Using AI to Power Custom Treatments for Rare Diseases with Steven Ringel of Nome Therapeutics 11-10-25

Summary

Podcast Summary: Becker Business

Episode: Using AI to Power Custom Treatments for Rare Diseases with Steven Ringle of Nome Therapeutics
Host: Chanel Bunger (for Scott Becker)
Guest: Steven Ringle (CEO, Kizuna Foundation & Nome Therapeutics)
Date: November 10, 2025

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Main Theme

This episode explores how Nome Therapeutics, under the leadership of CEO Steven Ringle, is harnessing artificial intelligence (AI) to radically improve access to custom treatments for rare disease patients—turning personalized medicine from a niche offering into a scalable reality. Ringle shares his personal connection to rare diseases, the challenges patients face, and how policy and technology are opening new doors for patient-driven innovation.

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Guest Introduction: Steven Ringle’s Story & Mission

[00:33–02:27]

• Personal Connection:
  • Steven is both an ultra-rare disease patient (diagnosed with an inherited retinal disease) and sibling.

    “For me, that was a lightning rod moment where I knew I had to do something about the problem.” (Steven Ringle, 00:33)

• Background:
  • After years in commercial roles in precision medicine, he founded Kizuna Foundation (a nonprofit developing custom gene therapy for a U.S. patient population of just 18).
• Broader Vision:
  • Experiences showed how difficult building a drug was for the average patient, family, or clinician.
  • Realized a tech solution could democratize the process:

    “Gnome is making it easy to go build those custom therapeutics. And it’s doing that by leveraging artificial intelligence...” (Steven Ringle, 02:05)

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How Nome Uses AI to Accelerate Rare Disease Treatments

[02:30–04:21]

• The "Access Gap":
  • Critical first steps—identifying appropriate patients for custom therapies—are traditionally expert- and time-intensive.
• AI Solution Mechanics:
  • Nome developed an AI “engine” that rapidly scans, interprets, and consolidates data from dozens of databases, replicating expert analysis.

    “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... makes it super clear whether it's a good idea...” (Steven Ringle, 03:14)

• User Experience:
  • Patients input limited data online, Nome’s AI assesses and produces a clear, actionable report (with a score out of 100 on feasibility/suitability for a custom therapy).
  • If high potential, Nome connects families to experts for next steps; if not, the report brings needed clarity.

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Trends Shaping AI and Healthcare for Rare Diseases

[04:24–07:17]

Steven outlines three key trends spurring this paradigm shift:

• 1. Regulatory Evolution:
  • The U.S. is issuing new guidance likely permitting “N=1” therapies—custom treatments for a single patient—under efficient regulatory protocols.

    “We’re hearing…new guidance about encoding the possibility for N equals one…in a way that is very efficient from a regulatory process.” (Steven Ringle, 05:11)

• 2. Rise of Platform Medicines:
  • Biotech R&D is shifting from single assets to scalable, platform-based drug design.
    • 40% of new biotechs in the past 5 years are platform companies, getting two-thirds of VC funding.
    • Drug development classes have expanded from 3 in the 1980s to 12+ today.
• 3. Improved Patient Identification:

  • Genetic diagnostics are driving rapid growth in rare disease identification—400,000 new patients per year (60% annual growth).

  • With better patient-finding, the case for scaling custom solutions is stronger than ever.

    “When you put those three trends together, it really starts to scream that there’s an opportunity here…” (Steven Ringle, 07:00)

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Looking Ahead: Hopes and Excitement for 2025–26

[07:20–08:38]

• Progress in Personalized Medicine:
  • Ringle is inspired by leading academic medical centers (e.g., University of Pennsylvania, Boston Children’s, Mayo, Columbia, Rady, St. Jude) that are now taking on rare-disease drug development.
• Rising Grassroots Momentum:
  • Patient and parent organizations (like his own) are successfully driving custom drug development, spurred by new approvals and broader awareness.

