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Pfizer acquires Bamboo Therapeutics

Deal brings gene therapy technology and key manufacturing capabilities

by Lisa M. Jarvis
August 5, 2016 | A version of this story appeared in Volume 94, Issue 32

Further bolstering its rare disease unit, Pfizer has paid $150 million to acquire gene therapy-focused Bamboo Therapeutics. The drugmaker, which earlier this year took a 22% stake in Bamboo, could shell out another $495 million to investors in the biotech if its gene therapies reach the market.

Bamboo was spun off in 2014 from Asklepios Biopharmaceutical, a biotech founded by three University of North Carolina, Chapel Hill, scientists with expertise in using adeno-associated viruses to deliver genes that teach cells how to make missing or improperly functioning proteins.

Through the purchase, Pfizer gains preclinical and clinical gene therapies, many of which complement the drug firm’s growing rare disease pipeline. For example, the deal brings Pfizer a therapy that could deliver a gene sequence to prompt the production of dystrophin, a protein missing in people with Duchenne muscular dystrophy (DMD). Pfizer already has a myostatin inhibitor, which could promote muscle growth in DMD patients, in Phase II studies.

But the attraction of Bamboo is about more than just its gene therapy pipeline, says Greg LaRosa, chief scientific officer of Pfizer’s rare disease unit. “Most importantly, they can manufacture gene vectors in amounts you can actually provide for clinical trials.” For a disease such as DMD, “a lot of virus is going to be needed,” LaRosa points out.

Bamboo bought its vector manufacturing facility from UNC in January and expects to be able to start a clinical trial for its DMD therapy in the second half of 2017. The biotech firm has already produced a gene therapy for giant axonal neuropathy, a rare nervous system disease, that is being tested in a clinical trial run by the NIH.

Bamboo has received substantial support from rare disease advocacy groups. CureDuchenne Ventures, for example, was one of several investors in the biotech’s first major round of funding—for $49.5 million—in January.

Portfolio building

Pfizer has made multiple rare disease deals since creating a rare disease unit in 2010.

2010 Acquires FoldRx
2011 • Links with Zacharon for lysosomal storage diseases
• Licenses Glycomimetics' sickle cell treatment
2012 Joins with Cystic Fibrosis Foundation to support CF drug development
2013 Licenses Repligen's spinal muscular atrophy treatment (returned in 2015)
2014 • Acquires Opko Health's long-acting growth hormone
• Creates gene therapy pact with Spark Therapeutics
2016 Acquires Bamboo Therapeutics
Credit: Pfizer


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