Bamboo Therapeutics, Inc., a biotechnology company focused on developing gene therapies for rare central nervous system and neuromuscular diseases, including Friedrich’s ataxia, announced that it has acquired the University of North Carolina (UNC) at Chapel Hill’s viral Vector Core gene therapy manufacturing facility. Financial details of the deal have not been disclosed.
Viral vectors are tools, tailored to specific applications, commonly used by molecular biologists to deliver genetic material into cells. An example is their use in the development of therapies to correct defective genes that cause certain diseases.
Founded in 1993 as a full-service viral vector production organization with experience in vector design and process development, UNC’s Vector Core manufactures research- and clinical-grade vectors at its 11,000-square-foot facility. The vectors are for use by pharmaceutical and biotechnology companies, universities, and foundations.
Bamboo and the Vector Core have developed a proprietary suspension cell-based production platform that utilizes serum-free media to increase scalability, efficiency, and purity. The company says the system decreases the number of processing steps necessary in viral vector production methods using adherent cells, while also reducing the variability and shortcomings associated with the use of animal serum, providing its clients with superior vectors at a lower cost.
“We believe that having a leading manufacturing facility fully integrated into our business provides flexibility and a competitive advantage,” says Dr. Jude Samulski, scientific founder and chairman of Bamboo Therapeutics, in a release. “We anticipate rapidly moving our programs forward, including our DMD [Duchenne muscular dystrophy] program, which is expected to enter the clinic in early 2017.”
Bamboo Therapeutics, based in Chapel Hill, N.C., was formed to advance the work of Dr. Samulski, who is also director of the University of North Carolina (UNC) Gene Therapy Center and a pioneer in the gene therapy field. Over 30 years ago, Dr. Samulski was the first to realize the potential use of the adeno-associated virus (AAV) as a vehicle to replace a defective gene with a healthy gene. Since then, he has re-engineered naturally occurring viruses to target delivery to certain tissues, de-target other tissues, and has improved the technique’s safety. This research and development has resulted in more than 20 patents related to the therapeutic use of AAV. Dr. Samulski continues to advance in the field of vector optimization and AAV re-engineering.
Based on the work being done in Duchenne muscular dystrophy (DMD), the recombinant adeno-associated virus (rAAV)-based genome engineering technology used by Bamboo is also a potential platform to treat other neurological and neuromuscular diseases, with the company’s pipeline focused on diseases of high unmet need, including Friedreich’s ataxia, giant axonal neuropathy (GAN), Canavan disease, and DMD.
Friedreich’s ataxia is one of a group of rare, progressive and often fatal genetically-based degenerative neurological disorders that affect an estimated 150,000 people in the United States, with symptoms that often impact coordination, hearing, and vision. Hereditary ataxias are caused by a defect in a certain gene present at birth. More information on ataxia is available at the National Ataxia Foundation’s website: www.ataxia.org
In collaboration with Dr. Paola Leone, a professor of Cell Biology at Rowan University, in Stratford, New Jersey, Bamboo is also applying the technology to develop a treatment for Canavan disease. Described by The Canavan Foundation as a “progressive, fatal neurological disorder that begins in infancy,” Canavan disease is caused by an inherited genetic abnormality — lack of an essential enzyme that causes deterioration of the white matter (myelin) in the brain — that prevents the proper transmission of nerve signals.
Dr. Leone directs all research activities at Rowan University and affiliated centers. Her research interests have included in vivo neurochemistry of epilepsy and gene transfer approaches for the treatment of neurological disorders, and she is studying pharmacological approaches in humans and in animal models of Canavan disease.
Bamboo says it is unique among gene therapy companies in having invested in the manufacturing process through the recent acquisition of the Vector Core facility from UNC. The company says that having a manufacturing facility that is well-known and respected will shorten its product development timelines, and help enhance the efficacy and performance of the company’s drugs.
Bamboo’s most advanced program, a potential treatment for GAN, is currently in a Phase 1/2 trial.
For more information, visit bambootherapeutics.com/our-company.
Bamboo Therapeutics, Inc.
University of North Carolina (UNC) at Chapel Hill
University of North Carolina (UNC) Gene Therapy Center
The National Ataxia Foundation
The Canavan Foundation