Curetopia Vision Paper

Abstract

Curetopia is the first biotech Decentralized Autonomous Organization (BioDAO) committed to eradicating rare diseases through decentralized science (DeSci). It addresses the unmet needs of over 10,000 rare diseases, which burden 1 in 10 people globally and impose a $1 trillion economic cost in the U.S. alone. Curetopia empowers patient communities with governance tokens to influence research and ensure anti-shelving of promising therapies. Focused initially on drug repurposing for inherited metabolic diseases, Curetopia leverages model organisms, tokenized intellectual property, and community-driven clinical studies. Its roadmap envisions scalable therapeutic solutions, fostering equity and sustainability in rare disease research and treatment innovation.

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Why Now?

The long tail of 10,000+ rare diseases is a silent scourge that devastates the lives of 1 in 10 people on Earth [1]. Ripple effects hurtle outward from affected families, burdening the entire healthcare system, and society at large. The EveryLife Foundation estimated the total economic cost of a subset of 5% of rare diseases in 2019 to be $1 trillion dollars in the US alone. Therefore, the aggregate cost burdens and lost productivity across the world today are astronomical, and are only projected to increase [2].

To date, the market has repeatedly failed rare disease communities because the commercial opportunity is too small, too expensive, and too geographically fragmented to attract TradBio investment and sustained drug development interest. Despite the fact that the average likelihood of success from Phase 1 to approval is twice as high for rare diseases (17%) versus all diseases (8%) [3].

We have let the rare disease long tail problem fester for far too long. Left to fend for themselves and told to go home and make memories while they can, rare disease patient advocates and rare disease patients are forced by the establishment to single-handedly raise awareness, build communities, and fundraise for research – all while taking care of a sick loved one or themselves.

It’s not fair. It’s not efficient. It’s not scalable. It’s time to leave this system behind.

Curetopia’s vision is that the long tail of 10,000+ rare diseases is not only biotech’s biggest challenge but also biotech’s biggest opportunity: the ideal sandbox for precision medicine where every patient is treated as a n-of-1 case [4,5]. We will reach the promised land of precision medicine with rare disease communities as cure guides. From their blood, sweat, and tears emerge vetted operational playbooks and derisked therapeutic modalities that are poised for scaling along the entire length of the distribution of diseases, including common diseases and aging itself [6].

While the “rare-to-common” thesis is not a new idea [7,8,9], its time has finally come thanks to the rise of Decentralized Science (DeSci). I founded Perlara as the world’s first biotech public benefit corporation (PBC) in 2014 on the premise and promise of partnering with patient communities from day one. However, Perlara was built on TradBio rails, and so in practice the ability to make patient communities codevelopment partners has been limited to a few exceptional families with means in the developed world. With an eye toward a fully level and global playing field, Curetopia represents an onchain fork of the patient-centric value proposition: from public benefit corporation to “public benefit community” aka a decentralized autonomous organization (DAO).

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Why DAO?

Curetopia is the world’s first biotech DAO (BioDAO) that unites rare disease communities and their allies, researchers and clinicians, DeSci enthusiasts (DeScientists), and the global crypto community in common cause for cures. Contributions from Curetopians – whether they be data of any kind, biospecimens, clinical trial participation or even compute time for analysis – are rewarded with utility tokens which afford critical governance rights to token holders. The most important governance right is the ability to control the fate of intellectual property (IP) created from community-sponsored research. Rare disease patient communities have all too often been rugged by the TradBio establishment in a predictable sequence of events. 

Families first find each other online, gel into communities, and corral researchers who are almost always academic scientists trying their best to help with limited resources. Families then rev into fundraising mode with bakesales, walkathons, golf tournaments and the like, kickstarting and derisking research projects that often lead to a patented drug candidate, knowhow, and other forms of IP. 

Patents and exclusive licensing rights are usually granted to a biotech company, which itself has to raise external funding from investors. Inevitably, the commercial winds shift and, cruelly, rare disease programs are the first on the chopping block [10]. This was the story behind two heartbreaking examples of commercial sponsors, Taysha Genetics [11] and Amicus Therapeutics [12], which abandoned promising rare disease gene therapy programs after intense fanfare and many promises to patients at the outset. 

