Accelerating Rare Disease Drug Development Through AI and Repurposing
How a tech-founder-turned-rare-disease-advocate leveraged AI to discover promising drug repurposing candidates for CMT1A in under a year, opening a reproducible framework for the entire pharmaceutical and rare disease community.

David Apple
Founder & CEO
Shark Tooth Biotech
apple@sharktooth.bio
September 2026
Christy Rohani-Montez: I’m delighted to be speaking to David Apple, who has an incredible story involving a career flip from being highly successful in the tech world to starting a biotech company for rare disease drug development. David, what catalysed that switch?
David Apple: My trajectory completely shifted nearly 4 years ago when my son, Ari, was diagnosed with Charcot-Marie-Tooth disease type 1A (CMT1A). Faced with this reality, I stepped back from my tech career to focus on finding a solution for him. I initially joined the board of the Charcot-Marie-Tooth Association (CMTA). Immersing myself in the scientific literature, I noticed some low-hanging research opportunities, and so, I founded Shark Tooth Biotech to explore these overlooked avenues. As a third-time founder, the entrepreneurial leap wasn’t intimidating, but entering biotech certainly was. And really it was driven by the realization that nobody would care more or fight harder for a cure than a parent.
Christy Rohani-Montez: CMT1A has traditionally been a very complex and difficult therapeutic target with past failures. What avenues did you explore initially, and what led you to your current lead program?
David Apple: Historically, there have been delivery and toxicity challenges in the human peripheral nervous system, but post-COVID, we saw an explosion in platform delivery technologies. Our initial approach was to explore non-viral delivery mechanisms for siRNA by pitching CMT1A to these platform companies. We currently have 2 ongoing projects in this area, in collaboration with the National Research Council of Canada and Express Biolabs.
However, we decided to pursue another avenue after I read a paper by Professor Jordan Verplank, who had discovered previously unexplored pathways relevant to CMT1A. His work was so compelling that he has since joined Shark Tooth as our Chief Scientific Officer and leads the science. Recognizing the potential of drug repurposing to reach my son much faster than developing a novel drug from scratch, we pivoted, and drug repurposing has now surpassed our genetic delivery efforts to become our primary lead program.
Christy Rohani-Montez: You recently completed a preclinical study for that drug repurposing program. What were the findings?
David Apple: We tested 19 repurposed drugs and are awaiting results. I’m hoping that more than one drug works so that we can combine the repurposed drugs to generate novel intellectual property (IP), which is a prerequisite to raising money and funding the next steps of this program.
Christy Rohani-Montez: What role has artificial intelligence played in your process?
David Apple: A big role. Initially, I used ChatGPT’s voice mode to better understand the science, map out hypotheses, and stress-test operational theories.
And then for target discovery, we partnered with E9 Genomics and used platforms like Claude and Biomni. We fed the models literature reviews, public high-throughput screening data, and proprietary RNA sequencing profiles of CMT1A mice. The AI helped map dysregulated pathways, evaluate toxicity profiles, and narrow down hundreds of off-patent generic candidates to the 19 drugs we tested
Christy Rohani-Montez: Repurposed drugs often face investor skepticism regarding commercial viability and IP protection. What is your strategy moving forward, and what type of partnerships are you looking for?
David Apple: Assuming we get positive results, our next immediate step will be conducting RNA sequencing on the sciatic nerves of our treated mice to map the precise upstream and downstream mechanisms of the drugs we tested. This mechanistic data will allow us to design synergistic drug combinations. We will then secure composition of matter patents for specific combinations and formulations. Our timeline goal is to achieve an Investigational New Drug (IND) clearance within 3 years, which would put our combination therapy into clinical trials before 2030.
We want to secure strategic partnerships with biotech and pharma companies. Our ideal structure would allow us to retain operational control and maintain our rapid pace, while leaning on a partner’s established legal and regulatory infrastructure to help us see around corners and avoid mistakes.
Ultimately, our goal is to bring a viable treatment into the hands of patients as fast as the science and the regulatory pathway responsibly allow.
Because we are targeting dysregulated pathways rather than a singular genetic mutation, we hope that our combination therapy could translate to other CMT subtypes and provide a highly reproducible, AI-driven framework for the broader rare disease community.
By