Uncommon Therapeutics

TL;DR

Noah, an experienced tech entrepreneur, was directly impacted by a severe genetic disease when his daughter was diagnosed with Rett Syndrome, a disease that afflicts 1 in 10,000 girls. Using an innovative polytherapy strategy, we’ve successfully improved her quality of life, and have designed new therapeutics that each could be worth billions and together could deliver a cure.

Our uncommon approach to biotech includes deep respect for the complexities of biology and disease, but belief that many of the orthodoxies of biotech can be challenged by ideas from tech.

Why Now?

AI is unlocking every field and biotech is no different. However in bio the value will not go to the platform builders, but the users of the tools and the drug assets they create. Today it is easier and cheaper than ever to build better drugs.

Meanwhile, biotech is no longer a closed system. So many of the core modality IP (mAbs, ASOs, mRNA, Car-T, CRISPR, etc) have expired or will expire by the time a drug comes to market. There are mature CROs for every stage of drug development from screening to running studies. And there are experts available to consult who have built at the platform companies that spent billions refining these tools.

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.

Why Us?

“Start companies to solve personal problems” — Michael Seibel

Imagine while you are building your high flying tech company, your 18 month old daughter starts to lose the ability to walk, speak, use her hands, and will soon develop a cascade of maladies (seizures, breath holding and hyper ventilation, scoliosis and on) – this is Rett Syndrome. Over the months that followed Noah dedicated himself to this problem, reading over 300 papers in the first 9 months alone, speaking with every expert until he became one. We identified multiple repurposed drugs, not commonly used for Rett, that while not curative could immediately push back against the skill loss and successfully give her a higher quality of life. Today, Ellie is 5 and in the top 1% of people with Rett. While still limited, she is happy, walking around, not in pain, without seizures, and is learning to read and communicate with her eye gaze device.

Expertise unlocks traction: With just $1M, we utilized the best tech and modality expertise to design three novel therapeutics that target the underlying cause of the disease, all of which are now working in patient cells.

Results in Patient Cells

Team

Noah Auerhahn (Co-Founder & CEO) - Noah bootstrapped his first company and sold it to Rakuten for $15M and raised over $75M for his second company. In 2021 his daughter was diagnosed with Rett Syndrome. He dedicated himself to becoming an expert on the disease and then identifying disease modifying-therapies, personally investing over $1M into those efforts. He is deeply involved across every aspect of Uncommon’s drug development efforts.

Ryan Lim, PhD (Co-Founder & CSO) - Ryan is a biotech entrepreneur and biomedical scientist with over 10 years of experience in neurological diseases. At Uncommon he’s pioneering gene-editing, ASO, and small-molecule therapies for Rett Syndrome. He led AI-driven drug discovery efforts, taking a therapeutic candidate from initial discovery through IND-enabling studies on its way to ALS patients at Modulo Bio, and at UCI identified a repurposed compound that is currently in Phase II trials for Huntington’s disease.

Asks

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