This is an exciting yet perplexing time for biotechnology. The industry delivered RNA vaccines to save countless lives during the COVID-19 pandemic and has created impressive early clinical results using CRISPR gene editing to treat patients with deadly genetic diseases. But the sector has been tarnished with safety concerns, drug failures, and layoffs.
To put all the industry’s ups and downs into perspective, the editors of GEN convened an all-star group of entrepreneurs, innovators, and commentators for two days of candid conversation regarding the latest breakthroughs, trends, and challenges across all facets of the biotechnology industry.
“The State of Biotech” was a celebration of innovation, emerging technologies, and clinical successes, as well as an appraisal of the headwinds and challenges facing the industry.
Progress in precision genome editing
The event featured a headline interview with Jennifer Doudna, PhD, CRISPR pioneer and winner of the 2020 Nobel Prize for Chemistry (with Emmanuelle Charpentier, PhD). Doudna talked about what it was like to win the award, how it has changed her life, and the importance it has for women in science. She also discussed her hopes for the second decade of CRISPR gene editing and where she sees genome editing making its biggest impact in the future—in the clinic, in agricultural biotech, and beyond. She insisted that in vivo gene delivery was a high priority for therapeutics, but she would not be drawn on when such delivery would become routine.
Another pair of precision genome editing leaders—John Evans, CEO of Beam Therapeutics, and Doudna’s colleague Fyodor Urnov, PhD, scientific director of the Innovative Genomics Institute—discussed recent innovations in genome editing technologies, including epigenome, base, and prime editing, and described how these technologies are inspiring the development of next-generation drugs, diagnostics, foods, fuels, and other products.
“I think we’re in the middle of a ‘Cambrian explosion’ of new tools,” Evans said. “That diversity is great,” Urnov agreed. “It’s great for patients, and it’s great for the acceleration of the entire field. The more, the merrier in the genomic therapy space. I don’t see it as a zero-sum game.”
Synthetic biology’s sustainability goals
Another field that has seen impressive growth over the past few years, both in scientific innovation and in financing, is synthetic biology. At the heart of the field is a mission to use biology to build a better, more sustainable way of living.
Emily Leproust, PhD, co-founder and CEO of Twist Bioscience, Eben Bayer, co-founder and CEO of Ecovative Design, and Deepak Dugar, PhD, founder and CEO of Visolis, discussed how their synthetic biology companies are contributing to this overarching goal in diverse ways. “We cover bacon to data storage,” Leproust declared. “So, it’s a really good spectrum!”
Bayer pointed out that enthusiasm for addressing sustainability issues such as climate change is palpable. “We see more and more folks who want to work with purpose,” he related. “There’s so much opportunity in this space.”
Several experts emphasized the importance of artificial intelligence (AI) in synthetic biology. AI, they said, is helping biotechnology address several global issues—infectious diseases, aging, food and energy sustainability, and climate change. Colin Hill, co-founder and CEO of GNS Healthcare, Andrew Hopkins, PhD, founder and CEO of Exscientia, and Daphne Koller, PhD, founder and CEO of Insitro, discussed how biotechnology companies are using AI to accelerate R&D, analyze large databases, improve decision-making, and reduce costs.
Emerging business opportunities
It’s been a busy and exciting year for next-generation sequencing. At least three new companies have entered the U.S. market, which has been dominated by Illumina for the past decade.
Eli Glezer, PhD, co-founder and CSO of Singular Genomics, and Shawn Levy, PhD, senior vice president at Element Biosciences, dug into the impact that these emerging platforms and new technologies will have in this space, what their priorities are, and how they plan to carve a niche for themselves.
Next-generation sequencing is also among the technologies of interest to ARK Invest, a high-profile investment firm. Two of ARK Invest’s senior analysts, Simon Barnett and Ali Urman, discussed how the firm came to focus on disruptive public companies, how it works with portfolio companies to advance innovation, and how it identifies technologies of interest.
Barnett noted that the firm has been looking to invest in private companies as well, particularly startups that have promising technologies and enough business expertise to commercialize them. “We want to continue to innovate with new types of vehicles … offering investors the opportunity to get in even earlier,” Barnett said.
“The State of Biotech” also featured an engaging conversation with the three co-hosts of the popular weekly “Biotech Clubhouse” podcast—Brad Loncar, CEO of Loncar Investments, Daphne Zohar, founder and CEO of PureTech Health, and Chris Garabedian, CEO of Xontogeny.
Predicting the future of the markets is a tricky undertaking. “It could take a while before we get back to the ‘hyper-bull’ market that we were in,” Garabedian stated. Loncar agreed. “We were in a bubble a year and a half ago,” he said. “With the pandemic and biotech being front and center every single day, money was thrown indiscriminately at our sector, and it caused the current crop of existing biotech companies to get out of control.”
