The Bio Report

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.

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.

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.  

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.

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.

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.

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.

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.

One of the key obstacles to developing new drugs and diagnostics is the lack of access to longitudinal biological and clinical patient data. Culmination Bio, a spin-out of Intermountain Health, is building the largest patient data platform with more than 40 years of de-identified patient electronic health records and biospecimen data. We spoke to Lincoln Nadauld, president and CEO of Culmination Bio, about the data driving the company’s platform, how partners are using its data today, and how it has the potential to accelerate the development of new diagnostics and therapies.

NAMPT is a critical enzyme that regulates how cells use energy. It can also play an important role in the ability of cancer cells to survive, grow, and spread. While it’s been recognized as a potential target to treat certain cancers, developing NAMPT inhibitors have been challenging because they can disrupt energy metabolism in healthy cells. Remedy Plan Therapeutics believes it’s overcome the challenges of NAMPT inhibition by a unique mechanism of action that can avoid the problem of toxicity. We spoke to Greg Crimmins, founder and CEO of Remedy Plan Therapeutics, about the role of NAMPT in cancer, the challenges of developing NAMPT inhibitors without toxicity, and why he believes the company has solved the problem.

The emergence of CRISPR, a powerful tool for gene editing, promises to correct devastating genetic diseases, but it also raises concerns about how it will be used and who will make those decisions. Neal Baer, in his new book “The Promise and Peril of CRISPR,” brings together a series of essays by bioethicists, geneticists, and others who explore ethical and policy considerations raised by the technology. We spoke to Baer, co-director of the master’s degree program in Media, Medicine, and Health at Harvard Medical School; about whether there are unique questions raised by CRISPR as a dual-use technology, the book’s recasting of the case of Chinese scientist He Jiankui, and why he thinks there needs to be public debate now over the use of the technology to make heritable changes to the genome.

Speeding Protein Engineering with Whether it’s the development of new biotherapeutics, or replacing hydrocarbons from their role in industrial manufacturing, engineering proteins suited for a task is a time consuming and expensive process. Cradle Bio has developed generative AI technology to accelerate protein engineering by reducing the number of experiments needed to arrive at a product candidate and enable the optimization of multiple properties at once. We spoke to Elise de Reus, co-founder of Cradle, about the company's generative AI technology, how it works, and how it’s changing the process of protein engineering.

With the emergence of GLP-1 agonists to treat obesity, there has been growing interest in the use of peptide-based medicines. Protagonist Therapeutics has developed technology that can take these target specific and potent therapies and allow them to be delivered orally. The company has a collaboration with Johnson & Johnson for an oral peptide that blocks the IL-23 receptor that’s in development to treat psoriasis and ulcerative colitis. We spoke to Dinesh Patel, CEO of Protagonist, about the company’s platform technology, the benefits of orally delivered peptide therapies, and where he sees the biggest opportunities for this approach.

While there have been a parade of novel therapeutic modalities in recent years, many of them are constrained by delivery challenges. Vesigen Therapeutics is developing novel drug delivery technology known as ARMMs that can transport a wide range of therapeutic payloads directly into the cytoplasm of target cells. We spoke to Paulash Mohsen, CEO of Vesigen, about the company’s delivery technology, its advantages over viral vectors and liquid nanoparticles, and how it enables the targeting of previously undruggable targets.

The body has a natural mechanism for breaking down and clearing proteins. Arvinas is among a group of companies that’s seeking to harness this mechanism for therapeutic purposes. It has platform technology that enables the development of targeted protein degraders to breakdown disease-causing proteins. Among the benefits of this approach is that it offers a way to target so-called undruggable proteins that conventional drugs are not able to bind with in a strong enough way. We spoke to Randy Teel, chief business officer and interim chief financial officer of Arvinas, about protein degradation, its evolution as a therapeutic strategy, and the range of indications the company is pursuing.

The traditional approach to treating autoimmune disease has relied on ways to suppress the immune system. COUR Pharmaceuticals is developing first-in-class therapies that instead seek to reprogram the immune system to create antigen-specific tolerance. We spoke to COUR CEO John Puisis and COUR Vice President of Research Adam Elhofy, about how the company’s immune-modifying nanoparticles work, how the approach preserves the immune response, and its partnership with Takeda focused on celiac disease.

The next blockbuster drug may already be in production by bacteria within your body. Bacteria produce unique chemical compounds that are bioactive and may have great therapeutic potential, but this chemical space has largely not been mined. Empress Therapeutics, a Flagship Pioneering company, is searching for such molecules by exploring the genes of bacteria that live in the human body to search for the molecules they produce and identify compelling drug candidates. Since its founding in 2020, the company has already identified 15 novel candidates. We spoke to Sabrina Yang, chief innovation officer for Empress and a principal at Flagship Pioneering, about how exploring the bacterial metagenome in the human body can point the way to the next blockbuster drug, the role AI plays in its platform, and how this approach can accelerate the time and reduce the cost of drug development.

In most cases, the immune system detects and destroys cells that could become cancerous every day. For cancer to ravage the body, it needs to evade the immune system by shutting off its response. SIRPant Immunotherapeutics is seeking to address cancer by activating and modifying a patient’s own macrophages to awaken the immune system to cancer. The activated macrophages are able to recognize and directly attack cancer cells throughout the body and stimulate other parts of the immune system to sustain a prolonged attack. We spoke to Robert Towarnicki, CEO of SIRPant, about the company’s immunotherapies, the advantages they offer over other approaches, and why its platform can be applied to other diseases involving immune dysfunction beyond cancer.

As the founder of SynBioBeta, John Cumbers has long evangelized to investors, entrepreneurs, and forward-thinking industrialists about the emergence of the bioeconomy. Cumbers, however, wants to share his sense of wonder about molecular biology with a broader audience through his new venture, Biological Enlightenment Studios. His ambition is for the studio to become a Pixar for curious-minded youngsters. The studio’s first effort will be Lee's Lab, an animated series that follows the 12-year-old title protagonist through the jungles of Borneo as she seeks to understand the secrets of molecular biology and the splendors of biodiversity. We spoke to Cumbers about Biological Enlightenment Studios, his desire to spark the imagination of a new generation about the marvels of biology, and why bioliteracy matters for the growth and health of the bioeconomy.  

Electronic health records are a rich source of real-world data that can provide insights into health and wellness. Neal Meropol, head of research oncology at Flatiron Health, said they not only have value as a research tool for retrospective studies, but can be a treasure trove of information for prospective studies as well. We spoke to Meropol about how real-world evidence can inform better clinical trial designs and accelerate drug development, how Flatiron works with its clients, and a recent study he was involved in that suggest drug developers who want to increase the diversity of clinical trials participants should consider broadening their inclusion criteria.