The Bio Report

Jeremiah Robison’s daughter Sofia was born with cerebral palsy, a congenital movement disorder. Watching her physicians use body monitors to measure her gait, and later apply functional electrical stimulation as physical therapy, gave him an idea to combine the two to create a sleeve that could be worn on her leg to improve her ability to walk. Now Cionic, the company he co-founded, is producing its Neural Sleeves to help people with a variety of mobility impairments better navigate the world. We spoke to Robison, CEO of Cionic, about the Neural Sleeve, how functional electronical stimulation helps people with movement disorders walk, and how the use of adaptive algorithms continuously optimizes the stimulation it delivers.

Physicians treating people with burns typically incorrectly assess the depth of burn wounds in about 25 to 30 percent of cases. That, in part, is due to a lack of diagnostic tools to assess the severity of a burn and to determine the best approach to treatment. Spectral AI has developed the DeepView System, a predictive device that offers clinicians an objective and immediate assessment of a wound’s healing potential prior to treatment or other medical interventions. We spoke to Pete Carlson, CEO of Spectral AI, about the company’s AI-driven DeepView System, how it works, and how it changes outcomes for patients.

New York City is not the first place that comes to mind when thinking of biotech clusters, but the Big Apple has been a growing center of biomedical innovation with its mix of academic research hospitals, finance, and growing number of biopharmaceutical companies. One place that’s seeking to serve as both a home to innovative companies and facilitate collaborations is Cure, which describes itself as a healthcare innovation campus. We spoke to Seema Kumar, CEO of Cure, about how it operates, its various initiatives, and what can be done to accelerate the movement of innovative technologies from the lab to the marketplace.

Funding levels, drug launches, and R&D success rates all grew in 2023, according to a new report from the IQVIA Institute for Human Data Science. Global funding of biopharmaceutical research and development increased to $72 billion, up from $61 billion in 2022. M&A activity jumped to $140 billion from $78 billion during the same period. We spoke to Murray Aitken, executive director of the IQVIA Institute for Human Data Science, about its Global Trends in R&D 2024 report, what he’s watching, and why the new patent cliff for the industry will be less threatening than the last one.

In January 2023, AstraZeneca agreed to acquire CinCor Pharma, the CinRx cardio-renal disease therapy subsidiary, for $1.3 billion. The deal included a potential additional $500 million milestone payment. The price represented a 121 percent premium to CinCor’s market value at the time and could grow to more than a 200 percent premium if the milestone is met. The sale represented validation of CinRX’s portfolio approach to build multiple biotech companies supported with a dedicated funding mechanism. We spoke to Jon Isaacsohn, founder and CEO of CinRx, about the company’s portfolio approach, how its business model allows it to accelerate the development of needed medicines, and its efforts to develop new obesity therapies.

Antibody-drug conjugates can deliver chemotherapy directly to tumors but more than 95 percent of the dose often ends up in healthy tissues, decreasing efficacy and increasing toxicity. Mythic Therapeutics FateControl technology improves the uptake of ADCs in cancerous cells while avoiding the release of their toxic payloads in healthy ones. This promises to increase efficacy of these therapies in a wide range of cancers without causing unacceptable side-effects. We spoke to Brian Fiske, co-founder, chief scientific officer of Mythic, about the challenges that have held back the benefits of ADCs, how the company’s FateControl technology addresses those, and how it is thinking of building a pipeline of ADCs across indications.

The HGF/MET neurotrophic system plays a critical role in the maintenance and repair of connections in the brain. Enhanced signaling along this pathway has the potential to reduce inflammation, slow neurodegeneration, and provide neuroprotection. Athira Pharma is pursuing small molecule drugs that target this pathway to address a range of neurodegenerative conditions. The company is currently conducting a phase 2/3 study of its lead therapeutic candidate, fosgonimeton, in mild-to-moderate Alzheimer’s disease. But the study comes after the experimental therapy failed to meet its primary endpoint in a phase 2 study in Alzheimer’s disease and a phase 2 study in Parkinson’s disease dementia and dementia with Lewy bodies. We spoke to Rachel Lenington, chief operating officer of Athira Pharma, about the case for targeting the HGF/MET neurotrophic system in neurodegenerative diseases, how its experimental therapy fosgonimeton works, and why its continuing to pursue the therapy despite earlier failures.