“That piece will sort itself out,” Zohar added. “The ones that make it through this tough period will be really high-quality companies.”
Lessons from the pandemic
Vaccine scientist and infectious disease expert Peter Hotez, MD, PhD, professor at Baylor College of Medicine, shared lessons that researchers and public health professionals have learned from COVID-19. Hotez, a frequent TV commentator on COVID-19 issues, assessed the success of FDA-approved vaccines, explained how his team co-developed a COVID-19 vaccine for use in developing countries, and discussed current public health challenges ranging from monkeypox to the spread of misinformation.
Hotez warned that the pandemic is not yet over—a bold prediction at the time the interview was recorded. “There is a possibility we’ll see yet another entirely new variant arise later in the fall,” he said. “[We’re] waiting for the other shoe to drop and trying to encourage people to max out their vaccinations. It’s still a serious wave.”
One of the pioneers of mRNA vaccines, Bob Langer, ScD, professor at MIT, was another major attraction during the event. He related how he first became interested in mRNA and Moderna, and he discussed his other scientific interests (such as tissue engineering and drug delivery), the researchers who have had a significant influence on his career, and his fascination with magic.
Meanwhile, in the virtual GEN Edge Lounge, Larry Worden, founder and principal, IVD Logix, gave a presentation entitled “How COVID-19 Forever Changed the In Vitro Diagnostics Marketplace.” He discussed how COVID-19 put a spotlight on clinical laboratory testing and the IVD industry, how this focus impacted non-COVID-19 testing, the trend toward at-home diagnostic testing, the rapid emergence and expansion of new companies and manufacturing capacity for diagnostic testing platforms, and the anticipated reemergence of traditional market dynamics.
From social justice to criminal justice
Sickle-cell disease (SCD) is a common genetic disorder that disproportionally affects Africans and those of African descent. “Anything that we can do … to help these patients—I’m signed up for that,” said Ted Love, MD, president and CEO of Global Blood Therapeutics (GBT). Over the past decade, GBT has been developing small-molecule drugs to treat the root cause of SCD. That quest led to FDA approval of the company’s first drug, voxelotor (Oxbryta), in 2019.
Love discussed the GBT drug pipeline, pricing considerations for the SCD community, and ongoing clinical trials. He also set this progress in perspective as he talked about decades of discrimination and racism against SCD patients, affecting everything from pain management and access to drugs to disparities in funding.
Last October, GBT was acquired in a $5.4 billion deal that was recently completed by Pfizer. “Although we have focused on using great science to make very powerful medicines to treat SCD, we’ve taken our social justice mission very seriously,” Love insisted. “We’re excited now that Pfizer will take up the mantle and hopefully accelerate our mission.”
Another form of justice was on the mind of David Mittelman, PhD, founder and CEO of Othram. “There are so many ways to do good in the world,” he said. “Othram uses DNA testing and advanced genomics not in biomedicine—but in this really interesting field of forensics.”
Othram’s crime-solving technology—based on advances in next-generation sequencing, informatics, and genealogy—has been critical in helping police departments solve a string of high-profile cold cases, some dating back decades. Mittelman, who helped GEN publisher Mary Ann Liebert launch the journal Forensic Genomics in 2021, shared recent highlights from Othram’s critical work solving cold cases, many of which have made national headlines.
Research on human aging is another hot field with growing interest and funding. Hana El-Samad, PhD, and Morgan Levine, PhD, from Altos Labs, a new biotechnology company that is focused on cellular rejuvenation programming to restore cell health and resilience, discussed how aging is defined in scientific terms and whether human aging can be slowed or stopped.
“We’ve increased life expectancy, but we haven’t moved the needle much when it comes to healthspan,” Levine noted. “People are living longer with diseases and chronic conditions. We need to focus on extending the time that people are optimally functioning.”
El-Samad—the founding editor-in-chief of GEN’s new sister journal GEN Biotechnology—pointed out that aging is biologically complex and that this must be acknowledged in the approach to research. She concluded, “We need to move away from the concept of a linear pathway and toward the concept of interconnected systems.”
“The State of Biotech” was held September 21–22, 2022. In addition to the talks highlighted in this article, the event featured some 16 corporate showcase presentations. Participating companies included BiomX, Cardea Bio, Deepcell, Element Bio, Generate Biomedicines, Lexeo Therapeutics, Metagenomi, Moma Therapeutics, Neurosense, Pairwise, Profound Therapeutics, Satellite Bio, Scribe Therapeutics, Synthego, Synthekine, and Tome Biosciences. A replay is available on demand at https://webinars.liebertpub.com/e/the-state-of-biotech-2022.
A replay of the event (and every thought leader talk) is available on demand at: webinars.liebertpub.com/e/the-state-of-biotech-2022.