In this episode of The Tech Leader's Playbook, Avetis Antaplyan sits down with Susan Ruediger, Founder and Chief Mission Officer of the CMT Research Foundation (CMTRF), and Laura MacNeill, the organization’s CEO. Together, they explore how patient-led research is revolutionizing drug development and catalyzing billion-dollar outcomes. Susan shares the remarkable story of CMTRF’s $128,000 seed investment in DTX Pharma that led to a $1 billion Novartis acquisition — a masterclass in strategic risk-taking and venture philanthropy. Laura explains how CMTRF’s unique “go-out-of-business” mission drives urgency, focus, and impact, while also inspiring other nonprofits to adopt similar models. The conversation dives deep into storytelling’s role in galvanizing donors, the importance of milestones and reinvestment, and how rare disease foundations can unlock breakthroughs for broader neurodegenerative diseases like ALS, Parkinson’s, and Alzheimer’s. Whether you’re a biotech leader, investor, or nonprofit executive, this episode offers actionable lessons on focus, partnerships, and creating outsized impact with limited resources.
Takeaways
Patient-led research can de-risk and accelerate drug development.
$128K seed funding led to a $1B Novartis acquisition.
CMTRF uses a venture-philanthropy model with milestone-based funding.
Mission: fund treatments, find a cure, close the foundation.
Storytelling drives awareness, donations, and partnerships.
Early investments keep promising science alive.
Biotech partnerships share risk and leverage expertise.
Novartis validated CMT as a major market opportunity.
Rare disease focus offers faster FDA pathways.
Staying laser-focused means saying no to distractions.
Chapters
00:00 Intro & Guest Welcome
01:20 From Grassroots Donations to Billion-Dollar Deals
02:30 Understanding CMT and Its Impact
05:00 Finding the Right Delivery Vehicle for Drugs
07:40 The $128K Bet That Changed Everything
09:50 Other Success Stories & Market Signaling
13:00 The Venture-Philanthropy Model Explained
16:30 The Power of Milestones and Flexibility
18:45 Reinvestment and Sustainable Funding
21:30 Role of Storytelling and Strategy in Movement Building
26:10 Velocity Campaign & Raising $20M
27:25 Why Biotechs Care About Rare Diseases
31:50 CMT as a Gateway Indication for Neurodegenerative Disease
33:30 Staying Focused and Saying No
38:30 The Drug Development Lifecycle and Staying Mission-Aligned
42:10 How to Get Involved and Follow CMTRF’s Work
45:10 Personal & Business Advice for Leaders
48:30 Favorite Books and Final Thoughts
52:00 Closing Remarks and Call to Action
Susan Ruediger’s Social Media Links:
https://www.linkedin.com/in/susan-ruediger/
Laura MacNeill’s Social Media Links:
https://www.linkedin.com/in/laura-macneill-m-b-a-97633732/
CMT Research Foundation’s Website:
Resources and Links: