September 13, 2016
Children born with Canavan disease – a fatal childhood neurological condition that usually causes death by age 10 – may one day lead a longer and healthier life thanks to Dr. Paola Leone, of the Rowan University School of Osteopathic Medicine.
Internationally, she is recognized as a pioneer of gene therapy directed to the central nervous system.
At RowanSOM, where she is a professor and director of the Cell and Gene Therapy Center in the Department of Cell Biology, she is known as a tireless researcher committed to discovering treatments for conditions such as Canavan disease, amyotrophic lateral sclerosis, Alzheimer’s disease, multiple sclerosis and traumatic brain injury.
A first
Most notably, she and her team have administered to the brain, for the first time, a genetically modified virus that expressed the healthy gene that is otherwise mutated in Canavan disease, a process that may change – and save – lives.
That has an even greater chance than ever now that Pfizer Inc., the New York City-headquartered corporation that is one of the world’s premier biopharmaceutical companies, is part of the picture and one day may produce therapeutics based on her work.
Commercialization
In July 2015, Rowan University entered into an asset transfer agreement with Bamboo Therapeutics, Inc. to commercialize a novel gene therapy developed by Leone and her team in the Cell and Gene Therapy Center in Stratford, New Jersey, for the treatment of Canavan disease, one of the most common and complex degenerative cerebral diseases in infants.
Bamboo Therapeutics, a clinical-stage company based in Chapel Hill, North Carolina, that develops gene therapies for serious neurological genetic diseases, acquired the exclusive worldwide rights to develop and commercialize Rowan University’s proposed treatment for Canavan disease.
The Cell and Gene Therapy Center was the first to demonstrate the long-term safety and benefit of a virus-based gene therapy in a clinical setting, and the study represented the first gene therapy study for a clinical neurological application ever approved by the National Institutes of Health as an Investigational New Drug application.
Acquired by Pfizer
Recently, Pfizer acquired Bamboo Therapeutics for $150 million and potential milestone payments in a deal to expand the drug giant’s presence in the experimental field.
According to Pfizer, 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.”
The corporation noted, “Gene therapy – a growing and experimental technology (whose) 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.”
“Validation of potential”
“The acquisition of Bamboo Therapeutics by Pfizer is a validation of the potential of gene therapy to provide viable therapeutic strategies for currently intractable diseases. The contribution of the Cell and Gene Therapy Center at RowanSOM to this partnership is the result of many years of research effort, institutional commitment and patient advocacy for this therapeutic platform,” Leone said. “It is especially pleasing to see recognition of the value of translational research for Canavan disease in contributing to the bigger picture so tirelessly and passionately promoted by affected patients and their families during the last 20 years.”
Bamboo will be eligible for additional payments of as much as $495 million if the company’s therapies clear various testing and regulatory benchmarks, Pfizer noted.
First sale of equity
Leone’s intellectual property was one of seven licenses Pfizer acquired; the others are owned by the University of North Carolina – Chapel Hill. Rowan, UNC, and the University of Pennsylvania are widely recognized university leaders in gene therapy. This is the first time Rowan has sold equity in a firm.
“We were able to strategically identify a company that was favorably positioned for this kind of acquisition eventually by a large pharmaceutical company,” said Mina Zion, director of Technology Commercialization at Rowan. “We cannot imagine a better commercialization path than for our technology to pass through the hands of Pfizer.”
Rowan is sharing in the commercialization income and also received funding to promote research discovery and development focusing on treatments for neurodegenerative diseases. The University also will receive royalties on the sale of treatments that arise from the work done by Leone, who earned a Ph.D. in neuroscience from the University of Padua in Italy.
Major step for Rowan
“This is the first intellectual property by a Rowan University faculty member to be commercialized by a major pharmaceutical company,” said Dr. Shreekanth Mandayam, vice president for Research.
Dr. Ali Houshmand, Rowan’s president, said, “This is a major step for Rowan University as we continue to grow our research and innovation initiatives. Most importantly, Dr. Leone’s work and affiliation with Bamboo – and Pfizer’s recent purchase – have the potential to make a huge medical impact that’s going to change people’s lives.”