Our featured investment:
- Uncommon Therapeutics takes the spotlight as our newest featured investment.
- Founded in 2024, Uncommon Therapeutics is a disease-focused biotech developing multiple high-value therapeutics per disease by combining cutting-edge tools with deep disease expertise to deliver cures. They're initially targeting Rett Syndrome, a severe genetic disease affecting 1 in 10,000 female births, with plans to expand to other rare diseases.
- Noah Auerhahn, Co-Founder and CEO, brings significant entrepreneurial experience, having bootstrapped his first company and sold it to Rakuten for $15M in 2013, before raising over $75M for his second venture, a smart assistant device to help doctors with their clinical notes and billing teams with near perfect medical codes. He holds a Bachelor’s in Business Entrepreneurship from the USC Marshall School of Business. Noah’s journey into biotech began in 2021 when his daughter Ellie was diagnosed with Rett Syndrome, prompting him to immerse himself in the field-reading over 300 scientific papers in nine months and consulting with leading experts until he developed considerable expertise himself. Noah personally invested over $1M into identifying disease-modifying therapies before founding Uncommon Therapeutics.
- Ryan Lim, PhD, Co-Founder and CSO, complements Noah's entrepreneurial drive with deep scientific knowledge as a biotech entrepreneur and biomedical scientist with over 15 years of experience in neurological diseases. His previous experience includes leading AI-driven drug discovery efforts at Modulo Bio, advancing therapeutic candidates from from initial discovery through Investigative New Drug (IND) studies for Amyotrophic Lateral Sclerosis (ALS) patients. Prior to his role as Director of Biology at Modulo Bio, Ryan was a Project Scientist at University of California Irvine, where he identified a repurposed drug candidate that is currently in Phase II trials for Huntington’s disease. At Uncommon Therapeutics, he's currently pioneering gene-editing, ASO (antisense oligonucleotide), and small-molecule therapies for Rett Syndrome
- Uncommon Therapeutics has already designed three novel drug candidates targeting the underlying cause of Rett Syndrome. All three candidates are already showing efficacy in patient cell lines and are now entering animal studies-remarkable progress for a young biotech company with minimal resources.
Why Uncommon Therapeutics?
- The Rare Disease Challenge: Rare diseases collectively affect millions of people worldwide, yet individually receive limited attention from major pharmaceutical companies due to small patient populations and high development costs.
- For Rett Syndrome specifically, patients experience devastating symptoms including loss of ability to walk, speak, and use their hands, followed by seizures, breathing irregularities, and skeletal issues-all with limited treatment options. Rett is more common than one thinks; it is the leading cause of severe intellectual disability in females after Down Syndrome.The traditional drug development path has historically been long, expensive and fraught with failure — only 5% of therapies that start in animal studies ultimately achieve regulatory approval.Rare disease patients and their families face extraordinary challenges, including delayed diagnoses, limited treatment options, prohibitive costs, and lifelong care requirements. Governments and healthcare systems struggle with the high cost caring for these patients and sticker prices on orphan drugs, while pharmaceutical companies face pressure to recoup R&D investments from small patient populations.
- The Biotech Orthodoxy Problem: Traditional biotech development follows costly, time-consuming paths dictated by industry orthodoxies that may no longer be necessary. Many core modality IP patents (mAbs, ASOs, mRNA, CAR-T, CRISPR, etc.) have expired or will expire by the time new drugs come to market. Third-party Contract Research Organizations now exist for every stage of drug development. And, AI tools are transforming biotech allowing companies to build more smarter and leaner than ever.
- Uncommon Therapeutics As A Game-Changer: Uncommon Therapeutics is challenging these orthodoxies by applying tech-inspired methodologies to biotech. The company operates on the premise that the convergence of the expiring IP on modern modalities and advancements in AI creates unprecedented opportunities for small, agile teams focused on specific patient population, to develop high-impact therapies faster, at a fraction of the cost, compared to existing institutional platforms.
- Future Outlook: While starting with Rett Syndrome, Uncommon Therapeutic's approach can be applied to numerous rare diseases that currently lack effective treatments. Their capital-efficient model could dramatically reduce the cost and time required to bring rare disease therapeutics to market.
“At Uncommon, we believe now is the best time ever to build a disease focused biotech. So while Biotech may not get the first 1 person billion dollar company, in this AI-driven open source system, it may deliver the first 2 person one."
Other investors include Y Combinator, Pioneer Fund, Ethos Fund and Roar Ventures.
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