The Bio Report

Advancing a Cell Therapy with the Potential to Cure HIV

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.

Harnessing More Efficient Organisms to Reshore BioManufacutring

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.

Using Digital Health Technology to Bring the Trial to the Patient

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.

Using the Body’s Housecleaning Mechanism to Target Undruggable Proteins

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.

Advocating for the Extension of Healthy Life

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.

Using Donated Living Skin as an Alternative to Animal Testing

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.

A Platform to Scale the Production of Personalized Cell Therapies

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.

Developing Small Molecule Therapies to Target Proteins Only Biologics Have Been Able to Hit

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.