The advent of induced pluripotent stem cells, cells that can be coaxed into becoming virtually any type of cell within the body, promised to usher in a new era of regenerative medicine and improved drug discovery. In practice, though, the ability to use these cells to develop desired cell types has proved challenging to do in a predictable way and at scale. Bit.bio, has developed a synthetic biology platform that it says allows it to industrialize this process and produce desired cells in a consistent manner. We spoke to Mark Kotter, founder and CEO of Bit.Bio, about the company’s platform technology, its effort to develop cell therapies, and its growing offering of precision reprogramed human cells for drug development.  

Some 100 trillion gigabytes of data are created and consumed each year, an amount that is expected to double by 2025. The demand for data storage carries the need for significant physical space and power requirements in the form of digital data centers. DNA, though, may represent a solution to what some see as unsustainable growth with environmental consequences. DNA, it turns out, is a dense and durable way to store information. At the end of last year, Paris-based Biomemory launched its DNA Cards, the first DNA data storage ever offered to the general public. The DNA memory cards, about the size of a credit card-sized today may seem more like a curiosity than a viable solution. At $1,000 each, they can store a kilobyte of data, enough to store a single, brief email. We spoke to Erfane Awani, founder and CEO of Biomemory, about the use of DNA for data storage, how it works, and how quickly the technology can scale to where it competes with today’s data storage infrastructure.

People tend to think of each person having their own unique genome. Quotient Therapeutics is taking an approach to drug discovery based on the reality that from cell to cell within a given individual, there can be trillions of divergent genomes. Changes in cells throughout the body can alter how a cell responds to disease and point to new ways to cure, treat, or prevent conditions. We spoke to Jacob Rubens, president of Quotient, about the company’s platform technology, how it uncovers genetic mutations at a cellular level, and its efforts to build a pipeline of therapies around the insights it gains.

Regardless of the cause of a disease, most diseases involve gene dysregulation. Omega Therapeutics is developing a new class of programmable, epigenomic, mRNA medicines designed to make specific epigenetic changes and correct abnormal gene expression to treat or cure diseases. We spoke to Mahesh Karande, president and CEO of Omega Therapeutics, about its pipeline of mRNA therapies, how they work, and its recently announced collaboration with Novo Nordisk to develop an epigenomic controller to treat obesity.

The effects of spinal cord injury extend beyond the loss of mobility. They can include a toll on mental health, a feeling of exclusion, and a general decline in the quality of life as a result of being able to stand and interact eye-to-eye with others. ReWalk Robotics’ Personal Exoskeleton is configured to an individual’s body and enables someone who has suffered a spinal cord injury to stand, walk, and even climb up and down stairs. We spoke to Larry Jasinski, CEO of ReWalk Robotics, about the company’s Personal Exoskeleton, how it works, and the impact it can have on the quality of life for someone with a spinal cord injury.

Mitochondria are often referred to as the “powerhouses” of the cell, but as scientists gain a greater understanding of these essential organelles, they are coming to discover they play a more expansive role in health and disease. The Foundation for the National Institutes of Health named Navdeep Chandel a co-recipient of the 2023 Lurie Prize in Biomedical Sciences for his research that revealed how mitochondria function as signaling organelles that control the body’s normal functions and impact diseases, including cancer and inflammation. We spoke to Chandel, professor of medicine in the Division of Pulmonary and Critical Care and of biochemistry and molecular genetics at the Feinberg School of Medicine, about the state of our understanding of mitochondria, why drug developers are pursuing therapies to target mitochondria across a broad range of diseases, and the need for a concerted effort to conduct fundamental research to better understand the biology of this organelle.

