The Bio Report

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.

Nadia Harhen, an expert in drug development, discusses the challenges in drug discovery and the potential of quantum-inspired computing. Sandbox AQ aims to accelerate drug development, reduce costs, and improve clinical success rates. The podcast also explores the history and business model of Sandbox AQ, the impact of AI in drug development, and utilizing failed clinical trial data and AI to design specialized trials.

The podcast discusses the exploitation of chromosomal instability in cancer cells as a key difference from healthy cells. Volastra is developing therapies to target this vulnerability in difficult-to-treat cancers, with expanding relationships with Big Pharma. Topics include the prevalence of chromosomal instability in different tumor types, targeted therapies for cancer cell death and tumor regression, and the use of KF8 inhibitors in standalone or combination treatments.

The podcast discusses the biotech industry in 2023, including new drug approvals and M&A activity. The rise of Rama Swami, a biotech scientist turned politician, and the upcoming JP Morgan conference are also discussed. The speakers express interest in gene therapy business and the upcoming approval of the first drug for mash. They also talk about an upcoming event and the year ahead in biotech, including financing for companies and M&A trends.