    “I’ve also been…inspired by many other parents and patients doing this work…as families start to wake up and realize that actually, it’s possible to do something about the diseases they’re suffering with.” (Steven Ringle, 08:19)

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Advice for Leaders in Health Innovation

[08:38–09:42]

• Self-Care & Passion:
  1. Prioritize health/mental clarity (meditation is key for Steven, especially on tough days).
  2. Pursue work you care deeply about—the intrinsic drive sustains you through the inevitable challenges.

  “There are so many different challenges…But for me, it’s so important that these efforts succeed, that I love every day working on it…” (Steven Ringle, 09:29)

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Final Call to Action

[09:47–10:07]

• For rare disease patients, parents, or families seeking answers and options for custom medicine:

  “Just visit us at gnome.bio and we can help you.” (Steven Ringle, 09:53)

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Notable Quotes

• “For me, that was a lightning rod moment where I knew I had to do something about the problem.” (Steven Ringle, 00:33)
• “Gnome is making it easy to go build those custom therapeutics. And it’s doing that by leveraging artificial intelligence...” (02:05)
• “We have built an engine that's able to call dozens of different databases... and put that together into a single score...” (03:14)
• “We’re hearing…new guidance about encoding the possibility for N equals one…in a way that is very efficient from a regulatory process.” (05:11)
• “There are so many different challenges…But for me, it’s so important that these efforts succeed, that I love every day working on it…” (09:29)

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Key Timestamps

• Guest Introduction & Personal Story – 00:33
• Founding Gnome & AI Approach – 02:05, 02:30
• How the AI Scoring System Works – 03:14
• Industry Trends in Rare Disease & AI – 04:28, 05:11, 06:31
• Academic & Patient-Led Developments – 07:26, 08:19
• Leadership Advice – 08:47, 09:29
• Call to Action for Listeners – 09:53

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Episode Takeaways

• Nome Therapeutics is uniquely placing AI in the hands of rare disease patients and families, demystifying and expediting the process of pursuing custom therapies.
• A combination of regulatory change, tech-enabled platforms, and improved diagnostics is creating a new era for rare disease solutions, making the once-impossible—personalized medicine at scale—realistic for many.
• Steven Ringle’s personal story and mission add authenticity and urgency to the hope and innovation that are reshaping this space.

Transcript

[A]

Hello.

[B]

And. Welcome. To. A. Special. Combined. Episode. Of. The. Becker's. Healthcare. Becker. Business. And. Becker. Private. Equity. Podcast. My. Name. Is. Chanel. Bunger. And. Today. I'm. Excited. To. Speak. With. Steven. Ringle. The. CEO. Of. The. Kizuna. Foundation. And. Gnome. Therapeutics. And. He. Joins. Us. Today. To. Talk. On. The. Subject. Of. Using. AI. To.

[A]

Build.

[B]

Treatments. That. Rare. Disease. Families. Deserve. Some. Trends. That. He's. Keeping. An.

[A]

Eye.

[B]

On. And. A. Bit. More. Stephen. Thank.

[A]

You.

[B]

So. Much.

[A]

For. Joining.

[B]

Me.

[A]

Thanks. So. Much. For. Having. Me.

[B]

Perfect. Now. Before. We. Get. Into. Everything. Can. You. Start. Us. Out. By. Introducing. Yourself. Self. And. Talking. A. Bit. About. The. Foundation. And. Gnome. Therapeutics.

[A]

Yeah. Of. Course. Well. First. And. Foremost. I'm. An. Ultra. Rare. Disease. Patient. And. Sibling. When. I. Was. Seventeen. I. Was. Diagnosed. With. An. Inherited. Retinal. Disease. Which. Means. I'm. Going. Blind. Over. Time. And. A. Few. Years. Later. My. Younger. Sister. Was. Diagnosed. With. The. Same. Thing. For. Me. That. Was. A. Lightning. Rod. Moment. Where. I. Knew. I. Had. To. Do. Something. About. The. Problem. I. Didn't. Know. What. So. I. Spent. The. Next. Decade. Working. In. Various. Commercial. Leadership. Roles. At. Large. And. Small. Companies. In. The. Precision. Medicine. Industry. Really. Just. Educating. Myself. About. The. State. Of. Healthcare. And. What. Could. Possibly. Be. Done. For. Rare. Patients. Like. Us. After. That. Length. Of. Time. Really. Figured. Out. That. It. Was. Possible. To. Build. A. Custom. Therapy. For. Ultra. Rare. Disease. Patients. Like. Us. With. Existing. Technologies. And. That's. What. Spawned. Kizuna. Foundation. Where. We.