The two biggest pain points that Curetopia will solve for rare disease communities are: (i) access to funding for rate-limiting rare disease research so that families don’t have to deplete savings or beg for money from friends and strangers; (ii) enshrinement of anti-shelving/clawback provisions in smart contracts so that no rare disease program that is scientifically derisked yet commercially unattractive is ever left out in the cold.

One can easily imagine smart contracts governing the use of data or cell lines that bind stakeholders to “use it or lose it” commitments and unlock token rewards in exchange for value-adding contributions. A decentralized biobank already exists [13]. Patents can be minted onchain and fractionalized in the form of IP-NFTs, whereafter provenance and chain of custody through licensing agreements can be tracked and audited by anyone with an internet connection, including the rare disease communities themselves. Onchain, fractionally owned patents will lead to approved drugs and revenues that would flow back to the Curetopia treasury aka community medicines [14].

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Drug Repurposing For All

Drug repurposing is the process of identifying a new use for an old medicine. Drug repurposing is the first stop on rare disease cure odysseys, but is not seen by TradBio as commercially viable. It opens the door to clinically actionable interventions that can provide real-world benefit even when little is known about a rare disease. Drug repurposing also serves as a launchpad to curative drug discovery approaches that target the root cause of disease. Family-driven decentralized 1-to-N observational trials that start with a pioneer patient are a dress rehearsal for future commercially sponsored, investigator-led clinical trials, allowing biomarkers, surrogate endpoints, and clinical outcome measures to be pressure-tested quickly and cost effectively in the wild.

As a Web3-native entity, Curetopia is a composable biotech that taps into networks of brick-and-mortar research service providers, some of which are companies. Perlara has spent the last decade successfully performing “drug repurposing as a service” for rare disease communities, focusing on inherited metabolic diseases (IMDs), which comprise 10-15% of all rare diseases. Examples of successful drug repurposing campaigns are highlighted on Perlara’s Substack Cure Odysseys [15]. The test-flight project on Curetopia is for an IMD called AARS2, details about which are on the Curetopia Projects page [16]. Perlara previously completed yeast-powered drug repurposing screens for two genes related to AARS2: FARS2 [17] and NARS1 [18].

After the test-flight yeast-powered drug repurposing project is completed and with community input, we will scale up to 60 IMDs comprising several end-to-end metabolic pathways in yeast, as described in the 2025 roadmap section below. Ultimately, it will be possible to leverage yeast, worms and flies for drug repurposing screens and achieve 90%+ coverage of all IMDs as part of the 2026 roadmap.

While model organisms like yeast are ideally suited for IMDs, Curetopia is also planning to deploy a completely disease-agnostic and country-agnostic drug repurposing strategy where any one of the 10,000+ rare diseases is in play, regardless of the size or geographical fragmentation of the rare disease community. Approximately ⅓ of all 10,000+ rare diseases are neurodevelopmental disorders (NDDs). Curetopia is currently in active discussions with two incredible partners to scale up drug repurposing for all rare disease communities.

On the patient advocacy side, CombinedBRAIN (CB) is an umbrella foundation with 120+ NDD rare disease community affiliates that share common research infrastructure like a biobank and a network of responsive clinicians willing to test drug repurposing candidates in collaboration with affected families.

On the biotech side, Unravel Biosciences is a Boston-based company that has developed a fully decentralized, direct-to-patient drug repurposing protocol called rareSHIFT that involves the at-home collection of nasal swabs on which gene expression analysis (RNAseq) is performed. A computational screen identifies drug repurposing candidates that restore healthy gene expression. Drug repurposing recommendations, in particular over-the-counter nutraceuticals and generic drugs available off-label, can be acted upon by families in consultation with their medical care team. Depending on the size of the Curetopia treasury, we can operationalize yeast-powered drug repurposing for IMDs and patient-powered drug repurposing for NDDs in parallel.

