Developing gene therapies for rare and devastating central nervous system and neuromuscular diseases

Bamboo was formed to advance the work of Dr. Richard Jude Samulski, the Director of the Gene Therapy Center at the University of...
See More North Carolina (UNC), from the laboratory into human clinical trials where it could meet the requirements for drug approval. Dr. Samulski is a pioneer in the field of gene therapy. Over thirty (30) years ago, Dr. Samulski was the first person to realize the potential to use the adeno-associated virus (AAV) as a vehicle to replace a defective gene with a healthy gene. Since that time, he has re-engineered the naturally occurring virus to target delivery to certain tissues, de-target other tissues, and improve its safety. This has resulted in over twenty (20) patents related to the use of AAV for therapeutic uses. Dr. Samulski continues to lead innovation in the field of vector optimization and AAV re-engineering.

Dr. Samulski, along with his first PhD student, Dr. Xiao Xiao (currently a tenured faculty member at UNC), were committed to using AAV to treat devastating diseases. Dr. Xiao has focused his research on the treatment of Duchenne Muscular Dystrophy(DMD). DMD is an extremely debilitating disease that leads to an inability to walk and eventually death. DMD results from a defect in one gene – the dystrophin gene. One difficulty in using AAV to treat DMD is that the dystrophin gene is the largest gene in the human body, and is too big to fit within the virus. Dr. Xiao resolved this problem by creating a miniaturized gene that fits within AAV and has the promise of the functionality of the full-length dystrophin gene. This gene was tested in a limited human clinical trial supported by the Muscular Dystrophy Association (MDA). This gene has been combined with proprietary rAAV technology developed by Dr. Samulski, and is expected to advance into human clinical trials in 2017.

Based on the work being done in DMD, the rAAV technology used by Bamboo is a platform for the treatment of other neurological and neuromuscular diseases. Bamboo, in conjunction with Dr. Paola Leone of Rowan University, is using the technology for the treatment of Canavan disease. Bamboo is also working on treatments for Friedreich ataxia and giant axonal neuropathy (GAN).

We are unique among gene therapy companies in that we have invested in manufacturing and processes. We acquired the Vector Core facility from UNC in January 2016. This facility is well known and respected in the industry, and has a track record of producing clinical grade material for big pharmaceutical companies. Having a captive manufacturing facility shortens Bamboo’s timelines for product development, and enhances the efficacy and performance of our drugs.

We have a strong management team, experienced in the gene therapy area, with a deep understanding of the regulatory process. We have strong ties to foundations and patient advocacy groups focused on the diseases that we are treating.