The Bio Report

Fighting Disease by Modulating the Translation of Proteins

The development of mRNA therapies is allowing drug developer to pursue targets once considered undruggable. Anima Biotech has developed a platform that allows it to use small molecule that selectively control mRNA and can decrease or increase the translation of proteins. The approach has broad applicability as evidenced by the company’s pipeline, which includes experimental therapies for oncology, neurology, infectious disease, and fibrosis. We spoke to Yochi Slonim, CEO of Anima, about the company platform technology, how it can modulate the expression of proteins, and how it is leveraging its technology through partnerships with some of the world’s largest pharmaceutical companies.

Fixing Biopharma’s R&D Productivity by Taking a Cue from the Tech Industry

The biopharmaceutical industry has long struggled with R&D productivity. Long time industry strategic consultant Mike Rea, founder and CEO IDEA Pharma, thinks he’s hit on a possible solution. Taking a cue from the tech industry, Rea in May announced the launch of Protodigm, which he describes as a contract skunkworks company. The approach is intended to allow a multi-disciplinary team work autonomously to take its clients early-stage assets and explore multiple development options at once with the intent of de-risking innovation while saving time and money. We spoke to Rea about the industry’s challenges with R&D productivity, the benefits Protodigm’s skunkworks approach could provide, and how it will work with industry.

An Affordable and Widely Available Drug Offers Global Hope against COVID

While cities across the country have lifted COVID-19 restrictions and public gatherings have returned, the lack of effective treatments for people who develop severe reactions to the virus continues to pose a public health problem. The situation is acute in less developed nations where vaccination levels are low, and hospitals continue to be overrun with patients. Researchers at Mount Sinai earlier this year reported in the journal Cell that a widely available and inexpensive drug targeting inflammatory genes has reduced morbidity and mortality in mice infected with the virus that causes COVID-19. The drug, topotecan, is now in clinical trials in India as a potential treatment for patients who suffer from a hyper-inflammatory response to the virus. We spoke to Ivan Marazzi, senior author of the study and an associate professor of microbiology at the Icahn School of Medicine at Mount Sinai, about topotecan, what makes it compelling as a potential treatment for COVID-19, and why it may be well suited for treating people with the condition across the globe.

A Gene Therapy to Program the Body to Produce a Treatment for Osteoarthritis

Osteoarthritis is a painful, progressive disease of the joints for which there is no cure. The condition affects more than 32.5 million adults in the United State and the incidence is growing as a result of aging, obesity, and sports injuries. Flexion Therapeutics is developing an experimental gene therapy to treat the condition. Rather than correcting an underlying genetic mutation, the experimental therapy delivered into the joint codes for the production of the anti-inflammatory protein interleukin-1 receptor antagonist. It is expected to deliver as-needed anti-inflammatory activity to joint tissues over the long-term. We spoke to Mike Clayman, co-founder and CEO of Flexion Therapeutics, about osteoarthritis, the problem with existing therapeutic approaches, and the company’s efforts to develop a gene therapy to get the body to produce an anti-inflammatory protein as needed.

A Test for Data-Driven Drug Development

Sumitomo Dainippon Pharma’s $3 billion strategic transaction with Roivant Sciences that gave rise to Sumitovant, represents an effort by the Japanese drug company to make the drug development process faster and more efficient. A critical piece of this effort is steps the company has taken to harness information technology to capitalize on existing knowledge to inform decision making. We spoke to Bill McMahon, chief algorithmic analytics officer of Sumitovant, about the approach Sumitovant is taking, the way uses information technology to improve the drug development process, and how the subsidiary may be influencing the way its parent company operates.

Bridging the Gulf Between the Promise and Reality of Precision Health

The ability to identify diseases at their earliest stages through the detection of minute levels of biomarkers offers the potential to make interventions before the outward physical manifestations of illnesses can bloom and long-term damage can occur. Quanterix is developing a platform of ultra-sensitive biomarker tests to give researchers greater insight into the transition from health to disease and the ability to detect diseases at an earlier point in their development. We spoke to Kevin Hrusovsky, chairman, president, and CEO of Quanterix, about the company’s ability to detect minute amounts of biomarkers, the implications this has for drug development and diagnostics, and how this can help drive a future of precision health.

Using Synthetic Biology to Craft One-Time, Programmable mRNA Therapeutics

The marriage of synthetic biology to mRNA is creating the potential for a range of programmable therapeutics that can provide new ways of treating deadly and chronic diseases. Strand Therapeutics is creating a platform for developing these long-acting mRNA drugs that it says can be precise, multi-functional, and deliver potentially curative treatments with a single dose. We spoke to Jake Becraft, co-founder and CEO of Strand, about the company’s programmable mRNA therapeutic platform, how it works, and the indications it is pursuing.

Using a Digital Cognitive Behavior Therapy to Treat Diabetes and Heart Disease

Cardiometabolic conditions, such as type 2 diabetes and heart disease, have long been treated with pharmacologic interventions. Better Therapeutics believes cognitive behavioral therapy delivered through its prescription digital therapeutics platform, will provide better outcomes at a lower cost. We spoke to David Perry, co-founder and executive chairman of Better Therapeutics, about the company’s prescription digital therapeutics platform, the case for personalized cognitive behavior therapy delivered this way, and what it will take to get doctor to prescribe it and payers to reimburse for it.