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gene therapy

Commercial-stage company — Orchard Therapeutics — has completed its Series C financing round, raising $150 million to be used in the advancement of its gene therapy pipeline. more

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The UCL Technology Fund has revealed that one of its investments, MeiraGTx — a clinical stage gene therapy company — has raised $75 million of gross proceeds upon the closing of its initial public offering (IPO) earlier this year. more

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Pfizer and Spark Therapeutics have announced that the Phase III programme for the investigational haemophilia B gene therapy is to be initiated by Pfizer. more

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Here, Tony Hitchcock, technical director at Cobra Biologics and Joanne Anderson, business development director at Symbiosis, discuss the challenges associated with manufacturing for gene therapies. more

, Opinion

Researchers based at King’s College London have shown in recent work that gene therapy can restore hand function after spinal cord injury in rats. more

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GlobalData has stated that according to its latest research immune-based gene therapy candidates should prevail in oncology, however, this novel approach is unchartered territory in oncology and will require new regulatory guidelines more

Analysis

An alliance has been formed between SIRION Biotech and Heidelberg University Hospital for the development of novel AAV capsids and technologies addressing shortcomings of clinical gene therapy trials. more

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Orchard Therapeutics, a commercial stage company, has been granted a Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its gene therapy candidate, OTL-200, for the treatment of patients with MLD more

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Producer of tailor-made recombinant glycoproteins and gene therapy vectors, CEVEC Pharmaceuticals, and global biotechnology company, CSL, have announced an exclusive licensing agreement to produce a recombinant C1 Esterase Inhibitor. more

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A strategic agreement has been signed by GlaxoSmithKline (GSK) and Orchard Therapeutics, involving the transfer of GSK’s portfolio of approved and investigational rare disease gene therapies to Orchard. more

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TrakCel, software developer for gene therapy supply chain tracking and orchestration, has announced it will be organisation that is responsible for tracking and documentation of cell and gene therapy products for the Northern Alliance more

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Healthcare solutions provider, Novartis, is set to gain further ground in the area of gene therapy through its agreement to acquire AveXis — US-based clinical stage gene therapy company — for $8.7 billion. more

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Cell & Gene Therapies: A guide to single-use fluid connections more

Biochromatography development and manufacturing company, BIA Separations, and biotech, RBPS Technology, have signed a partnership agreement to support the clinical-stage production of high purity Minicircle DNA for viral and non-viral gene transfer more

Technology

A partnership agreement has been signed by BIA Separations, a biochromatography development and manufacturing company, and Nuvonis Technologies, a biotech company, to support the production of vaccine and gene therapy. more

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Gene control company, Synpromics, has announced it will collaborate with the University College London (UCL) Great Ormond Street Institute of Child Health for the development of novel gene therapies for pathologies affecting the haematopoietic system more

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German-based company, SIRION Biotech has signed a two-year development agreement with a clinical-stage ophthalmology company, Acucela, to establish optimised adeno associated virus (AAV) vectors for clinical applications in ocular gene therapy. more

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US-based company, Spark Therapeutics, has announced a programme of improved access to its one-time gene therapy treatment, LUXTURNA, indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy more

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Lonza and Massachusetts Eye and Ear (MEE) have granted Akouos (a new biotechnology company) licences for exclusive rights to the ancestral adeno-associated viral vectors (Anc-AAV) gene therapy platform for all hearing and balance disorders. more

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Gene control company, Synpromics, has revealed its collaboration with University College London (UCL) for the generation of a range of synthetic gene promotors for the central nervous system (CNS) to develop a therapy for Parkinson’s disease. more

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