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Biogen Idec taps Sangamo for blood disorder drugs

Deal is worth up to $320m
Biogen Idec building

Biogen Idec has signed a deal worth up to $320m with Sangamo Biosciences focusing on the development of new drugs for blood disorders.

Biogen will pay $20m upfront to Sangamo in order to apply the latter's gene therapy technology to the development of new therapies for red blood cell disorders such as sickle cell disease (SCD) and beta thalassaemia, and up to $300m in development and commercialisation milestones.

The diseases - collectively known as haemoglobinopathies - are both inherited conditions that result from the abnormal structure or underproduction of haemoglobin, reducing the amount of oxygen that can be carried in the blood. There are estimated to be around 90,000 people with SCD in the US and another 2,000 with beta thalassaemia.

The collaboration will focus on using Sangamo's zinc finger nuclease (ZFN) genome-editing technology, which can be used to switch off a defective regulator sequence for a gene or replace a defective gene with a functional version.

At the moment, SCD and beta thalassaemia can be effectively cured using bone marrow transplantation from a matched donor, but the approach is limited by a scarcity of donors and the risk of graft-versus-host disease (GVHD), which occurs when the donated immune system starts mounting a response against the recipient.

Meanwhile, supportive therapies are also available, notably Novartis' Exjade (deferasirox) which was approved in the EU last year to manage iron overload in thalassaemia.

Sangamo has already been awarded a $6.4m grant from the California Institute for Regenerative Medicine (CIRM) to develop a therapeutic for beta-thalassemia and presented preclinical data from that programme at the American Society of Hematology meeting in New Orleans last month. It is now gearing up to start trials of the potentially curative approach.

The US biotech will be solely responsible for the development of the SCD and thalassaemia therapies through proof-of-concept studies, with Biogen taking over clinical development, registration and commercialisation activities thereafter. Sangamo retains a co-promotion option in the US.

Other companies looking at gene therapeutics for haemoglobinopathies include Alnylam, which has an RNA interference therapeutic in preclinical development, and Bluebird Bio whose LentiGlobin gene therapy candidate has reached phase I/II trials.

Article by
Phil Taylor

10th January 2014

From: Research



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