Bamboo Therapeutics – Developing a novel gene therapy for patients with Duchenne muscular dystrophy

Bamboo Therapeutics is a recently formed biotechnology company focused on advancing therapies for rare pediatric diseases of the central and neuromuscular systems. Bamboo was founded to advance the pioneering laboratory work of Dr. Jude Samulski, PhD, the former Director of the Gene Therapy Center at the University of North Carolina into human clinical trials. “Gene Therapy” describes the idea of introducing new genetic material into cells to correct an existing problem, often due to a disease-causing mutation. During the June 13, 2016 webinar, Dr. Samulski explained the basics of gene therapy and described Bamboo’s approach to developing a treatment for Duchenne; by utilizing a disabled virus to deliver a small, highly optimized version of the dystrophin gene systemically to skeletal and cardiac muscle. The gene therapy, originally called BMB-D001, has already shown positive effects in Duchenne animal models, improving life span as well as skeletal and cardiac muscle function in both mice and rats. Life extension and maintenance of ambulation up to eight years post-treatment resulted from long-term dystrophin production in a canine Duchenne animal model.

Speakers included:

Sharon Hesterlee, Ph.D., EVP Patient Advocacy and Public Affairs, Bamboo Therapeutics

Jude Samulski, Ph.D., Scientific Founder, Bamboo Therapeutics, and former Director of the Gene Therapy Center at the University of North Carolina

Jak Knowles, MD, Vice President Medical and Scientific Affairs, CureDuchenne, and Managing Director CureDuchenne Ventures

Debra Miller, Founder and CEO, CureDuchenne


Click here to watch the recording of the webinar. You’ll be prompted to enter your name and email address to view the recording.

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