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cell and gene therapy

This page shows the latest cell and gene therapy news and features for those working in and with pharma, biotech and healthcare.

FDA lifts hold on Vertex and CRISPR Therapeutics sickle cell therapy

FDA lifts hold on Vertex and CRISPR Therapeutics sickle cell therapy

Trial could begin by year end. The FDA has lifted the clinical hold on the trial of a CRISPR gene-editing therapy CTX001 in patients with of sickle cell disease (SCD), ... CTX001 is an investigational, autologous, gene-edited haematopoietic stem cell

Latest news

  • New chair for Europe’s key medicines committee New chair for Europe’s key medicines committee

    Like other major regulators, such as the US FDA, the EMA is updating its structures and review processes as new technologies such as gene and cell therapy emerge from industry pipelines. ... The CHMP has just recommended Luxturna, Spark's gene therapy,

  • Novartis cuts manufacturing jobs in Switzerland and UK Novartis cuts manufacturing jobs in Switzerland and UK

    The company says it has already invested more than $1bn in new production technologies in Switzerland such as its Schweizerhalle facility in Basel, which includes a continuous production plant, cell and ... gene therapy production facilities, a device

  • Sangamo’s first gene-editing trial doesn’t convince investors Sangamo’s first gene-editing trial doesn’t convince investors

    Investors and analysts seemed to be spooked by Sangamo’s inability to show that its one-off therapy was actually causing an increase in IDS levels, which would provide evidence that ... Elaprase costs hundreds of thousands of dollars per year, however,

  • Spark sputters on haemophilia A gene therapy safety scare Spark sputters on haemophilia A gene therapy safety scare

    Spark sputters on haemophilia A gene therapy safety scare. Investors compare data unfavourably with BioMarin rival. ... While there is more to do, I am excited about what Spark has achieved in both haemophilia A and haemophilia B and I have no doubt now

  • Regeneron and Bluebird sign $100m cell therapies deal Regeneron and Bluebird sign $100m cell therapies deal

    Big biotech’Regeneron and cell and gene therapy specialists Bluebird Bio have unveiled a new collaboration which gives both greater presence in next-generation cell therapies. ... and peptides, and Bluebird will contribute its own expertise in gene

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Latest Intelligence

  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    Bluebird Bio's chief executive Nick Leschly. Cell and gene therapy made the transition from an interesting but unproven science to medical reality in 2017, with two ‘living medicine’CAR-T ... If you look at how many gene therapy companies there are

  • The NHS and ABPI at 70: inching closer to the triple win The NHS and ABPI at 70: inching closer to the triple win

    Meanwhile, the pharma industry is hitting new heights in drug innovation, including cures for diseases such as hepatitis C, haemophilia and huge progress in cancer and cell and gene therapy - but ... This, and near-perpetual reform, continues to confound

  • A unique ecosystem A unique ecosystem

    medtech and eHealth companies to launch their products on the North American market. ... Regenerative medicine aims to repair injuries or diseased organs using biomaterials, gene therapy, cell therapy and tissue engineering.

  • Cell and gene therapies: on course for commercial success? Cell and gene therapies: on course for commercial success?

    Unless payers are willing to take a chance, it may be extremely challenging for cell and gene therapy manufacturers to attain reimbursement. ... Manufacturers of cell and gene therapies should be aware that developing an innovative therapy is not enough

  • Opening up innovation Opening up innovation

    So that is really about the late-stage portfolio. We [are still] focused on three areas in oncology.” These are cancer epigenetics, immuno-oncology and cell and gene therapy in oncology. “ ... This scientific platform is probably relevant for all the

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Latest appointments

  • Weekly industry appointments Weekly industry appointments

    Weekly industry appointments. GSK’s CSO cell and gene therapy heads for the exit, Gelesis adds to company, Abcam appoints Peter Allen and Joseph Swedish joins Mesoblast’s board. ... be a global leader in the development and commercialisation of cell

  • Celgene boosts CAR-T stake again with Bluebird Bio deal Celgene boosts CAR-T stake again with Bluebird Bio deal

    Mere weeks after completing its acquisition of CAR-T specialist Juno, Celgene has added to its cell therapy pipeline with another clinical-stage CAR-T candidate from Bluebird Bio. ... well as a pipeline of other cell and gene therapy candidates.

  • Orchard Therapeutics appoints Mark Rothera as CEO Orchard Therapeutics appoints Mark Rothera as CEO

    He said: “Now is an exciting time to be joining Orchard as the cell and gene therapy field is advancing successfully. ... The clinical data we’ve seen to date with Orchard’s therapy for ADA-SCID is exceptional, and the company has assembled and

  • Avita Medical appoints CEO Avita Medical appoints CEO

    chief scientific officer, global business development and licensing, and chief scientific officer for its cell and gene therapy unit. ... and Genetic Therapy Inc. Lou Panaccio, chairman of Avita, said: “Mike’s expertise across the value chain in cell

  • apceth Biopharma appoints Dusan Kosijer as CFO apceth Biopharma appoints Dusan Kosijer as CFO

    strategy. He joined Munich-based cell therapy-focused company in 2016 and was initially responsible for the company’s finance strategy and operations and the leadership of the accountant and purchasing ... successful track record as one of the leading

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Latest from PMHub

  • Training the next generation of biotech leaders

    Training the next generation of biotech leaders. Cell and gene therapy is no longer science fiction. ... As a result, he is at the forefront of inspiring the next generation of scientists to make breakthroughs in cell and gene therapy.

  • Healthcare in the Digital Age

    Drug discovery is buoyant and innovative technology is pushing medicine forward at unprecedented speed. ... Exciting developments in immunotherapy and oncology, and huge strides in gene and stem-cell therapy are matched by powerhouse collaborations

  • Life at the end of the Brexit tunnel

    An Advanced Therapies Treatment Centre to develop cell and gene therapies. ... Expansion of the Cell and Gene Therapy Manufacturing Centre in Stevenage.

  • Life at the end of the Brexit tunnel

    An Advanced Therapies Treatment Centre to develop cell and gene therapies. ... Expansion of the Cell and Gene Therapy Manufacturing Centre in Stevenage.

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