This three-part series includes discussions on new artificial intelligence (AI) platforms and their application in cancer, neurodegenerative diseases, non-alcoholic steatohepatitis (NASH), and rare diseases.

The first series of Front Row focused on Alzheimer’s disease. Here we meet a trio of pathbreakers at the forefront of research in AI for drug discovery and development. In parts two and three of this series, host Malorye Branca interviews Andy Beck, CEO of Path AI, and Daphne Koller, CEO of insitro.

Here in Part One, Malorye interviews Alice Zhang, CEO and founder of Verge Genomics. The Framework for this conversation is Using AI and Human Tissue to Break New Ground in Neuroscience.

As an MD/PhD candidate at Stanford, Zhang was inspired to leave early for industry. She realized one of the biggest bottlenecks in drug discovery and development is the drawbacks of animal and cell models and thought she could sidestep those by using AI and human tissue samples—lots of them. The key would be to get a better picture of what causes disease and what might be the best approaches to treat it.

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Verge Genomics now has more than 6,800 tissue samples already and that collection is growing every day. The company has partnered with some two dozen universities and brain banks to keep the samples flowing.  Verge says it has built an end-to-end, AI-driven drug discovery platform that includes one of the field’s largest proprietary genomic datasets from human brain tissue.”

In July 2021, the company announced a $706-million partnership with Lilly to develop new treatments for amyotrophic lateral sclerosis (ALS). Last December, it closed an oversubscribed $98-million equity financing.

Particularly exciting for Verge this year is that the company is aiming to launch a clinical trial of its first proprietary drug candidate, which addresses ALS and is based on a target the company found using its own platform. Another goal is to create gene therapies that address a wider range of patients.

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Having just sealed deals with Cleveland Clinic in March and with GlaxoSmithKline (GSK) in April, PathAI is having a successful start to 2022. The firm is only a few years old and already working with about 18 of the world’s top 20 biopharmas, according to CEO and co-founder Andy Beck, MD, PhD. In 2021, the company entered the diagnostics space by acquiring Poplar, an anatomic pathology laboratory services provider.

PathAI develops AI-powered research tools and services for pathology. The company has already collected more than one million slide images matched with expert pathologists’ annotations as well as partnerships with drug companies, labs, and about 400 board certified pathologists. The company launched their platform in 2017 and secured $165 million in Series C funding in 2021.

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The new deal with GSK is a strategic multi-year partnership in oncology and non-alcoholic steatohepatitis (NASH). The companies will leverage PathAI’s technologies in digital pathology including the use of PathAI’s AIM-NASH tool. NASH has emerged as a popular indication, both because of the increase in prevalence and the lack of available treatments.

With Cleveland Clinic, PathAI inked a five-year strategic collaboration to leverage its quantitative pathology algorithms both to conduct new translational research and for use as clinical diagnostics in multiple disease areas.

In part 2 of this series of Front Row, Malorye Branca talks to Beck about his company’s use of AI: Applying AI to NASH, an Emerging Opportunity with Huge Unmet Need.

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With healthy ($500 million plus) financing and major deals with pharma and Genomics England, insitro is one of the standouts of AI in drug discovery and development. Koller’s goal is simple: to revolutionize drug discovery and development. It’s a straightforward problem, she explains: “If you can reduce the probability of failure from 95–90%, you’ve doubled productivity.”

Not trained as a biologist, machine learning specialist Koller was drawn to drug discovery and development because it was more technological and aspirational than “telling spam from non-spam.” Koller has a PhD in computer science, was a Stanford professor for almost 20 years, and was co-founder and co-CEO of online learning site Corsera, among other endeavors.

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Human health became her passion though, and in 2018 she founded insitro, which combines induced pluripotent stem cells (iPSCs), genome editing, high content cellular phenotyping, machine learning, and other data-generating tools to build in vitro models of disease to be maximally predictive of human clinical outcomes.

Now, the company is applying that platform to data from partners like Genomics England, which has almost 150,000 whole genomes with corresponding phenotypic data from rare disease patients and their families. Genomics England also has multimodal and high-dimensional data, including histopathology images from its cohort of cancer patients.

In the final part of this second series of Front Row, Malorye Branca interviews Koller to learn about Advancing AI in Rare Disease and Cancer.

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Front Row, hosted by contributing editor Malorye Branca, takes us to the frontlines of research in a key area of biomedicine, talking to newsmakers and thought leaders about their research and the current challenges facing their field.

In this inaugural three-part series, Branca looks at the current state of Alzheimer’s disease research. Her special guests are three leading and talented scientists at the forefront of research:

Part 1 – Rudy Tanzi, PhD (Mass General Hospital, Boston) – New Genetic Findings

Part 2 – R. Nolan Townsend (CEO LEXEO Therapeutics) – Pioneering Gene Therapy

Part 3 – Hank Zetterberg (Guttenberg University) – Blood Biomarkers for Alzheimer’s

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In Part 1 of this series—“New Genetic Findings”—Branca interviews Rudy Tanzi, PhD, one of the leading and widely respected researchers in the field of Alzheimer’s disease for more than three decades.

A pioneer in the genetics of Alzheimer’s, a founder and/or advisor to multiple biotech companies, co-director of the McCance Center for Brain Health at Mass General Hospital in Boston, and a successful book author, Tanzi has strong views about the lack of translational success in turning genetics insights into meaningful therapies.

Gathering evidence from whole-genome sequencing studies and 3D Alzheimer’s-in-a-dish models, Tanzi stresses the power of genome analysis and explains where he thinks the pharma industry has gone wrong. He lays out the key steps in disease pathogenesis and the early promise in clinical trials of new therapies that are as much about when to target the disease as what or where.

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In Part 2 of this series on Breakthroughs in Alzheimer’s disease research—“Pioneering Gene Therapy”—Branca interviews R. Nolan Townsend, the CEO of LEXEO Therapeutics, a biotech company founded by gene therapy pioneer Ron Crystal. Townsend joined LEXEO Therapeutics as CEO in January 2020, having previously served as President of Pfizer Rare Disease. He has a strong financial background, previously working in the healthcare investment banking group at Lehman Brothers.

At LEXEO, Townsend is leading a multi-pronged gene therapy pipeline tackling several neurodegenerative diseases, including Batten disease, Friedreich’s ataxia, and Alzheimer’s disease. The approach to treating Alzheimer’s is a fascinating one, targeting apolipoprotein E (APOE), which serves as the major transporter of cholesterol in the brain. Variants in this gene on chromosome 19, specifically APOE4, are strongly associated with increased risk for Alzheimer’s disease. LEXEO is developing an AAV-mediated gene therapy treatment that delivers APOE2 to the patient’s central nervous system.

In this interview, Townsend shares the rationale and progress that his company is making in this quest to develop a therapeutic breakthrough for Alzheimer’s disease.

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In the third episode of this series—“Blood Biomarkers for Alzheimer’s”—Branca interviews Henrick Zetterberg, MD, PhD, Professor of Neurochemistry at the University of Gothenburg, in Sweden. Zetterberg discusses the exciting progress in recent years, from his group and others, on the development of blood biomarkers, which could help deliver treatment to patients earlier and save neurons. Zetterberg’s team studies many neurological diseases in addition to Alzheimer’s. He describes how technological advances have improved the detection of circulating biomarkers at very low levels, and how that information is being used diagnostically and therapeutically.

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