Sanofi’s voyage into gene therapy grows with CNS deal


It is not often that a venture-backed company goes from launch to $100m deal in the space of a year, but that is the feat Voyager Therapeutics pulled off with its partnership with Sanofi over four early-stage gene therapy projects.

The deal underscores the mounting interest in clinical approaches like gene and cell therapies that were long viewed as too risky for investors or big pharma partners. Last week saw Spark Therapeutics' enthusiastically received float on Nasdaq, and others will surely follow if gene therapies score more trial wins.

Heavy on CNS

The deal comprises $65m in cash, $30m in equity, and in-kind contributions, and gives the French group’s Genzyme division the option to license degenerative central nervous system disorders after completion of proof-of-concept trials. Potential milestones put the value of the collaboration at $845m before any royalties on sales.

Voyager’s lead project is for Parkinson’s disease. VY-AADC01 is intended as an add-on treatment to levodopa that improves the body’s ability to convert the mainstay drug into dopamine by stimulating production of an enzyme called aromatic L-amino acid decarboxylase. This is a therapy approach with which Genzyme experimented nearly a decade ago.

Sanofi’s option will allow it to license ex-US rights in Parkinson’s, along with a preclinical Friedreich's ataxia candidate and an undisclosed CNS programme. The preclinical Huntington’s disease research project coded VY-HTT01 is to be co-commercialised in the US, with a full licence to Sanofi elsewhere.

The two companies’ announcement states that Voyager will “drive research and development activities for all programmes”, suggesting that no funding will come from Sanofi. The company did not respond to a request to clarify the R&D funding arrangement by deadline; nevertheless, $65m in cash postpones any immediate need to tap its investors for a new funding round to finance clinical work.

Big pushoff

Those investors, which included Third Rock Ventures, made Voyager’s $45m initial financing the third-largest A round of 2014, which could help explain the short interval between launch and significant deal making.

With gene therapy having suffered early setbacks, a renaissance has been sparked not just by approval of UniQure’s Glybera in Europe but also by the furious interest in CAR-T therapies – which use genetically modified immune system cells to combat cancer (Therapeutic focus – Gene therapy slowly rises from the ashes, February 26, 2014).

Novartis is one big pharma active in this space, while AstraZeneca has entered in a big way the gene-editing technology field called CRISPR. Separately, Bluebird Bio's beta-thalassaemia data presented at December's ASH conference gave the gene therapy space a major push forward.

In addition to CNS, Sanofi also has put down a marker in ophthalmological treatments in an earlier tie-up with Oxford BioMedica. The size of the Voyager deal is yet another sign that the pall hanging over gene therapy might be lifting. The sector will now be looking to a phase III readout later this year for Spark’s own eye-disease project as a sign that gene therapy has worked out the bugs.

To contact the writer of this story email Jonathan Gardner in London at or follow @JonEPVantage on Twitter

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