Value of the Rare Disease Market
people suffer from a rare disease
of the 10,000 rare diseases have no treatment option today
The average likelihood of Clinical trial success from Phase 1 to approval is twice as high for rare diseases (17%) versus all diseases (8%).
There are 10,000 rare diseases with an average market size of $150M. Traditional pharma corporations are such large and complex institutions that they need a $1B+ TAMs to even begin exploring a disease. The same is true for a traditional VC backed biotech, with a single indication or platform. This makes the current market approach incapable of going after one of the thousands of $150M rare disease markets. However, these $150M markets are exactly where Curetopia shines and has a competitive moat.
Curetopia’s focus is specifically on this long tail of rare genetic diseases, which have a 2X higher rate of FDA approval. The family members of these rare disease patients are mission-driven warriors who have a high-risk tolerance and a 24/7 work ethic, doing whatever possible to help. Curetopia brings cures to market in a fraction of the time, with lower upfront costs.
Each rare disease uses the same underlying framework of data pooling, patient self-reporting, and feedback monitoring - Curetopia unifies this tooling and unique know-how to accelerate treatments. Family members are rewarded for contributing to the rare disease community, global researchers are incentivized to provide support, and capital is pooled together to afford drug development. Curetopia already has 8 biological targets qualified for their next set of drugs to bring to FDA trials.
There are 10,000 rare diseases with an average market size of $150M. Traditional pharma corporations are such large and complex institutions that they need a $1B+ TAMs to even begin exploring a disease. The same is true for a traditional VC backed bi...

First we confirm that yeast have an ancestral version of a human disease gene. Because yeast have been poked and prodded in the lab for decades, many of the rare disease genes we seek to model in yeast have already been studied.

We have had great success with a 8,400-compound drug repurposing library called TargetMol, which offers a wide selection of clinically actionable compounds including generic drugs and over-the-counter supplements/nutraceuticals.

In almost all cases we can leverage already existing yeast avatars that were generated and characterized by academic labs, sometimes as long as two decades ago. When necessary, we can dial in a specific mutation at practically any location in the yeast genome using gene engineering techniques that predate CRISPR-Cas9.
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Total Supply


Curetopia captures value by leveraging the power of a decentralized and mission-driven community to tackle the $1 trillion rare disease market, which has long been overlooked by traditional pharmaceutical companies and VC-backed biotech startups. Using a BioDAO framework, Curetopia raises funding via token sales, pooling resources into a treasury that is used to develop real-world scientific assets, including novel drug candidates for rare diseases. This model democratizes drug discovery by aligning incentives across patients, researchers, and funders. By involving patient families as core contributors and rewarding their participation, Curetopia not only accelerates research but also creates shared IP ownership, ensuring that the community directly benefits from the breakthroughs they help achieve.
The value capture flywheel begins with token-based funding that fuels highly capital-efficient R&D on under-addressed diseases. The resulting drugs and IP generate revenues through licensing agreements, partnerships, or direct product sales, with proceeds flowing back into the BioDAO treasury. This reinvestment drives the next wave of drug development and expands the network of patient-driven collaborators, increasing the DAO's impact and financial sustainability. Curetopia’s systematic approach to rare disease treatment, combined with its pioneering application of the BioDAO model, ensures a virtuous cycle where each success amplifies its capacity to tackle the next challenge, making it a transformative force in rare disease drug development.
Curetopia captures value by leveraging the power of a decentralized and mission-driven community to tackle the $1 trillion rare disease market, which has long been overlooked by traditional pharmaceutical companies and VC-backed biotech startups. Usi...


Perlara Substack

Perlara Substack

Perlara Substack