https://vibebio.substack.com/p/the-next-wave-of-biotechnology?utm_source=substack&utm_medium=email&utm_content=new-from-twitter-with-post
The trillion dollar global biotech industry has a major problem preventing it from flourishing into the holy grail of patient-driven personalized medicine. Thanks to leaps in technical advancements, including genomics and artificial intelligence, finding innovative therapies to cure patients is no longer the limiting factor. Rather, the overwhelming issue that most biotech research and development teams face is lack of adequate funding to run human trials. This is especially evident in rare-disease research where, by definition, there are a limited number of patients who are affected by any given disease. According to the Orphan Drug Act of 1983, any disease, condition, or illness affecting fewer than 200,000 people in the United States is considered a rare disease, otherwise known as an orphan indication.
At least 10,867 rare diseases have been cataloged, and this number continues to grow as diagnostic technologies improve. While the vast opportunity for drug development is apparent, and the cost to bring rare disease treatments through human clinical trials is lower than the industry average, the amount of private capital invested towards these programs pales in comparison to their common-disease counterparts. With the exponential progress we’re seeing in biotech research, curing patients with rare diseases won’t even be considered miracles in the near future - simply systems biology hacks of the human body.
Although government grants, private equity, and corporate partnerships currently supply biotech with the capital to conduct research and development, these traditional funding sources are often misaligned with patient community interests. Early-stage investors are obligated to heavily consider the likely ROI when supporting new ventures, and this threshold might not be reached for programs that are designed for the relatively small market share any individual rare disease represents.
Considering that 1 in 10 Americans suffer from a rare disease and fewer than 10% of these conditions have FDA-approved treatment, it’s time to put these historically overlooked patients in the driver’s seat for prioritizing drug development.
The emergence of blockchain technology1 and community-driven funding models create a new paradigm for doing just this. Biotech venture capital networks are also seeing the value of improved funding transparency, as observed in BIOS Community’s top 100 TechBio VCs list.
Today, the biotech community has the unique opportunity to utilize blockchain technology- the next wave of decentralized infrastructure (often referred to as Web3 2) to encourage early relationships between the producers and the consumers of life-saving drugs. Enabled by this new suite of tools, a future state could allow all stakeholders (including patients) to be aligned towards the common goal of developing rare disease therapeutics. Early-stage rare-disease biotech companies can raise capital more quickly, directly from the people who want it most. Decentralized communities of patients, scientists, and partners can collaboratively leverage shared resources, streamlining many of the time- and labor-intensive aspects of drug discovery. The passion, the market, and the resources already exist; novel blockchain-enabled DAO’s or decentralized autonomous organization infrastructure brings them all together.
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We already see major Patient Advocacy Organizations (PAOs) starting to take more influential roles in facilitating capital investments into disease-focused biotech groups. PAOs also exert influence on health policy which often determines the urgency of research development. Additionally, patient members are a great subject pool for future clinical trials recruitment. The activation of patient communities coupled with the power of transparent DAOs is a fundamental component in the next wave of biotechnology set to speed up the entire clinical approval process. As an example, the shift towards increased collaboration among Rare Disease Patient Organizations and the biotech industry is producing clear benefits to accelerate therapeutics innovation across rare disease neurological conditions yet their potential to disrupt biotech human clinical trials funding is still just on the horizon.
Let’s consider the next wave of biotech in the lens of Bill Gates’s famous quote,
“Most people overestimate what they can achieve in a year and underestimate what they can achieve in ten years.”
While potential therapeutics have historically taken around ten years to progress from discovery to approval, we’ve never before seen such a groundswell of enthusiasm by eager investigators and active patient advocates working optimistically in parallel.
In just the past few years, at least 117 Decentralized Science (DeSci) organizations have been established, each with their own unique community mission. While the hype and excitement from Web2 to Web3 has drawn some criticism from veteran biotechs, the real-world value that blockchain technology can unlock cannot be ignored in the long term.
Ecosystem landscape updated and curated by UltraRare Bio
