The Bio Report

Existing anticoagulants can prevent life-threatening blood clots for people with cardiovascular and metabolic diseases. The problem is that because of their mechanism of action, they also work to suppress the body’s ability to stop bleeding as it should. As a result, these therapies can be under utilized by patients who need them. Anthos Therapeutics is developing an experimental monoclonal antibody that can suppress coagulation without disrupting hemostasis, the biological process of stopping bleeding. We spoke to John Glasspool, CEO of Anthos, about the problems of existing anticoagulants, the novel target its monoclonal antibody acts on, and how it is able to uncouple the pathways for thrombosis and hemostasis.

Though the search for eternal youth has long been the fodder for myths and legends, science has been pushing us closer toward extending healthy years of life and has set some people off on efforts to defy death altogether. Peter Ward, in his new book The Price of Immortality: The Race to Live Forever, explores a subculture of immortality seekers who have turned to cryonics, as well as efforts to merge man with technology as a way to escape death. The growing understanding of the biology of aging and advances in regenerative medicine, though, are creating the potential to alter notions of human lifespans. We spoke to Ward about his book, distinguishing science fact from science fiction, and the growing understanding of the biology of aging that offers the potential for extending healthy years of life.

About 15 years ago, Harvard Business School professor Gary Pisano took at look at how small entrepreneurial biotechs fared against large and mature pharmaceutical companies and found the two sectors were about the same when it came to R&D productivity. Now, a new book From Breakthrough to Blockbuster: The Business of Biotechnology finds that the biotechnology industry is far more effective at bringing innovative therapies to market than Big Pharma and offers a prescription for large drug companies to decentralize decision-making to improve their ability to innovate. We spoke to venture investor and former biotech executive Don Drakeman, co-author of the book, about its findings, how decentralized decision-making can produce greater innovation, and lessons from COVID-19.

Poseida Therapeutics is leveraging its set of platform technologies to develop a range of next-generation cell and gene therapies. By using these technologies, alone or in combination, the company said it’s able to overcome limitations of the current generation of cell and gene therapies. The company’s most advanced candidates in its pipeline include a set of allogenic CAR T therapies. We spoke to Mark Gergen, CEO of Poseida, about the company’s efforts to develop off-the-shelf CAR T therapies, its platform technologies, and why it believes its CAR T cells will provide advantages over competitors’ therapies.

Though structure-based drug discovery has been long been used, technologic advances have given this approach greater power. ShouTi believes its next-generation, computational, structure-based drug discovery platform will allow it to develop small molecule drugs that are superior in safety and efficacy to biologic and peptide therapies its seeking to replace. We spoke to Raymond Stevens, CEO of ShouTi, about the company’s structure-based drug discovery platform, how it works, and why he believes it will be able to produce small molecule drugs that will be superior to biologics.

Virus-like drug conjugates, or VDCs, are a new class of cancer therapies Aura Biosciences is developing to target a broad range of solid tumors. These therapies have a dual mechanism of action. They deliver a toxic payload to cancer cells, but also activate a secondary immune mediated response to kill cancer cells. We spoke to Elisabet de los Pinos, CEO of Aura Biosciences, about the company’s VDCs, how they work, and its pipeline in development.

While the emergence of CAR-T therapies have been promising, these autologous cellular therapies are costly to produce since they require taking T cells from a patient being treated, altering them, and then infusing them back into the patient. Cellectis is among a growing list of companies pursuing off-the-shelf CAR-T therapies. The company’s U-CARTs, or universal chimeric antigen receptor T cells, are allogenic products that can be standardized and carry both time and cost advantages. We spoke to André Choulika, CEO of Cellectis, about the company’s off-the-shelf CAR-T therapies, the platform technology behind them, and its programs in development.

Though it’s been nearly 20 years since the Human Genome Project provided a blueprint for human biology, it still left much work to be done to understand health and disease at a molecular level. The Sweden-based Human Protein Atlas, which is seeking to map human proteins in cells, tissues, and organs, recently published significant updates to the open-access resource. We spoke to Mathias Uhlén, director of the Human Protein Atlas, about how the atlas is changing the diagnosis and treatment of disease, what’s known about the human proteome to date, and how this understanding will be essential to brining about an era of precision medicine.

In 2020, CSL Behring entered into a global license and commercialization agreement with UniQure for the company’s experimental hemophilia B gene therapy. The expected, one-and-done treatment carries the potential to free people with the genetic bleeding disease from reliance on regular infusions of clotting factor IX for which they are deficient. We spoke to Steve Pascoe, senior vice president and head of therapeutic areas and development strategy for R&D at CSL about hemophilia B, how the gene therapy fits into CSL’s broader therapeutic offerings, and the encouraging results from the recent pivotal study.

The promise of data-driven medicine is that it can accelerate the diagnosis of disease, provide patients with the most effective treatments for their particular conditions, and improve drug development. Sophia Genetics is drawing on extensive information from its global, data-sharing network to make data-driven medicine a reality. We spoke to Emily Paul, product director of platform for Sophia Genetics, about Sophia’s vision for data-driven medicine, the challenges of managing vast amounts of data and turning it into actionable information, and how its changing patient care today.

