Even the smallest mutations in our genetic code can have damaging effects, cascading into an array of disease pathologies. In an effort to negate these effects, VitaDAO, a key player in Decentralized Science, has partnered with Dr. Michael Torres and Artan BIO to engineer a first-in-class therapy that aims to suppress deleterious codon mutations. The VitaDAO community voted to provide $91,300 worth of funding to Dr. Torres via Molecule’s Intellectual Property Non-Fungible Token (IP-NFT) protocol. To increase the opportunity for community involvement and further funding, the IP-NFT will be tokenized into Intellectual Property Tokens (IPTs), granting governance privileges to IPT holders in the VitaDAO community and beyond.
What happens when DNA gets up to nonsense?
The ribosome, colloquially known as our protein printer, is continually walking itself down strands of genetic code, reading each building block like braille, and then summoning the corresponding amino acids, with the help of transfer RNA (tRNA). These amino acids string together, ultimately forming proteins. If a single building block, known as a nucleotide, mutates, it can alter the protein being created. Our proteins participate in every possible shade of bodily function, so changes in protein synthesis tend to cause a whole host of problems.
One such nucleotide change results in mutations known as nonsense mutations. They occur when a single nucleotide mutates and forms a stop codon, which is effectively a force-eject button. Stop codons are generally reserved for the end of the entire protein sequence when the protein is ready to be sent off into the body, but in the case of nonsense mutations, these stop codons accidentally crop up elsewhere. When this happens, protein formation is halted prematurely, resulting in half-formed, non-functional proteins which can go on to cause mayhem in the body. Finding and correcting nonsense mutations is not a novel concept. In fact, our bodies do it already! However, we’re far from perfect - issues arise when genuine stop codons are recognized as well, and overridden when they should not be. Currently, the existing treatments also fall prey to this issue.
The Science
To address the gap, Dr. Torres has invented a potential solution; an engineered system to suppress the outcome of a particular nonsense mutation, enabling the continuation of protein formation. His solution is unique in so far that it appears to be more discerning than other treatments, correctly identifying the problematic stop codon while ignoring genuine stop codons, and most importantly, not interfering with normal gene translation. Preliminary data produced in collaboration with Johns Hopkins University has supported Dr. Torres’ hypothesis, leading to VitaDAO’s decision to fund further investigation.
Additionally, Dr. Torres has identified a delivery system that is adept at sneaking into cells in the heart, liver, skeletal muscle, and central nervous system. By integrating the engineered codon suppressor into this vector, he suspects that it will be the perfect vehicle to carry the therapeutic to exactly where it needs to go. This vector comes with the bonus of already being clinically validated, which enables the team to optimize its path to first-in-human studies, as certain risks have already been addressed.
The Scientist
Dr. Torres, who holds a PhD in Cancer Biology from UT-Southwestern in Dallas, has previously co-founded ReCode Therapeutics, a clinical-stage genetic medicines company that has secured over $300M in funding. He now works as the CEO of CrossBridge Bio, an oncology-focused company focused on developing next-generation dual payload antibody-drug conjugates. Dr. Torres was introduced to Decentralized Science (DeSci) by longtime DeSci supporter Mike Baran from Pfizer Ventures, and was quickly attracted to the ability to leverage community engagement to critically evaluate and fund ideas. His extensive experience in drug development, as well as his aptitude for both science and entrepreneurship, make him the perfect candidate for VitaDAO and Molecule’s novel funding model.
“In essence, this model is a powerful tool for transforming ideas into tangible data, supporting future drug development, and creating valuable societal assets.” Dr. Michael Torres
A single solution for many diseases
As a seasoned drug developer, Dr. Torres understands the importance of having foresight when in the lab. In studying nonsense mutations, Dr. Torres observed that a specific nonsense mutation is prevalent in many proteins involved in cellular processes associated with aging such as DNA repair, epigenetic regulators, and tumor suppressors. As this mutation is conserved across many diseases, several clinical opportunities are possible. Under optimal conditions, this research has the potential to turn into a ubiquitous therapy that could be used to treat several health issues associated with aging. VitaDAO is particularly interested in the potential of a therapeutic that could reactivate tumor-suppressing proteins in cancer patients, aiming to slow down or even eradicate tumor growth, given their focus on age-related diseases. Some of the other diseases implicated with nonsense mutations are Duchenne muscular dystrophy, cystic fibrosis, spinal muscular atrophy, metabolic diseases, and neurologic disorders.
About VitaDAO
VitaDAO is a community-owned collective dedicated to funding and advancing longevity science that can improve people's lives. They have a strong presence in the Decentralized Science community and have utilized the IP-NFT model multiple times. In addition to this, together with Molecule and Dr. Viktor Korochuk, VitaDAO launched the first tokenized research project in the form of IPTs. The IPT tokens, known as VITA-FAST have enabled direct community governance - a world first.
Dr. Anthony Schwartz, a VitaDAO Entrepreneur in Residence, will act as the project manager for this project. He has founded at least 15 startups, primarily focused on autoimmune diseases and cancer, which have led to large financings and an FDA-approved product. He is also a lecturer at Johns Hopkins.
“The emerging VitaDAO model can potentially change how new drugs are funded. I'm excited and motivated to help bring that model to fruition through VitaDAO's funding of new companies and blockchain capital raises (IP-NFT/IPT) and seamlessly transition these companies into traditional VC/Pharma financing to bring new drugs to the market.” Dr. Anthony Schwartz
What is an Intellectual Property Non-Fungible Token?
Intellectual Property Non-Fungible Tokens (IP-NFTs) bring IP rights, such as the right to data and patents from drugs developed by funded research, onchain by attaching IP rights to smart contracts. By unifying data and intellectual property rights into programmable, transactable, blockchain tokens, IP-NFTs represent an evolutionary step in the development and management of scientific research IP. To date, IP-NFTs have been used to register IP of more than $1.95M in scientific research. The Torres IP-NFT adds to this growing list. You can find the IP-NFT here.
Intellectual Property Tokens invite the community to participate
“This revolutionary concept of IP tokenization allows community involvement in the drug development process, creating a mutually beneficial situation. For us, it enables unlocking additional capital in a collaborative manner. For the community, it gives them an opportunity to directly support projects in a manner previously unavailable.” Dr. Michael Torres
True to the belief of equitable and accessible scientific research, VitaDAO will be tokenizing the Torres IP-NFT into IPTs, fungible tokens enabling the distribution of IP rights to their holders, in order to raise funds for the science and empower the community to govern the resulting IP.
Funding from sales of the IPTs will enable Dr. Torres and his team to insert the engineered fragment into the chosen vector and perform in vitro testing to validate its effectiveness. IPTs will enable individuals to actively govern the project and its results, such as new drugs developed from the team’s research.
Granting community members the power to co-develop research projects harnesses the potential of incentive-aligned expertise, furthering decentralized science. Many decisions need to occur as this project unfolds, such as which other possible delivery vehicles to test, which disease indication is best suited for clinical testing, and how to raise additional funds. With the power of IP tokenization, the wisdom of the crowd now has the chance to influence those decisions.
