Pfizer is bolstering its growing gene therapy portfolio with a $150 million acquisition of Bamboo Therapeutics, the latest startup based on the research of one of the field’s pioneers, University of North Carolina Gene Therapy Center director Jude Samulski.
Pfizer (NYSE: PFE) initially signaled its interest in Bamboo’s work in the first quarter, when it paid $43 million for a 22 percent stake in the Chapel Hill, NC-based biotech company, an investment that Pfizer didn’t reveal until today. Now, the pharma giant has paid $150 million to buy the rest of Bamboo, whose shareholders could receive as much as $495 million more if the company’s experimental gene therapies for neuromuscular and central nervous system hit a variety of milestones.
Gene therapy aims to provide long-lasting, if not permanent treatments for genetic diseases with a single infusion. The technology has gone through a roller-coaster ride over three decades, but could finally be close to producing its first FDA-approved product, a drug for a genetic form of blindness from Spark Therapeutics (NASDAQ: ONCE), adding to the two gene therapies from UniQure and GlaxoSmithKline (NYSE: GSK), which are already approved in Europe. Bamboo’s gene therapy technology is based on the research of scientific founder and chief scientific officer Samulski, who runs UNC’s gene therapy center. According to Bamboo, Samulski, more than 30 years ago, was the first to realize the potential of the adeno-associated virus (AAV) as a delivery tool for gene therapy. AAV has since become the most commonly used delivery vehicle, or viral “vector,” for gene therapy. Bamboo’s experimental programs use the virus as well.
Bamboo’s most advanced gene therapy candidate is for giant axonal neuropathy (GAN), a rare genetic disorder that leads to numbness in the arms and legs, trouble walking, rapid back and forth movement of the eyes, and impaired cognitive development, according to the National Institute of Neurological Disorder and Stroke. Bamboo is currently enrolling patients in a Phase 1 trial for the GAN treatment.
Bamboo also has preclinical gene therapies for Canavan disease, a neurological disorder, and the muscular disorders Friedreich’s Ataxia and Duchenne Muscular Dystrophy. Bamboo expects to file papers with the FDA in the second half of 2017 to begin the first trial for its Duchenne candidate. But Bamboo isn’t the only company pursuing a gene therapy for Duchenne. Cambridge, MA-based Solid Biosciences also has a Duchenne gene therapy program in preclinical testing and has drawn an investment from Biogen. And Voyager Therapeutics (NASDAQ: VYGR) and Adverum Biotechnologies (NASDAQ: ADVM), the company formerly known as Avalanche Biotechnologies, are both developing gene therapies for Friedreich’s.
Bamboo was not Samulski’s first gene therapy company. His research led to the 2010 founding of Chapel Hill-based Chatham Therapeutics, which was acquired by Baxter International (NYSE: BAX) four years later for $70 million. Chatham’s gene therapy work focused on hemophilia, a program that is now in development at Shire, which acquired Baxter’s drugmaking spinoff, Baxalta (NYSE: BXLT), last year. Others pursuing gene therapies for hemophilia include Spark, UniQure, and two Cambridge, MA-based companies, Dimension Therapeutics (NASDAQ: DMTX), and Biogen (NASDAQ: BIIB), which earlier this year announced plans to spin out its hemophilia work into a separate company. Xconomy last week wrote about the progress to date in developing gene therapy treatments for hemophilia, citing the latest updates from the World Federation of Hemophilia World Congress.
Pfizer, meanwhile, has been steadily building up a gene therapy presence. It already has a partnership with Spark surrounding SPK-9001, the Philadelphia company’s experimental hemophilia B treatment. Over the past two years, the company has also formed a gene therapy group in London at its Genetic Medicines Institute based in London, and formed academic partnerships with King’s College London and the University of Iowa. Pfizer also has an alliance with Emeryville, CA-based 4D Molecular Therapeutics to develop next-generation AAV vectors for heart diseases.
With the acquisition, Samulski will now join Pfizer and Bamboo will operate as a Pfizer subsidiary. The deal also brings to Pfizer an 11,000-square-foot manufacturing facility that Bamboo acquired from the University of North Carolina at Chapel Hill earlier this year. Bamboo makes AAV viruses at the facility.