Category: beta thalassaemia

  • Precision Bio climbs on near-$1.5bn sickle cell pact with Novartis

    Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). The Swiss pharma group is paying $75 million upfronting cash and equity to get the ball rolling on the collaboration, with another $1.4 billion in potential […]

  • EHA: Vertex, CRISPR build case for gene-editing drug

    A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. New data from the CLIMB-111, CLIMB-121, and CLIMB-131 presented in a late breaker at the […]

  • Second unanimous FDA adcomm vote boosts bluebird bio

    bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. By the end of the week, it was two for two, with both recommended for approval by a unanimous vote. Thursday saw the Cellular, […]

  • ICER says bluebird bio’s $2.1m gene therapy is cost-effective

    US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. ICER’s assessment is still at the draft stage and could be changed after an ongoing comment period, but will be […]

  • Vertex builds in gene editing yet again, with $1.2bn Arbor deal

    Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 billion. The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 […]

  • EMA finds possible link between mRNA COVID jabs and myocarditis

    The EU medicines regulator has said that there is some evidence to suggest a possible link between mRNA-based vaccines for COVID-19 and rare cases of heart inflammation.  According to the EMA’s pharmacovigilance risk assessment committee (PRAC), labelling for Pfizer/BioNTech’s Comirnaty and Moderna’s Spikevax should be updated to reflect “very rare cases” of myocarditis and pericarditis […]

  • EHA21: Study raises BMS’ hopes for Reblozyl in less severe disease

    Bristol-Myers Squibb has taken another step towards achieving its blockbuster hopes for beta thalassaemia-associated anaemia therapy Reblozyl, after hitting the target in a phase 2 trial in patients who don’t require regular blood transfusions.  Reblozyl (luspatercept) is already approved to treat thalassaemia patients who depend on transfusions, but getting approval in patients with milder disease […]

  • Zynteglo halt re-ignites viral vector safety concerns; analysts

    Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface.  That’s the view of analysts at Jefferies, who suggest it could “re-ignite concerns over the […]

  • bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

    It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. NICE is assessing Zynteglo (betibeglogene autotemcel), a one-off gene therapy for the condition, which can have life-threatening consequences and is associated with a curtailed […]

  • Bluebird trumpets long-term data from beta-thalassaemia gene therapy

    bluebird bio has presented long-term data from its Zinteglo one-time gene therapy for the blood disorder beta-thalassaemia, as the company continues talks with payers in Europe to bring the ultra-pricey treatment to market. The European Medicines Agency (EMA) has granted a conditional marketing authorisation for the drug that will be marketed as Zinteglo (betibeglogene autotemcel), […]

  • ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

    A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. The data with the CRISPR/Cas9 drug CTX001 is still very early-stage, coming from just seven thalassaemia patients and […]