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Pfizer's $150m Gene Therapy Bamboo Shoot

So far the only big pharma group to have pursued gene therapy to the point of regulatory success is GlaxoSmithKline (NYSE:GSK). But Pfizer (NYSE:PFE) is redoubling its efforts, paying $150m up front for the 78% of Bamboo Therapeutics it did not already own.

This is despite the company, a spin-out from Asklepios BioPharmaceutical, not yet having any clinical-stage products. The deal is also notable for its rarity in this space. It appears that the Bamboo deal is the first time a big pharma has acquired a pure-play gene therapy company.


The focus of the deal is BMB-D001, Bamboo’s gene therapy for Duchenne muscular dystrophy, which is scheduled to enter a phase I/II trial by the end of the year. BMB-D001 is a recombinant adeno-associated viral (rhAAV) vector that delivers a shortened but functional copy of the dystrophin gene.

It also has pre-clinical gene therapies for Friedreich’s ataxia and the neurological disorder Canavan’s disease. The Friedreich’s programme uses an rhAAV vector to deliver a functional copy of the frataxin gene to patients with the movement disorder.

The Canavan’s therapy has been in development for some years, according to Bamboo’s website; an initial clinical trial of an AAV-delivered ASPA gene – which is mutated in Canavan’s patients – was begun in 2001. The product was directly injected into the brains of 13 patients between 2001 and 2005.

Five-year data showed decreased brain atrophy, improvement in several areas of...