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Gene therapy

Personalised medicine, batch size of ONE, the new challenge to fill-finish?

29 June 2017 | By Wenzel Novak PhD | Market Development Director – Pharma | Optima Machinery Corporation

Wenzel Novak PhD gives some considerations on container, environment, process and automation for small batch sizes in the downstream process of fill-finish…

news

Collaboration is ‘game changer’ in delivering cell and gene therapies

19 June 2017 | By Niamh Marriott (European Pharmaceutical Review)

Collaboration between the pharmaceutical industry, patients and health regulators will be a ‘game changer’ in delivering exciting new cell and gene therapies to treat conditions like cancer and vision loss.

news

Abeona Therapeutics receives FDA orphan designation for gene therapy

26 May 2017 | By Niamh Marriott, Junior Editor

The FDA has granted Orphan Drug Designation for Abeona Therapeutics’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa...

article

G is for Gene

22 November 2016 | By Niamh Louise Marriott, Digital Content Producer

G is for Gene - the next instalment of our daily pharmaceutical alphabet bringing you up to date with all the latest gene research...

news

Sanfilippo type A Gene therapy trial shows biopotency signals, provides Abeona Therapeutics

2 August 2016 | By Abeona Therapeutics Inc

Abeona Therapeutics provides an update on its first Sanfilippo patient and reports that early biopotency signals suggest a reduction of heparan sulfate in the urine of patients and if confirmed will suggest that intravenous administration of ABO-102 may ultimately provide both central nervous system and systemic benefits to patients...

news

Stem cell gene therapy for Sanfilippo disease ready for human trial

11 May 2016 | By Victoria White, Digital Content Producer

University of Manchester and CMFT researchers are to work with Orchard Therapeutics to test a stem cell gene therapy for Sanfilippo disease in a human trial...

article

Trends to transform healthcare in 2016

10 March 2016 | By Victoria White, European Pharmaceutical Review

In the latest issue of European Pharmaceutical Review, the Kaiser Associates European Healthcare team predict four trends to transform healthcare in 2016. We've created an infographic highlighting their key predictions...

article

European Pharmaceutical Review’s top 10 stories from 2015

28 January 2016 | By Victoria White (European Pharmaceutical Review)

Mega mergers, 3D printed drugs, a call for action on antimicrobial resistance: 2015 certainly was an interesting year for the pharmaceutical industry. Here we pick a selection of the top stories that hit the headlines.

news

Gene therapy offers hope for cystic fibrosis patients

3 July 2015 | By Victoria White

A therapy that replaces the faulty gene responsible for cystic fibrosis in patients' lungs has produced encouraging results in a major UK trial...

news

Global gene therapy deals sky-rocketed to nearly $5 billion in 2014, says GlobalData

26 June 2015 | By Victoria White

The number of deals in the global gene therapy market more than doubled in 2014, with their combined value rising spectacularly to $4.9...

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Gene therapy