Pfizer has acquired Bamboo Therapeutics, a privately held biotechnology company based in Chapel Hill, North Carolina, focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system.

This acquisition expands Pfizer’s expertise in gene therapy by providing Pfizer with a clinical and several preclinical assets that complement the company’s rare disease portfolio, an advanced recombinant Adeno-Associated Virus (rAAV) vector design and production technology, and a fully functional phase I/II gene therapy manufacturing facility that Bamboo acquired from the University of North Carolina earlier this year.

Gene therapy is an emerging area of medical research focused on highly specialized, one-time, transformative treatments addressing the root cause of diseases caused by genetic mutation. Gene therapy is a promising investigational technology, especially for patients with rare diseases, many of which are caused by a single genetic mutation. The technology involves introducing genetic material into the body to deliver a corrected copy of a gene to a patient’s cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.

“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,” said Mikael Dolsten, president, Pfizer Worldwide Research & Development. “We believe that gene therapy may hold the promise of bringing true disease modification for patients suffering from devastating diseases, and we hope to see this promise come to fruition—through new and existing in-house capabilities and potential partnership opportunities—in the years to come.”

Bamboo’s portfolio includes potential best-in-class rAAV-based gene therapies that will complement Pfizer’s rare disease and gene therapy portfolios in two priority areas: neuromuscular, with a preclinical asset for Duchenne Muscular Dystrophy (DMD); and central nervous system, with preclinical assets for Friedreich’s Ataxia and Canavan disease, and a phase I asset for Giant Axonal Neuropathy.

Bamboo’s approximately 11,000-square foot, fully staffed and operational manufacturing facility has experience producing phase I/II materials using a superior suspension, cell-based production platform that increases scalability, efficiency and purity. This helps enable the DMD program and other projects requiring large amounts of rAAV. The facility, previously known as the University of North Carolina Vector Core facility, has served as a qualified supplier of rAAV vectors for several healthcare companies and academic institutions.

“We believe Bamboo’s industry leading capabilities in rAAV vector design and manufacturing complement Pfizer’s rare disease strategy and help advance Pfizer’s mission to deliver life-changing innovation to patients with the greatest needs,” said Gregory LaRosa, chief scientific officer, Rare Disease Research Unit, Pfizer. “Bringing together Pfizer and Bamboo colleagues’ deep scientific understanding of both rAAV biology and complex biologic manufacturing will help position us for success in this area. We are pleased to welcome Bamboo colleagues to Pfizer and look forward to working together on transformative gene therapies for patients in areas of high unmet medical need.”

Jude Samulski, chief scientific officer and executive chairman of Bamboo, and a leading expert in the field of rAAV vectors with more than 25 years of experience, will be joining Pfizer. Samulski, together with the Bamboo team, will play a key role in helping to develop and accelerate Pfizer’s capabilities in gene therapy.

Pfizer previously acquired approximately 22% of Bamboo’s fully diluted equity during the first quarter of 2016 for a payment of approximately $43 million. Under the terms of this transaction, Pfizer acquired all of Bamboo’s remaining equity for an upfront payment of $150 million, and Bamboo’s selling shareholders will be eligible for potential milestone payments of up to $495 million contingent upon the progression of key assets through development, regulatory approval and commercialization. Following the acquisition, Bamboo is now a wholly-owned subsidiary of Pfizer.