[B]

Are.

[A]

A. Five. Hundred. One. C. Three. Building. A. Custom. Gene. Therapy. For. Our. Very. Small. Eighteen. Diagnosed. Patient. In. The. Us. Patient. Population. And. Through. That. Experience. I. Really. Started. To. Understand. That. This. Wasn't. Just. About. You. Know. R. One. Condition. Or. A. Few. Conditions. This. Was. Really. About. You. Know. Personalized. Medicine. Taking. Hold. And. Scaling. And. Really. Being. An. Option. For. Many. More. Patients. However. Through. My. Own. Experience. And. Watching. Others. Like. Us. Build. We. Thought. That. 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. Gnome. Is. Making. It. Easy. To. Go. Build. Those. Custom. Therapeutics. And. It's. Doing. That. By. Leveraging. Artificial. Intelligence. To. Scale. A. Lot. Of. The. Intelligence. That. Experts. Have. And. Put. That. In. The. Palm. Of. Any. Individual. Patient. Parent.

[B]

Or.

[A]

Clinician. So. That. They. Too. Can. Build. A. Custom. Drug. Which. Is. Something. That. As. A. Patient. I. Can. Say. Is. Very. Very. Desired.

[B]

Perfect. Thank. You. So. Much. For. That. Introduction. And. Now. Can. You.

[A]

Talk.

[B]

A. Bit. About. How. Gnome. Is. Using. AI. To.

[A]

Build.

[B]

Treatments. For. Rare. Diseases.

[A]

For. Sure. We. Really. See. That. There's. An. Access. Gap. To. Even. The. First. Steps. Of. The. Process. Of. Who. Is. The. Right. Patient. For. For. A. Potential. Custom. Medicine. These. Are. Complicated. Therapies. There. Are. Many. Factors. That. Go. Into. Whether. It's. Appropriate. For. Any. Patient. To. Receive. One. And. Whether. It's. Even. Possible. To. Go. Make. One. For. A. Given. Patient. It's. Not. Always. Something. That. Can. Be. Done. And. So. Instead. Of. Having. Experts. Kind. Of. Comb. Databases. For. Months. On. End. We. Thought. That. Artificial. Intelligence. Could. Do. That. Better. So. 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. Zero. To. One. Hundred. That. Makes. It. Super. Clear. Whether. It's. A. Good. Idea. And. Whether. It's. Possible. For. A. Custom. Drug. To. Be. Made. For. Any. Given. Patients. The. Way. That. We. Are. Going. To. First. Bringing. This. To. Market. And. Deploying. It. Is. By. Making. It. Really. Easy. For. Any. Patient. Just. To. Go. Online. Share. A. Little. Bit. Of. Their. Information. With. Us. Which. Of. Course. We. Protect. As. We. Would. With. Any. Patient's. Information. And. Then. We. On. Their. Behalf. Query. Our. Own. AI. Database. And. Come. Back. To. Them. With. A. Report. That. Report. Tells. Them. This. Is. A. Great. Idea. It's. Seventy. Out. Of. One. Hundred. Likely. That. This. Is. Going. To. Work. For. You. Or. Maybe. Something. Else. Maybe. It. Won't. Work. For. Them. But. Either. Way. We. Bring. That. Clarity. To. Patients. In. The. Event. That. It. Is. A. Good. Idea. And. That. There. Is. A. High. Probability. That. Something. Like. This. Could. Work. We've. Then. Got. Team. That's. Able. To. Help. Those. Patients. And. Families. Take. The. Next. Step. In. Developing. A. Custom. Medicine. Just. For. Their. Condition.

