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The North Carolina-based company will expand Pfizer's capabilities in gene therapy research
Pfizer has acquired Bamboo Therapeutics, a privately held biotechnology company based in Chapel Hill, N.C., 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 significantly expands Pfizer’s expertise in gene therapy by adding clinical and several pre-clinical 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 a promising investigational treatment, 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 pre-clinical asset for Duchenne Muscular Dystrophy (DMD); and central nervous system, with pre-clinical 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 produced Phase I/II materials using a suspension, cell-based production platform. 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. Pfizer previously acquired approximately 22 percent of Bamboo’s fully diluted equity during the first quarter of 2016 for a payment of approximately $43 million.
Pfizer is committed to becoming an industry leader in the field of gene therapy. In 2014, Pfizer established within the company’s Rare Disease Research Unit the Genetic Medicines Institute (GMI) in London, UK, which is a dedicated gene therapy research group under the direction of leading gene therapy researcher Michael Linden. Dr. Linden and the GMI are charged with identifying potential gene therapy projects and supporting them through scientific discovery, process development and translational advancement.
In addition to the London-based GMI investment, Pfizer and Philadelphia-based Spark Therapeutics established a collaboration on a clinical program for SPK-9001, which is being investigated as a one-time treatment for hemophilia B that incorporates a bio-engineered rAAV vector. Initial data from an ongoing Phase I/II trial for this treatment has shown promising early results, and SPK-9001 has received breakthrough therapy designation by the U.S. Food and Drug Administration.
Pfizer has research agreements with several leading academic institutions, including an agreement with King’s College London for the development of a series of rAAV gene therapy vectors, and an agreement with the University of Iowa Research Foundation for the development of a potential gene therapy for cystic fibrosis through the University of Iowa laboratories.
Pfizer has also entered into a collaboration and license agreement with Emeryville, Calif.-based 4D Molecular Therapeutics (4DMT) to discover and develop targeted next-generation rAAV vectors for cardiac disease. In addition, Pfizer made an equity investment in 4DMT in October 2015.