Drug discovery 2.0 fuels the next-generation of life-saving medicines

“The future of drug discovery in biotechnology is dependent on achieving scale, speed and cost efficiency,” according to Dr. Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies.

In this exclusive interview, Dr. Benzeno outlines the unique opportunity for biotechs to transform the drug discovery process and develop the next wave of life-saving medicines to treat patients with cancer, autoimmune disorders and neurodegenerative diseases.

Listeners can expect to hear about the role of generative biology that combines artificial intelligence (AI) and machine learning (ML) with innovations in biology and the lab to fuel scientific progress. Dr. Benzeno talks about the identification of T-cell signatures of disease, which can accelerate the discovery of novel druggable targets in autoimmunity, an area in which few effective treatments and cures are available. Dr. Benzeno also highlights recent scientific breakthroughs that are revolutionizing the drug discovery process, including improvements in structural biology and therapeutic applications of gene editing to precisely modify and change DNA in living organisms.

Check out the full interview below to learn about how these cutting-edge innovations in drug discovery can enable the next wave of potentially life-saving medicines to be made available to patients more quickly and effectively.

Rebecca: Hi there, I’m Rebecca Willumson. I’m the publisher of Fierce Biotech. I’m here today with Dr. Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies. Thank you for joining me.

Sharon: Thanks for having me.

Rebecca: While we think about the future of biotech, where do you see the biggest opportunity for the industry moving forward?

Sharon: One of the most significant opportunities in biotech is to transform how we discover drugs to help improve the treatments of patients with cancer, autoimmune disorders and neurodegenerative diseases. The traditional drug discovery process has three challenges or bottlenecks: 1) it is difficult to scale; 2) it’s costly; and 3) it’s time consuming. The next big opportunity is to try to fix these three problems. The future of drug discovery in biotechnology is dependent on scale, speed and cost efficiency.

An area that is fueling this transformation in drug discovery is generative biology. Generative biology is the pairing of artificial intelligence and machine learning with innovations in biology and the lab.

Generative AI tools have a scalable effect on R&D. Today, we can do more R&D in silico. We can also predict all sorts of different biological characteristics, as well as generate more informative data and larger data sets, which can be applied to many more patients.

For example, at Adaptive, we’re combining our immune medicine platform with AI/ML modeling in partnership with Microsoft to accelerate the discovery and validation of T-cell signatures of disease through the identification of T-cell receptors (TCRs) that sit on those T-cells. In a given disease, for example, we can quickly identify a set of T-cell receptors that are found in many patients with that specific disease and are not found in either healthy controls or other diseases. We also know that those T cells specifically recognize disease antigens. That’s opened a way for us to rapidly narrow the universe of possibilities by focusing on just those T cells to discover and validate novel druggable targets in autoimmune disorders—an area in which treatments are suboptimal and there are no cures. And so, we’re quite optimistic and excited by what we and others are doing in this space.

Rebecca: Tell me which emerging technologies or scientific advancements do you believe will have the most significant impact on the industry in the years to come.

Sharon: We just discussed the power of generative biology, or generative AI. Maybe it’s worth expanding a little bit on the scientific advancements that we are seeing and using. Today, scientists and researchers can produce and make proteins from scratch, designing them to be even better than what’s found in nature. This can be done much more quickly by using predictions, which remove liabilities in those designs to make the proteins more suitable to be made into drugs. Another area that is revolutionizing the field of structural biology are prediction programs such as AlphaFold and RosettaFold. These programs allow us to look at protein structure in a whole new way.

At Adaptive, we’re using these programs with our proprietary AI and ML models to better understand the structural combination of a T-cell receptor, a peptide, and an MHC, which presents that peptide to that T-cell receptor.

Another scientific advancement making headway is gene editing. Gene editing is the ability to precisely modify and change the DNA, even to a single base edit, in living organisms. There are big expectations later this year on a landmark regulatory decision for the first gene-edited cell therapy by Vertex and CRISPR Therapeutics. That’s going to validate the entire gene editing space.

At Adaptive, we partnered with Genentech to advance TCR-based cell therapies in oncology. This cell therapy relies on gene editing by knocking out the endogenous T-cell receptor found on a patient’s own T cells and knocking in Adaptive-discovered TCRs, which are specific to that patient’s tumor mutations. This approach enables gene editing of multiple TCRs and potential targeting of multiple tumor mutations. We’re excited by the progress we’re making in this personalized approach. Earlier this year, we announced that Genentech received FDA IND clearance for the first Adaptive-discovered TCR engineered cell therapy product, which Genentech is advancing into the clinic.

Rebecca: During the last 18 months, we’ve seen biotech companies that have shut down or are trading below cash. What are your views on the current and future state of biotech financing and how is the current capital-constrained situation affecting early-stage biotech decisions—and how should it affect decisions moving forward?

Sharon: Between September 2020 and September 2021, with the arrival of COVID vaccines, we saw a public biotech indices run-up of between 35% to more than 50%. During the past two years, these indices have returned to their pre-pandemic levels as investors rotated out of our sector to take advantage of the post-pandemic economic recovery. Recent VC private funding is at a six-year low. And, the number of exit or monetization opportunities are at their lowest point in more than a decade. So far in 2023, there have been just 56 VC exits. Last year there were 89, compared to 195 in 2021. That’s the reality today – down more than 70% in the past two years. We’re also seeing, unfortunately, several companies shutting down because of capital expenditure (CapEx) burn, and less differentiated or inadequately differentiated pipelines and drugs.

However, we are also seeing some industry green shoots. We’re seeing more sizable M&A transactions, although certainly fewer than in the past in terms of the type and number of acquisitions. Recent well-received M&A activity include Pfizer’s $43 billion acquisition of Seagen and Merck’s $10.8 billion acquisition of Prometheus Biosciences this past spring. We’re also seeing more sizable single asset acquisitions. Earlier this year, Takeda closed its TYK2 program acquisition from Nimbus Therapeutics, which included a $4 billion upfront and an additional $2 billion to Nimbus Therapeutics following future milestones. Six months following this transaction, Nimbus Therapeutics raised a private financing round of more than $200 million. Those are just a few examples of green shoots bringing welcome positivity to our space.

Generally, innovation, good science and differentiated therapies with robust data will continue to be attractive to investors, as will renewed focus on prioritizing CapEx, R&D investments and building operating efficiencies.

My view is that the future of biotech is very bright. We're excited by the ability to develop and bring transformative and life-saving new therapies to more patients around the world.

Rebecca: That’s great. That feels like a perfect way to close out. Thank you for joining me today. I appreciate the conversation.

Sharon: Thank you.

This interview has been edited and condensed.

The editorial staff had no role in this post's creation.