have discovered how to switch off a key ‘pain gene’, dramatically raising hopes of a long-term treatment to relieve the agony of serious illness for millions. The revolutionary technique alters a patient’s DNA, silencing a gene that transmits pain signals up the spine. Preliminary studies on mice have already proven successful and US researchers

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Strategies for the CRISPR-Based Therapeutics

The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many

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Gene therapy could be ‘total game changer for many patients’ with hemophilia A

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A published Gene therapy with valoctocogene roxaparvovec reduced annualized rates of bleeding events and led to discontinuation of prophylactic factor VIII among patients with hemophilia A, according to results of a prospective study with multiyear follow-up published in The New England Journal of Medicine. “The treatment burden is

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Injection of Virus-Delivered Gene Silencer Blocks ALS Degeneration, Saves Motor Function

Novel spinal therapy/delivery approach prevented disease onset in neurodegenerative ALS disease model in adult mice and blocked progression in animals already showing disease symptoms Writing in Nature Medicine, an international team headed by researchers at University of California San Diego School of Medicine describe a new way to effectively deliver a gene-silencing vector to adult amyotrophic

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Mayo clinic researchers test novel injection of gene therapy vectors into the kidney

Before gene therapy can be used to treat renal diseases, delivery of therapeutic genes to the kidney must become much more efficient. A novel approach in which three different gene delivery vectors were injected intravenously and directly into the kidneys of mice was reported in an article published in Human Gene Therapy, a peer-reviewed journal from

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Will Prime Editors be the New CRISPR?

Early results suggest that prime editors are cleaner than CRISPR–Cas9 and more versatile than base editors, but many questions remain. A paper recently published in Nature from David Liu and co-workers discloses a ‘prime’ gene-editing system many years in the making. In principle, the system, comprising a catalytically impaired Cas9 enzyme and an engineered reverse transcriptase, may

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