A note from Debra

Pfizer, Bamboo and CureDuchenne – An Endorsement on the Right Track

Posted on August 1, 2016 7:24 pm

 

Today, we are very pleased to announce that Pfizer has acquired Bamboo Therapeutics, a gene therapy company we invested in earlier this year through CureDuchenne Ventures. The deal is valued at up to $645 million. Bamboo, led by Dr. Richard Jude Samulski, PhD, the Director of the Gene Therapy Center at UNC, and Dr. Xiao Xiao, PhD, a Professor of Gene Therapy at UNC, who developed a mini dystrophin sequence that when encapsulated can utilize adeno-associated virus (AAV) to treat Duchenne patients who lack a functioning dystrophin gene. This dysfunction, caused by a genetic mutation, is the root of Duchenne muscular dystrophy, a disease that Dr. Xiao has dedicated his 30-year research career to understanding and curing.

The acquisition of Bamboo by Pfizer is a recognition that Bamboo is on the right track. Now with support of this industry powerhouse, Bamboo can more aggressively pursue its dystrophin gene therapy efforts and accelerate finding a cure for Duchenne.

The path to this point was bold, and yet cautious with significant due diligence along the way. CureDuchenne Ventures identified Bamboo as a leader in gene therapy and their focus on Duchenne has both impressed us and given us hope. We, in turn, provided a launching pad for Bamboo. Our investment in Bamboo enabled the company to acquire a viral vector facility from the University of North Carolina at Chapel Hill. The vector core is critical for manufacturing AAV, and by bringing this manufacturing capability in-house, Bamboo became one of the vertically integrated gene therapy companies. This manufacturing capability also positioned Bamboo for a Series A capital raise of $49.5 million and solidified the company’s position as an aggressive leader in the gene therapy space.

We hosted a recent webinar with Bamboo to educate patient and families about their exciting technology.

Today we celebrate the Bamboo team of Dr. Samulski and Dr. Xiao, as well as the company’s CEO Sheila Mikhail. Their determination and strategic focus has delivered a profound return on investment for CureDuchenne Ventures, but more importantly, their company has given us hope that human clinical trials to correct the dystrophin gene are within reach.

We appreciate both Bamboo and Pfizer’s dedication to Duchenne and we look forward to working with both companies in the future to bring treatments to those with Duchenne.

 

Thoughts about PTC

Posted on July 25, 20165:32 pm

The announcement that PTC recently submitted an appeal to elevate the “Refusal to File” of Translarna (also known as ataluren and PTC124) to the next level of FDA management is an opportunity for the Duchenne community to raise their voices and let their experiences and opinions be heard! Translarna is the only therapy under investigation Read more »

Summer Road Trip

Posted on July 13, 20164:49 pm

  Guest blog by Hawken Miller Many of the things I don’t think are possible for me to do really are. Once I get over my initial apprehension and thoughts about what I won’t be able to do and I open my mind I find that anything I set my mind to is possible. This Read more »