Podcast Summary: Using AI to Power Custom Treatments for Rare Diseases

Podcast: Becker’s Healthcare Podcast
Host: Chanel Bunger (A)
Guest: Steven Ringel (B), CEO, Kizuna Foundation & GNOME Therapeutics
Date: November 11, 2025

Episode Overview

In this episode, Chanel Bunger interviews Steven Ringel, a rare disease patient, advocate, and biotech entrepreneur. The conversation centers on how artificial intelligence (AI) is making custom treatments for rare diseases more accessible. Steven shares his personal journey, the founding of Kizuna Foundation and GNOME Therapeutics, and his vision for the future of personalized medicine powered by AI. The episode also explores regulatory shifts, trends in biotech investment, and practical advice for leaders in healthcare innovation.

Guest Introduction & Background

[00:25–02:27]

• Personal Motivation: Steven introduces himself as both an ultra-rare disease patient and sibling. Diagnosed at 17 with an inherited retinal disease, his experience prompted a career dedicated to solving the rare disease treatment gap.

  • “For me, that was a lightning rod moment where I knew I had to do something about the problem.” — Steven (00:41)

• Professional Journey:

  • Spent a decade in commercial roles within precision medicine, educating himself on therapy development.
  • Founded Kizuna Foundation (a 501c3) to build custom gene therapy for a very small U.S. patient group (18 diagnosed people).
  • Realized the need for greater accessibility—most families can't navigate building a drug themselves.
  • Founded GNOME Therapeutics to use AI for making custom therapeutics creation accessible to non-experts.
  • “GNOME is making it easy to go build those custom therapeutics…by leveraging artificial intelligence to scale a lot of the intelligence that experts have.” — Steven (01:57)

Using AI to Build Custom Rare Disease Treatments

[02:27–04:21]

• Identifying Candidates:

  • There’s an “access gap” at the first step—figuring out whether a drug can or should be made for a specific patient.
  • Rather than experts manually searching databases for months, their AI engine analyzes dozens of databases, synthesizes outcomes, and generates an interpretable score (0–100) for suitability.
  • “We have built an engine that’s able to call dozens of different databases, interpret what’s coming out of those databases like an expert would, and put that together into a single score, 0 to 100.” — Steven (03:08)

• Patient-Facing Tools:

  • Patients can submit information online, and the AI provides a personalized report indicating the likelihood that custom medicine will work.
  • If the probability is high, GNOME’s team helps patients and families take the next step in development.
  • "We bring that clarity to patients. In the event that it is a good idea and ... a high probability … we’ve then got team that’s able to help those patients and families take the next step in developing a custom medicine." — Steven (04:01)

AI and Healthcare Trends Shaping the Field

[04:21–07:17]

Steven highlights three major trends driving innovation in rare disease therapies:

1. Regulatory Shift

   • New U.S. guidance (imminent as of recording) is expected to allow for highly individualized, "N=1" patient-specific drugs in a much more efficient way.
   • "We're hearing comments ... about encoding the possibility for N=1 ... to get a drug made just for them in a way that is very efficient from a regulatory process." — Steven (04:47)

2. Rise of Platform Medicines

   • 40% of new biotechs focus on platforms rather than single drugs—these get two-thirds of the VC funding.
   • The field has grown from three basic medicine classes in the 1980s to “over a dozen and growing.”
   • "We're seeing drugs come to market as not individual therapies, but as platforms … it's more important than ever to think about the full ability for that platform to reach as many patients as possible.” — Steven (05:38)

3. Accelerated Patient Identification

   • 400,000 new rare disease patients are diagnosed yearly, rising 60% year-over-year due to improved genetic diagnostics.
   • It's now more feasible to find and support these patients with custom treatments.
   • “With those new diagnoses, it's now possible to understand which conditions are out there, where are these patients, and think about who is the right patient to receive a custom therapy.” — Steven (06:29)

• Summary:
  • When combined, these trends make a compelling case for scaling AI-powered rare disease therapies.
  • “It really starts to scream that there's an opportunity here to serve these patients differently, if we can do it at an affordable cost at scale.” — Steven (06:44)

Looking Forward: The Future of Personalized Medicine

[07:17–08:38]

• Academic & Patient-Led Innovation:
  • Steven cites University of Pennsylvania’s Baby KJ custom gene therapy as a breakthrough, and shouts out institutions like Boston Children’s, Mayo, Columbia, Rady, and St. Jude for their roles as “drugmakers.”
  • Also highlights a trend of parent- and patient-led drug development, referencing groups like LPITA Therapeutics.
  • “I'm really excited to see more and more of those form as families start to wake up and realize that actually it's possible to do something about the diseases that they're suffering with.” — Steven (08:23)

Leadership Advice for Healthcare Innovators

[08:38–09:42]

• Self-Care:

  • “We are definitely in a grind hustle culture, but it's really important to take care of your body. I personally meditate a lot and I meditate twice on really hard or stressful days.” — Steven (08:51)

• Passion:

  • “Really passion for what you're doing makes all the difference … for me, like, it's so important that these efforts succeed, that I love every day working on it, challenges and all.” — Steven (09:16)

Final Message & Call to Action

[09:42–10:06]

• Steven encourages rare disease patients or families interested in custom medicine to visit GNOME Bio for support.
  • “If you are interested in trying to figure out if custom medicine would be right for you … just visit us at GNOME Bio and we can help you.” — Steven (09:51)

Memorable Quotes

• “It wasn’t really possible for the average parent or patient or clinician to build a drug, which is very, very difficult. And that’s why we started GNOME Therapeutics.” — Steven (01:44)
• “Artificial intelligence is incredibly important here.” — Steven (07:12)
• “Take care of your body ... meditate a lot ... passion for what you’re doing makes all the difference.” — Steven (08:52, 09:16)

Timestamps for Key Segments

• 00:25–02:27 — Steven’s personal and professional introduction
• 02:27–04:21 — How GNOME Therapeutics uses AI to assess and develop custom treatments
• 04:21–07:17 — Three big trends in AI and rare disease healthcare
• 07:17–08:38 — The future: academic and patient-led advances
• 08:38–09:42 — Steven’s leadership and life advice
• 09:42–10:06 — Final message & call to action

This episode gives a comprehensive look at how AI is shaking up the field of rare disease treatment, pairing Steven Ringel’s compelling personal story with insightful analysis of industry and regulatory trends. It's a must-listen for anyone interested in the intersection of technology, medicine, and patient advocacy.