Early Stage Investment by CureDuchenne Ventures, LLC Enabled Bamboo Therapeutics to Accelerate Development of a Gene Therapy for Duchenne Muscular Dystrophy

Today, Pfizer Inc. announced its acquisition of Bamboo Therapeutics in a deal worth up to $645 million. Bamboo Therapeutics is an early-stage biotechnology company developing therapies for rare neuromuscular and central nervous system diseases. Seven months ago, CureDuchenne Ventures, LLC invested in Bamboo Therapeutics, giving the Raleigh, NC-based start-up the capital needed to acquire a viral vector core facility from the University of North Carolina at Chapel Hill (UNC).

The vector core acquisition was critical for manufacturing adeno-associated virus (AAV) which Bamboo utilizes for pre-clinical proof of principle experiments and human clinical trials. Bringing AAV manufacturing in-house allowed Bamboo to become one of the first vertically integrated gene therapy companies, accelerating the company’s development efforts for neuromuscular diseases like Duchenne muscular dystrophy, and positioning the company for a Series A capital raise of $49.5 million in February, 2016.

Bamboo Therapeutics was formed to advance the work of Dr. Richard Jude Samulski, PhD, the Director of the Gene Therapy Center at UNC, a pioneer in the field of AAV gene therapy. Dr. Samulski teamed up with Dr. Xiao Xiao, PhD, a Professor of Gene Therapy at UNC, who developed a mini-dystrophin sequence that once encapsulated, can utilize AAV as a delivery vehicle to treat Duchenne patients who lack a functional dystrophin gene. Dr. Xiao has dedicated his 30-year research career to understanding Duchenne, a devastating rare genetic disease that deteriorates muscles, leaving patients unable to walk by their mid-teens. The average life expectancy of someone with Duchenne is mid-20s.

“The visionary support from CureDuchenne Ventures was the catalyst needed for Bamboo Therapeutics to rapidly advance its mission. Now in partnership with Pfizer, Bamboo will be able to move even faster to advance our gene therapies to clinical trials and impact the lives of Duchenne patients, regardless of their genetic mutation,” said Sheila Mikhail, Chief Executive Officer of Bamboo Therapeutics.

CureDuchenne Ventures, LLC, the investment arm of CureDuchenne, a leading non-profit dedicated to curing Duchenne, supported Bamboo’s multi-stage investment strategy with both equity financing and advisory direction from Dr. Jak Knowles, Managing Director of CureDuchenne Ventures. The focus of Bamboo’s Duchenne program is on delivering a mini-dystrophin gene sequence to muscle cells. Mutations in the dystrophin gene, create a lack of dystrophin protein which is the overarching cause of Duchenne; Bamboo’s gene therapy will help all patients with Duchenne, regardless of their genetic mutation. CureDuchenne hosted a recent webinar with Bamboo to educate patients and families about their exciting technology.

“On behalf of our boys with Duchenne, we have to be bold yet prudent as we imagine the future of a cure,” said Debra Miller, CEO of CureDuchenne Ventures and CureDuchenne. “This needs to be backed by a strong understanding of the science. Bamboo Therapeutics is delivering some of the most promising work in gene therapy. Their progress is giving hope to this and future generations of boys with Duchenne and their families.”

“Bamboo’s transformational approach is leading the way to new medical breakthroughs where genetic mutations will one day be corrected, and those with Duchenne will be able to live long productive lives,” added Dr. Jak Knowles. “Partnerships between patient-centered funds like CureDuchenne Ventures, and groundbreaking biotechnology companies, like Bamboo, can generate game-changing therapies for patients with rare diseases.”

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 nine research projects that have advanced to human clinical trials. CureDuchenne has leveraged over $100 million in pharma and biotech research and development investments. 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.

About CureDuchenne
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, multiple research projects have progressed to human clinical trials. 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.  CureDuchenne also is the innovator bringing physical therapy and standard of care to local communities around the country through CureDuchenne Cares.  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.