The Bio Report

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.

The body has a natural cellular recycling machinery known as the ubiquitin proteasome system that breaks down unwanted proteins. Kymera Therapeutics has developed a drug discovery platform that exploits this natural biologic process to target disease-causing proteins that had been previously considered undruggable using small molecule therapies. We spoke to Nello Mainolfi, co-founder, president, and CEO of Kymera, about the company’s discovery platform, how it exploits a natural housecleaning mechanism within the body, and why this approach could enable the targeting of proteins that previously had been considered beyond the reach of small molecule therapies.

About 70 percent of daily deaths are caused by aging or age-related diseases. The newly formed Alliance for Longevity Initiatives, or A4LI, is an independent nonprofit advocating for greater investment in scientific research, new measures to recognize the value of extending healthy life expectancy, and steps to expedite the development gerotherapies and regenerative medicines. We spoke to Sonia Arrison, chair of the Alliance for Longevity Initiatives, about the need the new organization is seeking to address, its agenda, and how therapeutic advances may be able to alter our notions of longevity.

As concerns about the use of animals to test drugs, scientist have sought new ways to analyze the efficacy and toxicity of their products. Genoskin is seeking to enable better, faster, and safer drug development through the use of its proprietary ex vivo human skin platforms for preclinical drug testing. The company provides natural human skin obtained from patients that it maintains in a living and functional state. We spoke to Pascal Descargues, founder and CEO of Genoskin, about the company’s ex vivo human skin models, how they provide an alternative to animal experiments, and how they can accelerate the generation of reliable human data in drug development.

While cell therapies have been advancing rapidly, therapies that rely on taking a patient’s own cells, altering them, and reinfusing them back into a patient are costly. Part of the reason why is that the process for doing this is labor intensive. Cellino has developed a platform for producing personalized autologous cell therapies in an automated and scalable fashion. We spoke to Marinna Madrid, co-founder and vice president of product, for Cellino, about the company’s platform technology for cell-based therapies, how it works, and why it may help enable a new era of personalized regenerative therapies.

Integrins are a diverse family of proteins that play an essential role in many cellular biological processes. They also have been implicated a number of autoimmune, cardiovascular, and metabolic diseases, as well as fibrosis and cancer. While a number of biologics have come to market that target integrins, drug developers have been stymied in efforts to develop oral therapies that can target these proteins. Morphic Therapeutic is developing a new generation of oral integrin drugs it believes can transform the treatment model for a range of serious medical conditions. We spoke to Praveen Tipirneni, president and CEO of Morphic Therapeutic, about intgerins, the challenges of using small molecule therapies to target them, and why this can have a dramatic effect on how patients with a range of conditions get treated.

As the treatment of cancers has moved toward an increasing emphasis on the role the immune system can play in fighting tumors, a range of new ways to enlist and train the immune system have emerged. Candel Therapeutics is developing oncolytic viral immunotherapies, which it says combines anti-tumor activity while also stimulating the immune system. We spoke to Paul Peter Tak, president and CEO of Candel Therapeutics, oncolytic viral immunotherapies, how they work, and why they may be able to bring benefits to the treatment of a range of solid tumors.

The discovery and cell line development of biotherapeutics has been traditionally a distinct process. Absci is taking what it describes as a more wholistic approach by collapsing the process down and addressing the functionality and manufacturability of therapeutic candidates simultaneously. We spoke to Sean McClain, founder and CEO of AbSci, about how it is using AI and synthetic biology to reinvent the discovery process, how this is expanding the therapeutic potential of proteins, and how it translates into time and cost benefits.

While CAR-T cell therapies have been a promising new area of cancer treatments, they are costly to produce, have had limited success in treating solid tumors, and can carry sometimes serious side effects. Shoreline Biosciences is developing off-the-shelf natural killer and macrophage cellular immunotherapies derived from induced pluripotent stem cells for cancer, inflammatory, and genetic diseases. The company programs these cells to target and kill tumors, as well as repair tissue homeostasis. The company said its approach allows for the creation of a streamlined, affordable, and scalable manufacturing process that can deliver cell therapy treatments to patients in a faster and more cost-effective manner. We spoke to Kleanthis Xanthopoulos, co-founder and CEO of Shoreline Biosciences, about the company’s off-the-shelf cell therapies, its focus on the innate immune system, and how it’s leveraging its technology through recent partnerships with BeiGene and Kite Pharma.