Beyond Drug Repurposing

Every rare disease could potentially benefit from drug repurposing, but the expectation is that drug repurposing therapies will not be curative because they don’t target the root cause of disease. In some instances, drug repurposing will actually lead to targeted therapies. For example, we serendipitously discovered that an old antifungal drug (anidulafungin) belonging to the echinocandin class is a cryptic pharmacological chaperone for a mitochondrial enzyme called MECR [19]. 

However, in most cases, we don’t expect drug repurposing to deliver a lasting cure because drug repurposing targets disease-compensating mechanisms but not necessarily the disease-causing protein. One can imagine using GenAI to optimize a repurposed drug creating a novel chemical entity (and attendant novel IP) with enhanced disease-modifying properties.

Genetic therapies that fix the root cause of disease will ultimately be needed. One of the most de-risked programmable therapeutic modalities are antisense oligonucleotides (ASOs). Starting with milasen, the world’s first individualized medicine created for one patient, rare disease parents have demonstrated that they can lead a team that advances ASO projects from inception to n-of-1 testing of their child [20, 21]. For example, Yiwei She, founder of Project Baby Lion, partnered with Creyon Bio to develop, in record time, an allele-specific knockdown ASO that targets a toxic gain-of-function mutation in a gene called TNPO2 [22]. Curetopia will develop plans to partner with Yiwei on scaling up the success of her son’s n-of-1 medicine leosen and treat newly diagnosed patients in the first year of life.

ASOs are just one of many programmable/designable therapeutic modalities. Gene therapy, mRNA therapy, and cell therapy are among the modalities in scope after Curetopia exhausts drug repurposing options. The 2026 roadmap will address the landscape beyond drug repurposing and leverage learnings and IP generated in 2025.

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Governance

Curetopia is currently set up as a Swiss non-profit association (“verein”). Curetopia is dedicated to accelerate the discovery of treatments and cures for rare diseases, and so all actions voted on and approved by the DAO must be consistent with the Verein’s purposes to be executed.

The DAO itself will work through the $CURES token, which is a governance token issued under Swiss law. According to its initial setup, holding a $CURES token is a utility token that enables voting on the actions of the DAO, one vote per token. Proposals for actions by the DAO will be posted publicly, discussed by the community, and then voted on by $CURES token holders.

Discussion and voting will be done in a manner which conforms to industry standards.

At present, this involves the following:

Ideation of new proposals typically begins in the network’s preferred group communication channel or collaborative online space;
Proposals with sufficient community interest are discussed on Commonwealth (commonwealth.im) and subject to informal voting
Following positive discussions and informal voting, proposals are formally posted to Snapshot (snapshot.org), with voting by $CURES token holders with a typical voting period of three to four days;
Shorter discussion and voting periods may be used in the case of urgent matters.

After votes have been successfully concluded, passed proposals will then be acted on the DAO.

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Tokenomics

There will be a total pool of 100,000,000 $CURES tokens. Inspired by the rare disease memecoin $MIRA [23], which launched on Solana on Christmas Day 2024, $CURES will serve as the initial rally point for rare disease communities seeking liquidity for research. 

$CURES tokens are allocated as follows:

• Core team — 20.0%
• Service providers — 7.0%
• Future token sales — 10.0%
• Community auction — 15.0%
• Community incentives — 7.0%
• Airdrop — 1.0%
• Treasury reserves — 40%

1. Core Team

   The core team will receive 20% of the total token supply, subject to a four-year linear vesting schedule with a one-year cliff. This means that no tokens will be distributed to the core team during the first year (the "cliff" period). After the cliff period, ¼ of the tokens become vested with the remaining ¾ of the tokens vesting linearly over the remaining three years.

