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beta-thalassemia

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Novavax’s COVID-19 vaccine candidate receives CHMP recommendation for use as a booster

The protein-based vaccine induced a ‘robust antibody response’ when used as a heterologous third booster dose, data from a UK-sponsored trial showed

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Novo Nordisk to acquire Forma Therapeutics in deal worth $1.1bn

The acquisition will focus on expanding the company’s blood disorders portfolio

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Pfizer set to acquire Global Blood Therapeutics in deal worth $5.4bn

The acquisition will help accelerate research into sickle cell disease

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MHRA grants marketing authorisation to Global Blood Therapeutics for sickle cell disease drug

Oxybryta is the first treatment in Britain to directly block sickle haemoglobin polymerisation

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Novartis and Precision BioSciences announce in vivo gene editing collaboration

The companies will work together to develop potentially curative treatments for disorders including sickle cell disease and beta thalassaemia

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Positive CHMP opinion issued for PTC Therapeutics’ Upstaza

The treatment for AADC will be the first marketed gene therapy to be administered directly into the brain

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Biogen retracts EMA application for Alzheimer’s drug Aduhelm

The application was pulled after the CHMP said the data provided was not enough to support a marketing authorisation

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NICE recommends Novartis’ Adakveo for sickle cell disease

The treatment for sickle cell disease is the first in more than 20 years

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Vertex expands further into cell and genetic therapies with a deal worth up to $1.2bn

A new deal will see Vertex harness Arbor Biotechnologies gene-editing technology to enhance its discovery efforts

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bluebird bio to restart marketing of Zynteglo in EU after previous suspension

EMA’s safety committee PRAC has confirmed favourable risk-benefit balance of gene therapy

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bluebird bio lodges counterclaim against Spark Therapeutics

Spark previously accused bluebird of misusing trademarks relating to the word ‘spark’ in marketing/educational materials

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MHRA grants bluebird bio’s LentiGlobin gene therapy an ‘innovation passport’

The Innovative Licensing and Access Pathway scheme aims to help speed approval of innovative treatments

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