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gene-editing

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

Bluebird bio’s sickle cell gene therapy shows early-study benefit

When patients have copies of the functional gene, their red blood cells can produce anti-sickling haemoglobin which decreases the amount of the faulty sickled haemoglobin. ... The partners recently revealed initial positive data from the first two

Latest news

Vertex reveals promising data from first CRISPR-treated patients

The CRISPR/Cas-9 method of gene editing allows precise alterations to DNA sequences of living cells on demand. ... Vertex paid CRISPR Therapeutics $105m upfront in 2015, with up to $420m in milestone payments, to tap into its gene-editing expertise.
NIH, Gates pledge $200m for HIV and sickle cell gene therapies

Aim to bring therapies to patients in lower-income countries. The US National Institutes of Health (NIH) has joined forces with the Bill &Melinda Gates Foundation to develop gene-based therapies ... Earlier this year, scientists reported that they had
Vertex eyes type 1 diabetes cure with biotech acquisition

Exonics is developing gene-editing therapies for DMD and other genetic neuromuscular disease. ... That deal also focuses on gene-editing technology directed at DMD and DM1.
Bayer takes control of BlueRock stem cell joint venture

BlueRock was set up in late 2016 with $225m in start-up funding from Bayer and investment firm Versant, shortly after Bayer backed gene-editing specialist Casebia via its Leaps by
FDA starts speedy review of Novartis’ sickle cell antibody

Hope of a long-term cure for the disease lies with gene therapy, and there has been encouraging progress on that front as well. ... Meanwhile, a CRISPR-based gene-editing therapy is in development at CRISPR Therapeutics and Vertex, with trials of the

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Latest Intelligence

Bluebird: on a mission to ‘recode’ the DNA of healthcare

Europe is the test ground for the gene therapy pioneer’s outcomes-based model, which could set the standard for the emerging sector. ... These include pipeline gene-editing therapies from Sanofi and luspatercept from Celgene to name but a few, although
Vertex: a vision of treating all CF patients worldwide draws closer

A gene therapy cure for all the world’s CF patients would be a truly remarkable achievement. ... These include alpha-1 antitrypsin deficiency and focal segmental glomerulosclerosis. It’s also working with CRISPR Therapeutics on phase 1/2 clinical
2019: CRISPR and therapeutic gene editing comes of age

Can gene-editing deliver safe and effective therapeutics for patients with intractable diseases? ... Meanwhile, CRISPR isn’t the only gene-editing technology being used in human therapeutics, although its devotees claim it is the simplest, easiest and
Cutting through the noise: CRISPR

Gene editing as a therapeutic tool has ignited an explosion of excitement within the industry over the past few years: the science fiction concept of being able to simply remove and ... With an array of such tools in play, scientists seem to be on the
Bluebird Bio: on the cusp of a gene therapy revolution

That means gene products hitting that Glybera price of $1m are, for now at least, unlikely. ... CRISPR Therapeutics, one of the companies working with the exciting cutting-edge CRISPR/Cas9 gene editing technology, is looking to begin a phase I trial with

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Latest appointments

Eric Rhodes becomes CEO of ERS Genomics

In these roles, Rhodes was responsible for commercialising the company's genome editing technologies as well as driving its research and development. ... Biosciences. Foy said: “With his deep understanding of the gene-editing landscape and extensive
Marc Becker joins CRISPR Therapeutics

He becomes chief financial officer. CRISPR Therapeutics has appointed Marc Becker as its new chief financial officer with remit to drive development of its gene-editing technology platform.

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Latest from PMHub

Spotlight interview: 15 minutes on medical and scientific writing

A number of current therapeutic approaches – such as gene editing, gene therapy, and stem-cell therapy – were not in existence when many healthcare professionals (HCPs) underwent their training, so there is
'Fake news': battling misinformation in healthcare

After reading this headline, it’s easy to assume that some form of gene editing has led to a cure for HIV. ... Should you read on, you would also discover that gene-editing was in no way used in this treatment.
Random42 Partner with MedCity for BioJapan 2018

J. Leading UK health scientists, universities and national research institutions will provide insight into the UK life sciences sector, and their work in ageing, gene editing and artificial intelligence.
Training the next generation of biotech leaders

Novel technologies, coupled with programmes designed to drive cell and gene research and development, are ushering in a new era of therapies. ... It’s such a disruptive advancement – it’s just incredible to think it has all been made feasible
Perspective on biotech

The emerging technologies revolutionising the biotech industry – from immunotherapy breakthroughs to cutting-edge gene editing techniques.

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