RealPharma: Conversations with Pharma Pathfinders

From Rock Star to Rare Disease Revolutionary: Casey McPherson’s Fight for His Daughter’s Life


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Episode Title: From Rock Star to Rare Disease Revolutionary: Casey McPherson’s Fight for His Daughter’s Life

In this deeply personal and inspiring conversation, hosts Na-Ri Oh and Ian Wendt sit down with Casey McPherson, founder and CEO of AlphaRose Therapeutics—and acclaimed recording artist—to explore how one father’s determination to save his daughter’s life is shaking up the rare disease drug development model.

Casey shares his journey from fronting rock bands to founding a biotech company, sparked by his daughter Rose’s diagnosis with an ultra-rare genetic disorder. He discusses why the current rare disease model is broken, how Alpha Rose is reimagining the path from diagnosis to treatment, and the emotional and ethical complexities of innovating in this space.

What You’ll Learn in This Episode:

  • How Casey’s music career prepared him to run a biotech startup.

  • Why rare disease families face a “diagnostic odyssey” and what needs to change.

  • The gap between available technology and systemic barriers to treatment.

  • How AlphaRose Therapeutics is creating scalable models for ultra-rare diseases.

  • The promise—and challenges—of engaging with regulators like the FDA in new ways.

  • Why the Leonard Cohen classic Hallelujah has become a personal anthem for Casey.

    Links & Resources:

    • AlphaRose Therapeutics

    • StartEngine – Invest in Alpha Rose (crowdfunding equity round open until August)

    • Leonard Cohen – Hallelujah (Jeff Buckley cover)

    • People Magazine feature on Casey McPherson

    • Follow Casey on Instagram

      ...more
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