NEWPORT BEACH, Calif.--(BUSINESS WIRE )-- CureDuchenne  Ventures LLC, a venture philanthropy organization that funds research to find a cure for Duchenne muscular dystrophy, announced today that they have invested in Bamboo Therapeutics, Inc. , an early-stage biotechnology company dedicated to developing gene therapies for orphan diseases. The development of Bamboo's Duchenne gene therapy was made possible by more than 30 years of groundbreaking scientific research performed by Jude Samulski, PhD , and Xiao Xiao, PhD , at the University of North Carolina, Chapel Hill. Utilizing a proprietary rAAV technology platform developed by Dr. Samulski, Bamboo's gene therapy will seek to deliver a mini-dystrophin gene sequence developed by Dr. Xiao that can be used to treat all Duchenne patients.
The gene therapy, initially called BMB-D001, has shown positive effects in Duchenne animal models including improved life span as well as skeletal and cardiac muscle function in both mice and rats. In a canine Duchenne animal model, long-term dystrophin production resulted in life extension and maintenance of ambulation up to eight years post-treatment. CureDuchenne Ventures' financial support will enable critical pre-IND enabling studies to accelerate advancement of the therapy to patient clinical trials.
"We have dedicated our careers to harnessing the power of gene therapy to correct devastating genetic diseases," said Dr. Samulski, Scientific Founder and CSO of Bamboo Therapeutics. "We are excited to collaborate with CureDuchenne as we move our therapeutic into the clinic."
"We are very excited and appreciative for the generous support of CureDuchenne," said Dr. Xiao. "It is a vote of confidence in our 20 years' devoted work in Duchenne gene therapy. This funding is timely and crucial in accelerating the translation of lab discoveries to an effective therapy for all Duchenne patients."
"We are honored to have the support of Debra Miller and CureDuchenne as we aggressively advance our therapeutic for the benefit of boys suffering from Duchenne and their families," said Sheila Mikhail , Chief Executive Officer of Bamboo Therapeutics. "Together, we are a highly motivated, powerful force."
Duchenne  is a fatal genetic disease that causes muscles to degenerate. It impacts approximately 1 in 3,500 boys. Those with Duchenne are usually diagnosed by age 5, lose their ability to walk by 12 and most don't survive their mid-20s. There is currently no approved treatment or cure for Duchenne.
"Duchenne is a progressively debilitating disease and we believe it is critical to support the next-generation advances that may lead to a cure," said Debra Miller, president of CureDuchenne Ventures. "We are delighted to partner with Bamboo and their illustrious scientific team to hasten development of a gene therapy treatment for Duchenne."
In addition to financial support, Jak Knowles, Managing Director of CureDuchenne Ventures, will join the Bamboo Board of Directors in an observational and advisory capacity.
About CureDuchenne Ventures
CureDuchenne Ventures LLC collaborates with pharmaceutical and biotechnology companies to facilitate the development of drugs to treat Duchenne muscular dystrophy. CureDuchenne Ventures LLC was formed by CureDuchenne, a national nonprofit that has funded seven research projects  that have advanced to human clinical trials. CureDuchenne has leveraged $100 million in pharma and biotech research and development investments . Now three of the projects CureDuchenne supported with funding, BioMarin Pharmaceutical, Sarepta Therapeutics and PTC Therapeutics, are the closest to becoming the first drugs to be approved for the treatment of the disorder. CureDuchenne has been working to treat the whole disease  with a multi-pronged approach to find treatments for the many effects that Duchenne has on the body.
CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, three pharmaceutical treatments could be approved by the FDA within the next year. These treatments may lessen the effects of the disease for those with certain mutations of Duchenne, but there is still much to be done to find a cure. For more information, please visit CureDuchenne.org  and follow us on Facebook , Twitter  and YouTube .
About Bamboo Therapeutics, Inc.
Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from debilitating disease. Bamboo is focused on some of the most devastating neurological and neuromuscular diseases that affect children. Our lead therapeutic is a gene therapy product for the treatment of Duchenne muscular dystrophy. Bamboo's therapeutics are derived from an exclusive rAAV platform technology, and are manufactured using a proprietary scaled up process at an established GMP vector core facility recently purchased by the company.
View source version on businesswire.com: http://www.businesswire.com/news/home/20160110005016/en/  CureDuchenne Source: CureDuchenne Ventures LLC
Karen Harley, 949-872-2552
View source version on businesswire.com: http://www.businesswire.com/news/home/20160110005016/en/ 
Source: CureDuchenne Ventures LLC