CureDuchenne Applauds Pfizer’s Encouraging Data from Phase Ib Mini-Dystrophin Gene Therapy Trial for Duchenne Muscular Dystrophy


Newport Beach, CA, May 15, 2020 –­­­  CureDuchenne, a leading global nonprofit focused on funding and finding a cure for Duchenne muscular dystrophy, congratulates Pfizer for announcing data from subjects that have completed the 12-month Phase Ib clinical trial presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. 

Today’s announcement included updates on the safety and efficacy data from muscle biopsies and the North Star Ambulatory Assessments (NSAA) for the 9 participants enrolled. The treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, including sustained levels of mini-dystrophin expression and improvements on the NSAA rating scale.

“Pfizer is proud to be developing this potentially transformative therapy for the Duchenne muscular dystrophy community.  We’d like to thank all of the participants, partners and families who have helped us progress towards initiation of our planned pivotal study,” said Mikael Dolsten, Chief Scientific Officer and President of Worldwide Research, Development and Medical at Pfizer Inc.

CureDuchenne Ventures played an integral role in the progression of this technology, with an early investment in Bamboo Therapeutics at a critical time. This capital infusion enabled Bamboo to proceed with vector manufacturing and other critical experiments which underpin the data released today. Pfizer acquired Bamboo and their Duchenne program, soon after. 

“CureDuchenne provided funding at a crucial time which allowed Bamboo Therapeutics to de-risk and accelerate their clinical program for Duchenne,” said Dr. Jude Samulski, Co-Founder of Bamboo Therapeutics and current President, Chief Scientific Officer and Co-Founder at AskBio.  “We are thrilled for the Duchenne community to see this science and clinical development program advance.”

“We are pleased to see such encouraging data from Phase Ib and to see Pfizer progress to planning a global Phase III gene therapy clinical trial for Duchenne muscular dystrophy.  CureDuchenne is proud to have played a vital role in this advancement through our investment into this program at Bamboo Therapeutics.  It’s investments like these that take a long-term vison of what the Duchenne community needs and is continued proof of our success leveraging venture philanthropy to provide it,” said Debra Miller, Founder and CEO of CureDuchenne.

Funding to Bamboo Therapeutics is an exemplary model of the CureDuchenne venture philanthropy approach. CureDuchenne Ventures provides funding and domain expertise to accelerate critical research to enable the greatest likelihood for clinical success for early to mid-stage Duchenne companies.

About CureDuchenne

CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 individuals living today. CureDuchenne is dedicated to finding and funding a cure for Duchenne by breaking the traditional charitable mold through an innovative venture philanthropy model that funds groundbreaking research, early diagnosis, and community education. For more information on how to help raise awareness and funds needed for research, please visit

Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.