Dr. Derrick Rossi is a leading innovator doing work on some of today’s most promising and exciting therapies in the life sciences. Dr. Rossi, currently the CEO of the biotech startup Convelo, also helped found three other promising startups that are working to bring advanced treatments to patients: Moderna, Magenta Therapeutics, and Intellia Therapeutics.
These treatments promise to use stem-cell science, gene editing, and other advanced sciences to bring long-hoped for treatments and cures to patients. They are fundamental enough to be applicable to a wide range of conditions, including cancer, multiple sclerosis, Zika, heart failure, and diseases affecting the liver, eyes, muscles, and central nervous system.
According to an interview with The Alliance of U.S. Startups & Inventors for Jobs, while his primary focus has been transformative science that promises to help patients, he cites intellectual property (IP) as the foundation that enables him and his colleagues to bring research from lab to market to patients. Dr. Rossi has seen innovation from several perspectives, starting with its birth in a university lab, to spin out to a startup, to successful public offering. He served as a faculty member at Harvard’s Medical School and its Department of Stem Cell and Regenerative Biology, doing stem cell research. After helping to commercialize his own work as an academic founder of several startups, he eventually left academia to serve as CEO of Convelo.
He began his work as a researcher but was always directed toward the practical goal of delivering treatments. His passion is good science that leads to treatments for patients with unmet clinical needs. Reflecting on his success so far and the promise it holds for patients, he observed that, “It’s fun to think about how simply reading a cool paper on pluripotent stem cell science could lead to all of this.”
Dr. Rossi and his research have been at the heart of several transformative companies, including: Moderna, which arose from Dr. Rossi’s work on what he characterized as a “side project” based on his interest in the work of Nobel Prize winner, Shinya Yamanaka, who first showed that mature cells could be reprogrammed back to their embryonic state. Dr. Rossi hoped to use this discovery to create treatments that used the human body’s own ability to produce therapeutic proteins, but there were many obstacles to overcome. Dr. Rossi and his team engaged in laborious research and ultimately discovered a way to modify messenger RNA (mRNA) so that the body no longer recognizes it as a viral attack. This allowed them to use mRNA to convey “instructions” to induce cells to make a desired protein, including, crucially, antibodies to fight viruses. The potential of this innovation is vast. Since it can be used to express any protein, it could treat thousands of diseases.
What excites Rossi is that Moderna has over 20 treatments in clinical development. It has achieved multiple successful phase 1 trials and has ongoing phase 2 trials for treatments such as cancer vaccines and treatments for rare diseases. He says, “when we finally make an improved medicine, then I will be satisfied.” Recently, Moderna has become the topic of worldwide attention and frequent news for deploying what many consider to be the most effective and safest COVID-19 vaccine.
Once Dr. Rossi had the experience of, “doing work that could be spun into a biotech and brought to patients,” he found it, “addictive.” And so, he has continued to innovate. Magenta was launched in 2016 with venture funding to try to cure blood cancers and other disorders, autoimmune diseases and other genetic diseases that have shown a response to an immune system reset through bone marrow transplant. Unfortunately, these transplants are unavailable for many patients and conditions, and the ones that receive them endure serious side effects. Dr. Rossi saw a need for a change. He explained that ,“bone marrow transplants can be lifesaving, but they are also risky. One-year survival rates for patients with matched donors is 70% — unmatched is 55%.”
Magenta has developed a much more effective transplant regime, that combines a specially targeted antibody with a drug to prepare the patient for transplant. This development could allow the use of stem-cell transplants for a much wider range of disorders. The therapy even shows promise for transplants from unmatched donors.
Intellia is now a publicly held company in the early stages of clinical trials. It seeks to apply CRISPR/Cas9 technology to cure a number of diseases including various cancers and other diseases affecting the liver, eyes, muscles, and central nervous system.
Today, Intellia and Regeneron Pharmaceuticals reported positive interim results from the Phase I clinical trial of their CRISPR therapy candidate, NTLA-2001, to treat transthyretin (ATTR) amyloidosis.
Conducted by Intellia, the ongoing trial involves adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) in the UK and New Zealand.
Convelo is working to develop treatments to address the deterioration of the material that sheaths nerve fibers. This deterioration plays a role in multiple sclerosis and other nervous system disorders. Regeneration of myelin does not happen naturally, so a treatment that stimulated this regrowth would be transformative for many patients. Convelo is partnering with larger life science companies to develop a plan for commercialization which is essential to bringing such a treatment to patients. “Moving drugs toward people is the goal. MS trials are big expensive trials, and anyone ultimately bringing an MS drug to market will need to spend hundreds of millions.”
Dr. Rossi focuses first on strong science and unmet clinical needs, but he observes that IP is a support and foundation for the work of bringing new treatments to patients. He said, “you can be working on the coolest thing, but investors need to know that there is some protection for their investment, plain and simple.”
For a synthetic biology startup and its investors, compelling science is the threshold condition, but the IP is “the future prospect that reassures investors.” For Convelo, he wanted a robust portfolio of patent filings before engaging with pharmaceutical companies. It was not only to protect the company’s work prior to talking to potential investors, “but also to assure pharma that we had protectable assets. I put a massive push on this from day one.” Dr. Rossi observed that while patents are essential to bringing a treatment from lab to patients, neither the final version of that treatment, nor the patents that protect it are likely to emerge from an academic setting. It is often the case that university research discovers the first step toward a treatment, but rarely, if ever, the particular treatment itself. Correspondingly, there is no patent on the yet-to-be developed treatment. Instead, there is both early-stage research and IP.
Convelo was a classic example of a startup built on early, foundational research that needed further development. The very early research that Convelo had licensed from Case Western showed a platform for drug discovery, but not a drug itself. Convelo would need to combine investment capital, a high performance team and significant additional IP to find and develop an effective treatment to launch. Dr. Rossi says, “As often is the case, you get that IP to get the initial investment, but you know full well that the real value will come post-launch of the company.”
In any event, said Dr. Rossi, “early patents have limited life because of how long it takes to market.” Clinical trials can consume many years of patent life before the drug ever gets to the commercial market.
Source: company reports and The Alliance of U.S. Startups & Inventors for Jobs