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Haemophilia

Five recent cell and gene therapy discoveries

27 December 2022 | By Izzy Wood (Drug Target Review)

This article highlights five of the latest findings using cell and gene therapy techniques that could be used in the development or design of new therapies.

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Complete DNA sequence of the X chromosome revealed

15 July 2020 | By Hannah Balfour (Drug Target Review)

Researchers capitalised on novel sequencing technologies to produce the first end-to-end DNA sequence of the X chromosome.

article

CRISPR: kick-starting the revolution in drug discovery

13 December 2019 | By Dr Pushpanathan Muthuirulan (Harvard University)

Gene editing using the CRISPR system has been established as the most powerful tool in the search for new drugs and is now being exploited for therapeutic purposes. Here, Pushpanathan Muthuirulan discusses the promises and wider opportunities of using CRISPR technology to open up the possibility of large-scale screening of…

Groundbreaking gene therapy trial set to cure haemophilia

news

Groundbreaking gene therapy trial set to cure haemophilia

14 December 2017 | By Drug Target Review

A cure for haemophilia is one step closer, following results of a gene therapy trial led by the NHS in London.

news

Microcapsules to enhance the efficiency of genome-editing

6 October 2017 | By Dr Zara Kassam (Drug Target Review)

Researchers have found that polymer and hybrid silica-coated microcapsules are efficient in genome-editing...

news

Apitope raises €12 million in Series B funding

29 September 2015 | By Victoria White

The funds will enable Apitope to progress the clinical development of its innovative pipeline which includes therapies for Factor VIII intolerance in Haemophilia A patients...

news

FDA Grants Orphan Status for ATX-F8-117 for the treatment of haemophilia A patients in the US

9 June 2015 | By Victoria White

Apitope’s product candidate ATX-F8-117 has been granted Orphan Drug Status by the FDA for the treatment of patients with haemophilia A in the US...

news

Alnylam and collaborators publish preclinical results on ALN-AT3 for the treatment of haemophilia

14 April 2015 | By Victoria White

Preclinical results on ALN-AT3, an investigational RNAi therapeutic targeting AT for the treatment of haemophilia and RBD, have been published...

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