[B]

That's. Amazing. To. Hear. And. Now. Zooming. Out.

[A]

A.

[B]

Bit.

[A]

Can.

[B]

You. Talk. About. The. Trends. That. You're. Watching. In. AI. And. Healthcare. Overall. Today.

[A]

Yeah. For. Sure. I. Mean. This. Really. Matters. As. A. Problem. So.

[B]

Just.

[A]

At. The. Top. Line. There's. Over. Three. Hundred. Million. Patients. Globally. Living. With. Rare. Disease. And. That's. About. Six. Times. The. Amount. Of. Cancer. Patients. Despite. That. There's. A. Fraction. Of. The. Amount. Of. Investment. Into. Rare. Disease. Because. As. Everybody. Knows. Traditionally. It's. Been. Very. Hard. To. Make. A. Return. Building. Rare. Disease. Therapeutics. So. That's. Kind. Of. The. State. Of. Affairs. But. Some. Stuff. Is. Changing. And. Specifically. There's. Three. Trends. That. I've. Really. Been. Paying. Attention. To. That. Got. Me. Excited. To. Try. To. Scale. Up. An. AI. Solution. Here. The. First. One. Is. A. Major. Shift. In. Regulatory. Guidance. Specifically. In. The. United. States. Right. Now. We're. Hearing. Comments. From. Prasad. That. Likely. In.

[B]

The.

[A]

Next. Few. Weeks. Even. There's. Coming. New. Guidance. About. Encoding. The. Possibility. For. N. Equals. One. You. Know. One. Individual. Patient. To. Get. A. Drug. Made. Just. For. Them. In. A. Way. That. Is. Very. Efficient. From. A. Regulatory. Process. And. That's. Something. That's. Been. A. Little. Bit. In. The. Gray. Area. Historically. And. So. Having. That. Clarity. Is. Really. Going. To. Further. Accelerate. The. Field. Of. Building. Individualized. Medicines. The. Second. One. Is. That. There. Are. A. Lot. Of. Companies. Out. There. Who. Are. Trying. To. Develop. What. We'll. Call. Platform. Medicines. So. Something. That. Can. Be. Thought. Of. As. A. Process. Or. Technology. Backed. Versus. A. Single. Asset. In. The. Past. Five. Years. Alone. Forty. Percent. Of. New. Biotechs. That. Have. Been. Created. Are. Platform. Companies. And. Those. Companies. Have. Received. Two. Thirds. Of. All. Venture. Capital. Funding. That. Is. A. Trend. That's. Really. Accelerating. Now. And. I. Even. Had. One. Bms. Executive. Tell. Me. That. Back. In. The. Eighties. When. The. Original. Orphan. Drug. Act. Passed. There. Were. About. Three. Basic. Classes. Of. Medicine. But. Now. There's. Over. A. Dozen. And. Growing. And. So. We're. Seeing. Drugs. Come. To. Market. As. Not. Individual. Therapies. But. As. Platforms. And. As. That. Happens. It. Means. It's. More. Important. Than. Ever. To. Think. About. The. Full. Ability. For. That. Platform. To. Reach. As. Many. Patients. As. Possible. And. Then. The. Last. Thing. Is. That. Not.

[B]

Only.

[A]

Are. There. Lots.

[B]

Of.

[A]

Patients. Out. There. With. Rare. Diseases. But. We're. Actually. Finding. Them. Faster. Than. Ever. About. Four. Hundred. Thousand. New. Patients. Are. Diagnosed. Every. Year. With. A. Rare. Disease. And. That's. Growing. Sixty. Percent. Year. Over. Year. As. Genetic. Diagnoses. Continue. To. Penetrate. Healthcare. And. 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. So. When. You. Put. Those. Three. Trends. Together. It. Really. Starts. To. Scream. That. There's. An. Opportunity. Here. To. To. Serve. These. Patients. Differently. If. We. Can. Do. It. At. An. Affordable. Cost. At. Scale. And. That's. Really. Why. We. Think. Artificial. Intelligence. Is. Intelligence. Is. Incredibly. Important. Here.