2. Service Provider (BIO)

   BIO, as the service provider, will receive 7% of the total token supply with no vesting period. BIO provides essential services, including but not limited to:

• Support with tokenomics design and strategy
• Assistance with DAO formation and organizational structuring
• Legal and regulatory advisory
• Web3 technology and infrastructure support

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3. Future Token Sales
   10% of the token supply is reserved for future sales. These sales will occur at market price at the time of execution, with vesting conditions to be determined based on the specifics of each sale.
4. Initial Token Sale
   15% of the total token supply will be auctioned during the initial token sale. These tokens will be distributed with no vesting period.
5. Community Incentives
   To foster community engagement and incentivize participation, 7% of the token supply is allocated for community incentives. These tokens will also have no vesting period.
6. Airdrop
   To reward $BIO token holders, 1% of the total $CURES token supply will be airdropped following the initial auction. This airdrop will be distributed pro-rata based on their participation in the auction, with a maximum of 10% bonus tokens allocated to each participant.

Scalability

The Curetopia treasury will become evergreen at the confluence of three income-generating streams, namely: (1) income from direct-to-consumer commercial spinouts; (2) income from licensing deals with companies; (3) income from mission-aligned investments in companies or projects.

In regards to the first two income streams, rare disease research funded by Curetopia will result in IP ownership rights that may be monetized using established B2B and B2C business models: licensing deals with biopharma companies, and selling over-the-counter supplement stacks directly to consumers via a commercial spinout, respectively.

The initial focus on drug repurposing for groups of inherited metabolic diseases (IMDs) is no accident. Perlara has already demonstrated the potential for “platforms in a pill,” meaning one compound, or a combination of compounds, that can treat multiple rare diseases. Drug repurposing screens for IMDs consistently yield nutraceuticals as top clinical candidates, so the deck appears to be stacked in our favor.

Curetopia will follow in the go-to-market footsteps of the pioneering BioDAO VitaDAO, which recently launched a spinout company to market a direct-to-consumer nutraceutical product [24]. Bryan Johnson’s Blueprint is another proof point. With over a decade of experience in the space, Perlara is the logical partner for the clinical-stage drug development of IMD programs once there is sufficient safety and efficacy data from 1-to-N studies.

Partnering with Unravel Biosciences and Combined Brain will create the potential for multiple commercial codevelopment programs. Unravel is committed to working in partnership with patient communities and has already delivered in the clinic with two existing programs. Therefore Unravel would be an ideal investment opportunity for Curetopia, capturing upside potential in Unravel as an allied rare diseases platform company.

A note about the Priority Review Voucher (PRV) program [25], which provides a $150M market incentive to develop a medicine for any rare pediatric disease. The long-term value creation potential of Curetopia via the licensing and investment income streams depends on reauthorization of the PRV in the US, which failed to happen as part of an omnibus must-pass budget bill at the end of the last Congress. However, the emerging consensus is that PRV reauthorization will occur sometime in the first half of 2025.

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2025 Roadmap

Assuming a $2.5M treasury and 12 months of runway, Curetopia will focus on scaling up from one inherited metabolic disease to 60 inherited metabolic diseases (IMDs):

Q1 (January - March)

• Complete test-flight AARS2 drug repurposing screen using TargetMol library (started in Dec ‘24)
• $CURES token auction (February)
• Curetopia community votes on yeast-powered IMD drug repurposing proposal (February - March)

Q2 (April - June)

• Prepare 60 IMDs for the 8,400-compound TargetMol library screen using the existing high-throughput drug screening infrastructure
• Initiate AARS2 N-of-2 studies with two pioneer families and run for 12-24 weeks

Q3 (July - September)

• Complete TargetMol library screens for 60 IMDs
• Socialize results with patient communities and clinicians
• Share updates from AARS2 N-of-2 studies

Q4 (October - December)

• Multiple decentralized, parent-led 1-to-N clinical studies begin and run for 12-24 weeks
• Establish safety and validate biomarkers and outcome measures
• Community votes and approves 2026 Roadmap

The audacious goal for 2026 is to scale drug repurposing to 1,000 rare diseases spanning all aspects of human biology and representing 10% of all rare diseases. Given rapid and unpredictable advancements in AI, such a data corpus may represent a sufficient training set enabling generalization to the remaining 90% of rare diseases.

Disclaimer

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