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Big pharma investment in gene therapy continues with Sanofi deal

16 February 2015
Appeared in BioNews 790

Sanofi has signed a partnership deal worth potentially US $845 million with Voyager Therapeutics, a comparatively small biotech firm, becoming the latest in a series of major pharmaceutical firms to commit financially to gene therapy research.

According to a joint statement by the two companies, Genzyme, the Sanofi subsidiary involved in the deal, will work collaboratively with Voyager Therapeutics to develop and ultimately commercialise 'novel adeno-associated virus (AAV) gene therapies for Parkinson's disease, Friedreich’s ataxia, Huntington’s disease and other debilitating central nervous system disorders.

Dr Steven Paul, president and CEO of Voyager Therapeutics, says the alliance will ensure both companies 'are at the forefront of converting the promise of gene therapy into innovative therapies for CNS disorders that make a meaningful difference in patients' lives'.

According to the agreement, Voyager Therapeutics will oversee research and development of the specified gene therapy programmes, receiving an initial US $100 million upfront payment from Genzyme, including US $65 million in cash.

Should the programmes be successful and ultimately produce commercialised therapies, Voyager Therapeutics could receive up to US $745 million in 'development and sales milestones payments' and royalties.

Currently Voyager has two products - in Parkinson's disease and Friedreich's ataxia - in early clinical development, and others in preclinical trials.

Voyager Therapeutics will retain the rights to the Parkinson's and Friedreich’s ataxia programmes in the USA, and Genzyme will hold outside-US rights. The two companies plan to share any eventual US profits from the project in Huntington's disease. Genzyme will have licensing options for several other programmes after completion of proof-of-concept clinical trials.

Voyager's AAV gene therapies involve silencing disease-causing gene mutations or replacing them with healthy versions. The AAV viruses are used as vectors, delivering the repair genes to the target.

According to industry analysts FierceBiotech, the company is 'also at work on new viral delivery vehicles that could mark a major improvement in the field'.

In the last year, three other important pharmaceutical players have made similar collaborative deals with biotech firms to develop gene therapies.

Pfizer (see BioNews 784), Biogen Idec (BioNews 788) and Bayer Healthcare's collaborations, however, are all focused on haemophilia A and/or B.

Genzyme's interest in gene therapy goes back over twenty years, enduring a fallow period when the technique appeared to many too problematic to ever bear commercial fruit. But last year Glybera, produced by the Dutch biotech UniQure, became the first gene therapy product to go on sale in Europe (see BioNews 782).

'We really carried the torch for years when some other companies left the space,' Mark Barrett, Genzyme's vice president of business development, told FierceBiotech.

11 December 2017 - by Charlotte Spicer 
Scientists have successfully used gene therapy to prevent bleeding in a small trial of patients with the inherited blood disorder haemophilia B...
24 April 2017 - by Kulraj Singh Bhangra 
A million-dollar gene therapy treatment for a rare blood disease will be withdrawn from the European market due to lack of demand...
2 February 2015 - by Dr Lucy Freem 
Biogen Idec has made an investment deal to develop gene therapy treatments for haemophilia A and B....
15 December 2014 - by Sean Byrne 
Pfizer has entered into a deal with biotech company Spark Therapeutics to conduct research into gene therapy for haemophilia B...
1 December 2014 - by Arit Udoh 
Glybera, the first gene therapy to go on sale in Europe, is set to cost €1.1m (£870,000) per patient, making it the world's most expensive drug...
25 June 2012 - by Holly Rogers 
A single dose of an 'antisense' drug has been shown to slow, or even partially reverse, Huntington's disease in animal studies, according to a study published in Neuron....
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