[B]

Absolutely. Got. It. Got. It. And. Now. Looking. Into.

[A]

The.

[B]

Future. A. Bit. Can. You. Talk. About. What. You're. Most. Focused. On. And. Excited. About. Going. Into. The. End. Of. Twenty. Twenty. Five. And. Into. Twenty. Twenty.

[A]

Six. For. Sure. I'm. Really. Excited. To. See. The. Continued. Drum. Beat. Of. Progress. From. Many. Different. Angles. And. Organizations. In. The. World. Of. Personalized. Medicine. So. Building. On. The. Success. Of. Baby. Kj. This. Summer. At. University. Of. Pennsylvania. A. Custom. Gene. Therapy. There. We've. Continued. To. See. Many. Other. Academic. Institutions. Take. On. The. Role. Of. Drugmaker. Places. Like. Boston. Children's. Mayo. Columbia. Rady. Saint. Jude. Just. To. Name. A. Few. We're. Seeing. Tremendous. Work. Out. Of. Those. Groups. And. I'm. Very. Excited. To. See. Them. Continue. To. Invest. In. Their. Programs. At. The. Same. Time. I'm. Also. Very. Excited. To. See. That. Same. Type. Of. Work. Going. On. In. Patient. Organizations. I. Have. One. Myself. As. You. Mentioned. And. We're. Building. A. Custom. Medicine. But. I've. Also. Been. Just. So. Inspired. By. Many. Other. Parents. And. Patients. Doing. This. Work. One. That. I. Call. Out. That. Has. Recently. Gotten. Some. Interesting. And. Exciting. Approvals. Is. Lpita. Therapeutics. But. There. Are. Dozens. That. I've. Had. The. Privilege. Of. Meeting. In. The. Last. Year. Or. Two. And. 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.

[B]

Got. It. Got. It. And. With. All. Of. Your. Experience. And. Passion. I'm. Curious. To. Hear. What. Advice. You. Would. Give. To. Evolving. Litters. Maybe. Looking. To. Have. The. Same. Drive. And. Success. In. Their. Careers. As. You.

[A]

Have. Two. Main. Things. I. Think. The. First. One. Is. 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. I. Think. It's. So. Important. To. Make. Sure. That. You. Have. That. Ability. To. Take. A. Step. Back. Breathe. And. Get. Some. Mental. Clarity. And. Focus. The. Other. One. Is. Just. What. You. Said. Where. Really. Passion. For. What. You're. Doing. Makes. All. The. Difference. I. Mean. There. Are. So. Many. Different. Challenges. Of. Both. Being. A. Visually. Impaired. Patient. Itself. Let. Alone. You. Know. Building. Two. Organizations. At. Once. But. For. Me. Like. It's. So. Important. That. These. Efforts. Succeed. That. I. Love. Every. Day. Working. On. It. Challenges. And. All. So. I. Think. For. Any. Leader. Listening. Hopefully. Those. Are. Two. Useful. Pieces. Of. Advice.

[B]

Excellent. Advice. Well. I. Want. To. Thank. You. For. Your. Time. Today. But. Before. I. Let. You. Go. Is. There. Anything. Else.

[A]

That.

[B]

Listeners. Should. Know.

[A]

Thanks. So. Much. For. Having. Me. The. Only. Thing. To. Know. For. Listeners. Is. If. You. Are. Interested. In. Trying. To. Figure. Out. If. Custom. Medicine. Would. Be. Right. For. You. If. You're. A. Rare. Disease. Patient. Or. Parent. Or. Family. Member. That. Hasn't. Been. Served. With. Any. Options. And. You. Want. To. Change. That. Just. Visit. Us. At. Gnome. Bio. And. We. Can. Help. You. Well.

[B]

That's. A. Great. Way. To. End. Our. Conversation. Steven. I.

[A]

Want.

[B]

To. Thank. You. Once. Again. For. Joining. Me. Today. And. For. Sharing. Your. Insights. On. The. Becker's. Healthcare. Becker. Business. And. Becker. Private. Equity. Podcast. Thank. You. So. Much.