The growing convergence of information technology and biotechnology are creating a compelling new group of companies that live in both these worlds at once. Lux Capital, which has long invested in both sectors, has a growing portfolio of these emerging TechBio companies. We spoke to Adam Goulburn, partner at Lux Capital, about his investment process, how he tempers the promise of technology with management realities and market timelines, and the changing landscape for venture investing.

Cell and gene therapies are among the most promising approaches to treating diseases because they carry the potential to cure chronic and progressive conditions. The problem is that the high cost of producing these therapies, which often need to be tailored to individual patients, limits access to them, particularly in low- and middle-income countries. Caring Cross is seeking to change that through its nonprofit model that focuses on enabling hospitals and health systems to manufacture advanced therapies locally and deliver them in a cost-effective manner. We spoke to Boro Dropulić, co-founder of Caring Cross, about how the nonprofit is seeking to lower the cost of these therapies, how it operates, and why its initially focused on HIV and sickle cell disease.

KeifeRx is developing a pipeline of orally delivered tyrosine kinase inhibitors to treat neurodegenerative diseases. It has a portfolio of these drugs that it has optimized to penetrate the brain, clear damaged cells, and treat these conditions through the bulk disposal of disease-causing toxic proteins. It believes its approach offers the potential to significantly improve on current treatments, which it calls primarily palliative because they fail to adequately eliminate the toxic proteins at the root of these deadly diseases. We spoke to KeifeRx CEO Chris Hoyt and co-founder and head of the company’s scientific advisory board Charbel Moussa, about how it has repurposed and optimized a cancer therapy to treat neurodegenerative diseases, and why it may have broad applications across these conditions.

When AzurRX BioPharma merged with First Wave Bio last year it renamed itself First Wave BioPharma. The company is developing targeted, non-systemic therapies for gastrointestinal diseases with its lead candidate niclosamide, an approved therapy to treat tapeworms that it believes has anti-viral and anti-inflammatory properties. The company is developing niclosamide in six GI-indications including COVID-19 related GI disease, Immune Checkpoint Inhibitor-associated colitis and diarrhea in advanced oncology patients, ulcerative proctitis/ proctosigmoiditis, ulcerative colitis, and Crohn’s disease. We spoke to James Sapirstein, CEO of First Wave BioPharma, about the merger, niclosamide, and its potential to treat a range of gastrointestinal disorders.

HCW Biologics is developing immunotherapies to target chronic inflammation associated with aging. The company’s therapeutics are designed to disrupt the link between chronic, low-grade inflammation and age-related disorders. The company believes this type of inflammation is a significant contributing factor to cancer and several chronic conditions, including cardiovascular disease, diabetes, neurodegenerative disease, and autoimmune disease. We spoke to Hing Wong, CEO of HCW Biologics, about the link between chronic inflammation and diseases of aging, the company’s immunotherapies, and its platform for developing therapies that target this unwanted inflammation to treat cancer and other diseases.

As 2021 fades into the history books, it will be remembered as one with exuberant IPOs, punishing aftermarkets, and a lingering pandemic that once again is turning the annual JPMorgan Healthcare Conference into a virtual event. We continue our annual tradition of sitting down with STAT Senior Writer Adam Feuerstein to discuss the year that was in biotech, the best and worst CEOs of 2021, and what’s ahead in the new year.

The p53 protein is known as the “guardian of the genome.” It plays an essential role in suppressing tumors. Rain Therapeutics is targeting a regulator of p53 that is overexpressed in certain cancer and can inactivate it, allowing certain cancers to grow and progress. We spoke to Avanish Vellanki, chairman and CEO of Rain Therapeutics, about the company’s experimental precision therapy milademetan, how it works, and its potential to treat a range of cancers.

Though HIV has fallen out of the headlines, the virus continues to represent a significant public health threat. American Gene Technologies is developing an experimental cell therapy that it says is potentially curative for HIV. We spoke to Jeff Galvin, CEO and founder of American Gene Technologies, about the state of HIV, the company’s experimental cell therapy for HIV, and why the one-time treatment has the potential to free patients from chronic use of antiviral therapies.

The COVID pandemic has called attention to the United States’ reliance on a supply chain that makes access to critical medicines dependent on the ability to make them overseas and ship them in a timely manner. At the same time, harnessing new ways of making biologics, is making it possible to gain significant savings over traditional manufacturing approaches. rBIO is betting it will be able to cost-effectively produce biologics in the United States and its starting with insulin to prove its point. We spoke to Cameron Owen, co-founder of rBIO, about the how the company is engineering different organism to increase the efficiency of biomanufacturing, why it is starting with insulin, and why reshoring biomanufacturing should be viewed as a critical issue for the United States.

The use of smartphones, low-cost sensors, and ubiquitous connectivity is changing the way researchers think about recruiting, monitoring, and interacting with participants in biomedical research. The use of evolving technology is not just eliminating geographic barriers to participation, but also enabling the collection of new types of data. The Scripps Research Digital Trials Center is pioneering the use of digital health technologies to re-engineer the way studies are conducted. We spoke to Edward Ramos, director of digital clinical trials for Scripps Research Digital Trials Center, about how digital health technology is transforming biomedical research, how it is changing what is possible, and some of the ongoing research projects the center is conducting.