The Bio Report

Using AI to Discover Small Molecule Alternatives to Biologics

One of the promises of AI is to redefine what’s possible by enabling the discovery of compounds that exist in a much larger chemical space than scientists have previously been able to explore. Deepcure is using AI and physics to discover small molecule therapies that can bind to difficult to target proteins. We spoke to Kfir Schreiber, co-founder and CEO of Deepcure, about the company’s AI technology platform, its focus on autoimmune diseases, and why he believes its technology will allow it to develop small molecule drugs that can provide alternatives to biologics to treat these conditions.

A BET on a Novel Approach to Treat Autoimmune Conditions

The BET family of proteins regulates gene expression. Their overexpression has been implicated in both cancer and inflammatory diseases. Vyne Therapeutics is developing oral and topical BET inhibitors that treat inflammatory conditions ranging from rheumatoid arthritis to the skin condition vitiligo. We spoke to Vyne CEO David Domzalski and Vyne Chief Scientific Officer Iain Stuart, about the role BET plays in autoimmune diseases, its platform technology, and its efforts to develop BET inhibitors that are both potent and specific.

Cell Therapies that Can Do a Solid for People with Cancer

In August, the U.S. Food and Drug Administration approved Adaptimmune’s Tecelra, the first engineered cell therapy to treat a solid tumor. The T cell receptor gene therapy is approved to treat synovial sarcoma, a rare soft tissue cancer that most often affects young adults. We spoke to Adrian Rawcliffe, CEO of Adaptimmune, about the approval, how TCR therapies differ from CAR T therapies, and what other cancers might benefit from such an approach.  

Going to Extremes to Discover New Drugs

Hibernation is not just a matter of deep sleep. Animals that hibernate are able to do so without suffering damage to tissue and muscle. Understanding the biology of hibernation can unlock potential insights into obesity, heart attack and stroke, muscle atrophy, neuroprotection, and longevity. Fauna Bio is studying genomic data from so-called "extreme mammals" and applying its proprietary AI platform to perform comparative genetic analysis to find gene-disease links and leverage millions of years of evolutionary adaptations to identify new therapeutic opportunities for human health. We spoke to Ashley Zehnder, founder and CEO of Fauna Bio, about the types of insights that can be gleaned from the genetics of hibernating animals, the wide range of human health conditions that might be addressed with such an approach, and the company’s deal with Eli Lilly to apply its AI platform to discover new obesity drugs.

Targeting Senescent Cells to Treat Aging-Related Diseases

Senescent cells, ones that no longer divide but are metabolically active, are associated with aging. They are also implicated in a broad range of aging-related diseases including cancer, diabetes, and neurodegenerative conditions. In the case of cancer, these cells can help protect tumors from a person’s immune system. Immorta Bio is seeking to address aging-related diseases by targeting senescent cells and killing them. We spoke to Thomas Ichim, president and chief scientific officer of Immorta Bio, about aging-related diseases, the role senescent cells play in these conditions, and why the company’s therapeutic approach may also have promise of addressing aging itself and extending healthy years of life.

Bringing Cellular Diversity into Sharper Focus

From cell to cell within a given tissue, variability exists. Single-cell sequencing technologies from 10X Genomics is helping researchers and drug developers understand cellular diversity in tissue once thought to be made up of homogenous populations. This has the potential to lead to new understandings of diseases, open up new targets for drug developers, and provide for more tailored approaches to the treatment of diseases. We spoke to Michael Schnall-Levin, chief technology officer and founding scientist of 10X Genomics, about the company’s platform technologies, the diversity of the cellular populations within the body, and how this is changing our understanding of health and disease.

Developing Cancer Therapies for When Damage Control Goes Awry

The DDR pathway plays a critical role in repairing DNA damage in healthy cells that would otherwise cause mutations or cell death. When this pathway is altered and becomes unable to repair this damage, mutated cells can grow out of control and become cancerous. They can also be more resistant to standard chemotherapies and radiation. Aprea Therapeutics is developing therapies to target mutations in genes in this pathway to treat certain cancers.  We spoke to Oren Gilad, president and CEO of Aprea Therapeutics about the DDR pathway, the use of so-called synthetic lethality to treat these cancers, and why Aprea’s approach may result in safer and more targeted therapies. Editor's note: We are have deleted out the original post because there were problems with the file and have reposted the interview here.

Delivering the Goods

Nearly 70 percent of patients with chronic conditions fail to take their medicines as prescribed. This can lead to poor outcomes and greater healthcare costs. Vivani Medical, which has developed implantable drug delivery technology, said it can provide steady drug release for up to six months with a single implant. This not only can address the problem of non-compliance, but can also reduce side effects associated with fluctuating drug release from conventional delivery methods. We spoke to Adam Mendelsohn, CEO of Vivani Medical, about its implantable drug delivery technology, how it works, and why its lead indication is a GLP-1 implant for weight loss and diabetes.