The Bio Report

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.