The Bio Report

Swimming in the New Oil

One of the key obstacles to developing new drugs and diagnostics is the lack of access to longitudinal biological and clinical patient data. Culmination Bio, a spin-out of Intermountain Health, is building the largest patient data platform with more than 40 years of de-identified patient electronic health records and biospecimen data. We spoke to Lincoln Nadauld, president and CEO of Culmination Bio, about the data driving the company’s platform, how partners are using its data today, and how it has the potential to accelerate the development of new diagnostics and therapies.

Does This Drug Represent “A Paradigm Shift for Small Molecule Drug Development?”

NAMPT is a critical enzyme that regulates how cells use energy. It can also play an important role in the ability of cancer cells to survive, grow, and spread. While it’s been recognized as a potential target to treat certain cancers, developing NAMPT inhibitors have been challenging because they can disrupt energy metabolism in healthy cells. Remedy Plan Therapeutics believes it’s overcome the challenges of NAMPT inhibition by a unique mechanism of action that can avoid the problem of toxicity. We spoke to Greg Crimmins, founder and CEO of Remedy Plan Therapeutics, about the role of NAMPT in cancer, the challenges of developing NAMPT inhibitors without toxicity, and why he believes the company has solved the problem.

Exploring the Promise and Peril of CRISPR

The emergence of CRISPR, a powerful tool for gene editing, promises to correct devastating genetic diseases, but it also raises concerns about how it will be used and who will make those decisions. Neal Baer, in his new book “The Promise and Peril of CRISPR,” brings together a series of essays by bioethicists, geneticists, and others who explore ethical and policy considerations raised by the technology. We spoke to Baer, co-director of the master’s degree program in Media, Medicine, and Health at Harvard Medical School; about whether there are unique questions raised by CRISPR as a dual-use technology, the book’s recasting of the case of Chinese scientist He Jiankui, and why he thinks there needs to be public debate now over the use of the technology to make heritable changes to the genome.

Transforming Protein Engineering with Generative AI

Speeding Protein Engineering with Whether it’s the development of new biotherapeutics, or replacing hydrocarbons from their role in industrial manufacturing, engineering proteins suited for a task is a time consuming and expensive process. Cradle Bio has developed generative AI technology to accelerate protein engineering by reducing the number of experiments needed to arrive at a product candidate and enable the optimization of multiple properties at once. We spoke to Elise de Reus, co-founder of Cradle, about the company's generative AI technology, how it works, and how it’s changing the process of protein engineering.

Delivering Peptide Therapies Orally

With the emergence of GLP-1 agonists to treat obesity, there has been growing interest in the use of peptide-based medicines. Protagonist Therapeutics has developed technology that can take these target specific and potent therapies and allow them to be delivered orally. The company has a collaboration with Johnson & Johnson for an oral peptide that blocks the IL-23 receptor that’s in development to treat psoriasis and ulcerative colitis. We spoke to Dinesh Patel, CEO of Protagonist, about the company’s platform technology, the benefits of orally delivered peptide therapies, and where he sees the biggest opportunities for this approach.

Creating A Drug Delivery ARMMs Race

While there have been a parade of novel therapeutic modalities in recent years, many of them are constrained by delivery challenges. Vesigen Therapeutics is developing novel drug delivery technology known as ARMMs that can transport a wide range of therapeutic payloads directly into the cytoplasm of target cells. We spoke to Paulash Mohsen, CEO of Vesigen, about the company’s delivery technology, its advantages over viral vectors and liquid nanoparticles, and how it enables the targeting of previously undruggable targets.

A Seek and Destroy Approach to Disease-Causing Proteins

The body has a natural mechanism for breaking down and clearing proteins. Arvinas is among a group of companies that’s seeking to harness this mechanism for therapeutic purposes. It has platform technology that enables the development of targeted protein degraders to breakdown disease-causing proteins. Among the benefits of this approach is that it offers a way to target so-called undruggable proteins that conventional drugs are not able to bind with in a strong enough way. We spoke to Randy Teel, chief business officer and interim chief financial officer of Arvinas, about protein degradation, its evolution as a therapeutic strategy, and the range of indications the company is pursuing.

Teaching Tolerance to Address Autoimmune Diseases

The traditional approach to treating autoimmune disease has relied on ways to suppress the immune system. COUR Pharmaceuticals is developing first-in-class therapies that instead seek to reprogram the immune system to create antigen-specific tolerance. We spoke to COUR CEO John Puisis and COUR Vice President of Research Adam Elhofy, about how the company’s immune-modifying nanoparticles work, how the approach preserves the immune response, and its partnership with Takeda focused on celiac disease.