2007 saw the launch of numerous groundbreaking technologies. The iPhone. YouTube. Twitter. Kindle. Streaming on Netflix. The Amazon Kindle. Bitcoin.
A similar sea change is under way for the biotechnology industry, according to Hari Pujar, Ph.D., Operating Partner at Flagship Pioneering and Chief Operating Officer of Tessera Therapeutics, in the opening plenary of the 2024 AAPS National Biotechnology Conference.
“I think we’re at another time in history when we’ll look back in ten to 15 years at a revolution in the making,” he said.
He then shared data showing that the number of drugs developed per billion dollars of R&D has consistently gone down even as other technologies have expanded.
“Collectively, as an industry, we’re not making much,” Pujar said, citing a 2018 Endpoints News article. “The only way to solve this problem is to develop new modalities.”
New modalities in the biotechnology space have traditionally led to further innovative products.
“DNA goes to RNA and RNA goes to proteins,” he said.
In addition, “we’re getting a better understanding of the genetic basis of disease.”
Interest in cell and gene therapies continues to skyrocket with a rising number of products approved by FDA. He briefly touched on how these new therapies present manufacturing challenges.
“As we get into these new therapies, manufacturing becomes important. It is complex and costly to manufacture.”
Next, Pujar covered the exponential growth in mRNA therapeutic products. These were considered “unstable” in 2010 but by 2020, an mRNA vaccine was approved by the U.S. FDA for COVID-19. Oligonucleotides are seeing a similar rapid growth.
In light of these developments, “2024 is likely to launch another revolution. Certainly, the world is changing at a pace we don’t realize.”
Venture Creation for Bioplatforms
Pujar detailed Flagship Pioneering’s background and organization.
Flagship Pioneering builds companies driving breakthroughs in human health through bioplatforms. The company’s model is to encourage innovative thinking and entrepreneurship.
“The idea here is we are sampling the uncertain space,” Pujar explained. “There may be opportunities of low risk or high risk. We seek to capture low risk opportunities.”
This leads to a four-phase model:
Phase 1 Exploration: A “what if” question leads to a hypothesis. A team of scientists conducts initial research into the “exploration” for potential feasibility.
Phase 2 ProtoCompany: Promising explorations become proto-companies. Research and tests show if a proto-company concept is feasible. Proto-companies that do not show sufficient potential are discontinued.
Phase 3 New Company: A proto-type company that sufficiently answers to “what if” question becomes a new company under Flagship. New companies receive funding to develop proprietary bioplatforms.
Phase 4: Growth Company: Successful new companies attract outside investors and become growth companies, often going public.
Moderna, Ring Therapeutics, and Sana Biotechnology are some of the companies to come out of this process.
Potential for One-Time Cures
The final third of Pujar’s presentation covered Tessera Therapeutics, the Flagship company of which he is Chief Operating Officer.
As he explained it, the mission of this company is to cure diseases by “writing in the code of life” with Tessera’s Gene WritingTM platform, which currently consists of two Gene WritersTM.
RNA Gene Writer technology can write or rewrite a genome with a protein delivered as RNA to change base pairs, make small insertions, delete or replace genes, and integrate entire genes. DNA Gene Writer technology can make long-lasting changes to a genome via DNA template.
Pujar sees the potential of Gene WritingTM as providing one-time, permanent cures, often with same-day manufacturing of the therapeutic.
“The goal of RNA and DNA Gene Writing is that it is permanent,” he said.
Ultimately, he views Tessera Therapeutics’ technology as an industry game changer part of the currently expanding universe of new modalities, much like how 2007 saw the launch of the iPhone and similar platforms.
“The ability to engineer genomes will be a defining technology of this generation.”