Sandbox AQ, the Alphabet spin-out backed with $500 million in investment, in June 2023 unveiled it AQBioSim division. The division is working to bring SandoxAQ’s AI and quantum-inspired computing to develop new treatments for intractable medical conditions including neurodegenerative diseases and cancer. The company is working with leading drug developers and university medical centers to accelerate drug development, reduce costs, and improve the rate of clinical success. We spoke to Nadia Harhen, general manager of simulation and optimization for SandboxAQ, about the challenges of drug development it is seeking to address, how Sandbox AQ is leveraging quantum technologies, and why it is seeking to tackle an intractable set of diseases. One note: As we were preparing to publish this episode, Sandbox AQ announced it acquired Good Chemistry, a computational chemistry company that leverages AI, quantum, and other advanced technologies to accelerate drug discovery. The deal is expected to enhance SandboxAQ’s exis

It’s long been understood that chromosomal instability (CIN) is a characteristic of cancer cells, but it also represents a key difference between cancer cells and healthy cells that can be exploited. Volastra is developing medicines for a wide range of difficult-to-treat cancers that target the vulnerabilities of these cells. We spoke to Charles Hugh-Jones, CEO of Volastra, about chromosomal instability in cancer cells, how the company is developing a pipeline of therapies to exploit this, and its expanding relationships with Big Pharma.

Though the year began with a banking crisis and has been marked with layoffs and restructurings, we have managed to avoid a feared recession. It’s been a big year for new drug approvals, M&A activity has been brisk, and biotech stocks have rallied in recent weeks pushing the widely watched S&P biotech index into positive territory. We continue our annual tradition to look back across the year in biotech and ahead to JPMorgan and beyond with Adam Feuerstein, Polk award-winning journalist and senior biotech writer for STAT. We spoke to Feuerstein about the biotech news that shaped 2023, the year’s best and worst CEOs, and what’s ahead in 2024.

Vik Bajaj has long lived at the intersection of physical sciences, engineering, data science, and the life sciences—first as an academic working to improve diagnostic imaging at Stanford University and later as a chief scientific officer at GRAIL and Verily. Now, as a founder and CEO of Foresite Labs, the technologist turned venture capitalist is using his experience to create new companies that are leveraging AI and other technologies to transform healthcare. We spoke to Bajaj about the changing data landscape around healthcare, the potential for technology to improve health outcomes, and what it takes for an entrepreneur to get his attention today.   

Interleukin-2 therapies have been seen as promising ways to treat solid tumors, but they have proven challenging because of the ability of IL-2 to both activate and suppress the immune system. Their effectiveness has been limited because of potentially toxic side effects, which have included vascular leak syndrome and pulmonary edema. Aulos Biosciences believes the AI-based approach to computational drug design used for its lead experimental therapy allows it to unlock the power of IL-2 without triggering the concerning side effects of existing therapies. We spoke to Aron Knickerbocker, president and CEO of Aulos Bioscience, about the potential for IL-2 therapies to treat solid tumors, the limits of today's IL-2 therapies, and the AI-based design behind its experimental IL-2 therapy in development.

Real-world data promises to provide insights that lead to better therapies, change how we treat diseases, and improve outcomes for patients. One of the limits of realizing benefits from all of the health information we are swimming in is the lack of access to high-quality data in a usable format. Verana Health has built partnerships with medical societies to provide access to exclusive, real-world data from a network of more than 20,000 clinicians. We spoke to Sujay Jadhav, CEO of Verana Health, about the potential for real-world data to improve our understanding of health, how Verana is addressing challenges these data can present, and the areas of medicine on which it is focused.

Artificial intelligence promises to reshape the healthcare landscape and deliver new insights into the molecular drivers of health and wellness, provide rapid diagnoses of patients, and discover and design therapies that extend beyond human imagination. Swaroop ‘Kittu’ Kolluri, founder and managing director of Neotribe Ventures, is using his experience as a Silicon Valley entrepreneur to invest in the emerging TechBio space. We spoke to Kolluri about the state of AI, the potential of technology to transform healthcare, and Neotribe’s approach to investing.