The Bio Report

About 98 percent of the the human genome consists of non-protein coding regions known as the “dark genome.” Once derided as “junk DNA,” these regions are increasingly understood to play a critical role in the regulation of the genome and offer a novel means of targeting diseases. Haya Therapeutics is exploring long non-coding RNAs as potential therapies to treat a range of diseases. We spoke to Sa...

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 technol...

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...

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...

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 platfo...

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...

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...

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 i...

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 e...

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 B...

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 ch...

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 tech...

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 candidat...

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 developm...

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,...

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 prot...

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 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 f...

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 d...

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 youn...

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 clini...

Earlier this year, Profluent announced it had edited the human genome with OpenCRISPR-1, the world’s first AI-created and open-source gene editor. Though the open-source aspect helped garner attention for Profluent, it also served as a demonstration of the company’s generative AI platform to create novel CRISPR gene editors. We spoke to Hilary Eaton, chief business officer for Profluent, about how...

About 500,000 people suffer from a spinal cord injury each year. Treatments can involve surgical procedures to stabilize the spine and physical rehabilitation, which can have limited benefits. There are currently no FDA-approved therapies that can promote repair and improve function following a spinal cord injury. NervGen Pharma is seeking to change that by developing therapies that allow the nerv...

One of the challenges of treating neurodegenerative diseases is delivering therapies across the blood-brain barrier. Aliada Therapeutics is developing therapies that use its platform technology that enables the delivery of large molecules, like antibodies, across that barrier. Its lead program is in development to treat Alzheimer’s disease. We spoke to John Dunlop, chief scientific officer of Alia...

SalubrisBio, rather than shy away from complexity, embraces it. The company’s lead experimental therapy is an antibody fusion protein in development for both heart failure and the rare, neurodevelopmental condition amyotrophic lateral sclerosis. We spoke to Sam Murphy, CEO of SalubrisBio, about the challenges of pursuing complex diseases with complex therapies, its pipeline, and how its China-base...