• Pharmaceutical giant gains technology, drugs in development
  • Bamboo shareholders may get up to $495 million in milestones

Pfizer Inc. said Monday that it acquired Bamboo Therapeutics Inc., a biotechnology firm developing gene therapies, for $150 million and potential milestone payments in a deal to expand the drug giant’s presence in the experimental field.

Closely held Bamboo is developing treatments for neuromuscular conditions and central nervous system disorders. Pfizer said in a statement that it will take over the company’s treatments in development, which haven’t yet been tested in people, as well as Bamboo’s manufacturing facility. Bamboo’s most advanced drug, for a rare nerve-cell disorder called giant axonal neuropathy, is enrolling patients for its first human trial. It is also working on a treatment for Duchenne muscular dystrophy, a rare and deadly muscle disease.

Gene therapy -- a growing and experimental technology that’s backers hope can be effective after only one treatment -- treats diseases caused by a genetic mutation by giving the patient a working copy of a missing or dysfunctional gene. No gene therapy drugs have been approved in the U.S., though many are being tested.

Along with the up-front payment, Chapel Hill, North Carolina-based Bamboo will be eligible for additional payments of as much as $495 million if the company’s therapies clear various testing and regulatory benchmarks.

Gene Therapy Investments

Pfizer, based in New York, has been investing in gene therapy in recent years, and in 2014 it created a research group in London to identify potential gene therapy projects. It also has a partnership with Spark Therapeutics Inc. to develop a treatment for the blood disorder hemophilia B.

“The field of gene therapy research has made tremendous strides in recent years, and we are pleased to be able to further enhance our leadership position in this area through this transaction with Bamboo,” Mikael Dolsten, Pfizer’s head of research and development, said in the statement. “We believe that gene therapy holds the promise of bringing true disease modification for patients suffering from devastating diseases.”

Gene therapy relies on using a virus, known as a viral vector, to deliver a payload of functioning genes to a patient’s cells. Bamboo’s vectors are designed to improve delivery efficiency, Pfizer said in the statement.

The Pfizer deal “represents a significant step toward bringing Bamboo’s portfolio into the clinic and, ultimately, potential new medicines to patients,” said Jude Samulski, Bamboo’s chief scientific officer and a co-founder of the company, who will stay on with Pfizer.

Samulski was the first person to realize the potential to use the adeno-associated virus as a vehicle to replace a defective gene with a healthy gene, according to Bamboo’s website. He’s the director of the Gene Therapy Center at the University of North Carolina, according the center’s website.

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