Pfizer acquires gene therapy developer Bamboo Therapeutics in a $645 mn deal

US pharmaceutical giant Pfizer Inc yesterday said that it has acquired privately-held gene therapy developer Bamboo Therapeutics Inc in a deal potentially worth up to $645 million, in order to boost its portfolio of drugs in the treatment of rare diseases.

Under the terms of the transaction, Pfizer, which had earlier acquired around 22 per cent of Bamboo for $43 million, acquired all of Bamboo's remaining equity for an upfront payment of $150 million.

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 commercialisation.

Bamboo will now be a wholly-owned subsidiary of Pfizer.

''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.''

North Carolina-based Bamboo focuses 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.

Its portfolio includes potential recombinant Adeno-Associated Virus (rAAV) -based gene therapies that will complement Pfizer's rare disease and gene therapy portfolios in two 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.

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 was the first person to discover the potential of using the adeno-associated virus as a vehicle to replace a defective gene with a healthy gene.

Pfizer said that the acquisition will expand its expertise in gene therapy by providing it with a clinical and several pre-clinical assets that complement the company's rare disease portfolio, an advanced rAAV.

Gene therapy is an emerging area of medical research focused on highly specialised, one-time, transformative treatments addressing the root cause of diseases caused by genetic mutation. It 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.

Although there are over 7,000 known rare diseases, only five per cent have an approved medication, but there have been no gene therapy products approved in the US to date.

''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.

Pfizer has a pipeline of more than 20 compounds and a global portfolio of more than 20 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, and pulmonology.

Pfizer established its Rare Disease Research Unit, the Genetic Medicines Institute in the UK in 2014, and established a collaboration with Philadelphia-based Spark Therapeutics 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.

It also has research agreements with several leading academic institutions, including one with King's College London for the development of a series of rAAV gene therapy vectors, and 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 also entered into a collaboration and license agreement with California-based 4D Molecular Therapeutics to discover and develop targeted next-generation rAAV vectors for cardiac disease.