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Rethinking Rare Disease: A New Model for Ultra-Rare Therapies with Beth White
In this episode of She Leads Biotech, I’m joined by Elizabeth (Beth) White, COO and Co-Founder of Orphan Therapeutics Accelerator, for a conversation that really stayed with me.
Beth has spent her career at the intersection of science, strategy, and commercialization, and today she’s focused on one of the biggest challenges in our industry: how do we bring therapies to patients with ultra-rare diseases when the traditional biotech model simply doesn’t support them?
As Beth explains, the science has moved forward, especially in gene and cell therapies, but the business model hasn’t kept up. And that’s leaving too many promising therapies sitting on the shelf, never reaching the patients who need them.
What she and her team are building with the Orphan Therapeutics Accelerator is a completely different approach. A nonprofit biotech model designed to rethink how therapies are funded, developed, and ultimately delivered to patients.
We talk about what that actually looks like in practice, from new ways of funding and partnering, to the creation of the CGT Exchange, a marketplace designed to unlock shelved assets and give them a path forward.
This is one of those conversations that really challenges how you think about drug development, investment, and impact.
And a reminder that behind every “ultra-rare” disease are real patients, real families, and a very real urgency to find solutions.
📌 Chapters
00:00 Introduction
01:52 Why ultra-rare diseases remain underserved
03:44 What the Orphan Therapeutics Accelerator is
05:00 Building a completely new development model
06:32 How the nonprofit structure works in practice
08:39 Creating awareness and industry engagement
10:34 Major partnerships and recent momentum
11:39 What the CGT Exchange is and why it matters
13:26 Can this model work at scale?
15:08 Beth’s journey into rare disease and gene therapy
17:01 Bridging science and business
19:18 Leadership across pharma, biotech, and nonprofit
21:19 Big pharma vs biotech mindset
23:09 Leading with limited resources
24:21 Advice for women in biotech
25:37 Risk-taking and career decisions
26:17 The future of rare disease development
28:40 The role of AI in accelerating progress
🎙️ She Leads Biotech celebrates the journeys of women shaping the future of life sciences.
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