The Bio Report

Earlier this year, Profluent announced it had edited the human genome with OpenCRISPR-1, the world’s first AI-created and open-source gene editor. Though the open-source aspect helped garner attention for Profluent, it also served as a demonstration of the company’s generative AI platform to create novel CRISPR gene editors. We spoke to Hilary Eaton, chief business officer for Profluent, about how the company’s generative AI engine works, its business model, and why its platform has the potential to create other protein therapeutics of varying modalities.

About 500,000 people suffer from a spinal cord injury each year. Treatments can involve surgical procedures to stabilize the spine and physical rehabilitation, which can have limited benefits. There are currently no FDA-approved therapies that can promote repair and improve function following a spinal cord injury. NervGen Pharma is seeking to change that by developing therapies that allow the nervous system to repair itself. We spoke to Mike Kelly, CEO of NervGen, about the potential for using therapies designed to allow the nervous system to repair itself, how the company’s lead experimental candidate for spinal cord injury works, and why the same approach holds promise in treating a range of neurodegenerative diseases.

One of the challenges of treating neurodegenerative diseases is delivering therapies across the blood-brain barrier. Aliada Therapeutics is developing therapies that use its platform technology that enables the delivery of large molecules, like antibodies, across that barrier. Its lead program is in development to treat Alzheimer’s disease. We spoke to John Dunlop, chief scientific officer of Aliada, about its platform technology, its origins at Johnson & Johnson’s Janssen, and the company’s partnering strategy for leveraging the technology.

SalubrisBio, rather than shy away from complexity, embraces it. The company’s lead experimental therapy is an antibody fusion protein in development for both heart failure and the rare, neurodevelopmental condition amyotrophic lateral sclerosis. We spoke to Sam Murphy, CEO of SalubrisBio, about the challenges of pursuing complex diseases with complex therapies, its pipeline, and how its China-based parent has provided it financial freedom from the vagaries of the capital markets.

One of the challenges facing genetic medicines is delivering payloads across the human cell membrane and inside cells both effectively and with high tolerability. Aegis Life is capitalizing on its parent Entos Pharmaceuticals’ nucleic acid delivery platform technology to address infectious diseases. It recently secured investment from the Bill & Melinda Gates Foundation to help in the fight against malaria, HIV, and other conditions. We spoke to John Lewis, founder and CEO of Aegis Life, about the need it is addressing, the company’s platform technology, and how it is leveraging that to address the barriers to the delivery of vaccines and antibody therapies in developing economies.

The idea of developing cancer vaccines that harness dendritic cells is not new, but Diakonos Oncology thinks it’s been able to solve the lack of efficacy that has plagued this approach. The company combines its vaccines with RNA and proteins from a patient’s tumor to trigger a robust response by tricking the immune system to recognize cancer cells as being virally infected. We spoke to Jay Hartenbach, chief operating officer of Diakonos, about its dendritic cell cancer vaccine technology, how it works, and why its lead indication is an aggressive form of brain cancer.

Kinase inhibitors have given rise to an era of precision medicine for the treatment of cancer, but the ability of cancer cells to mutate enables tumors to develop resistance to existing therapies. Nuvalent is developing therapies that both target the original tumor as well tumors with emergent resistance. We spoke to James Porter, CEO of Nuvalent, about how it designs drugs that address the challenges of both kinase resistance and selectivity, its pipeline in development, and why it’s critical that its experimental therapy for non-small cell lung cancer can penetrate the brain.

Each year, an estimated 400,000 people in the United States will be diagnosed with Lyme disease, a billion-dollar healthcare problem caused by a bacterial infection that is transmitted through the bite of a tick. The condition can cause joint pain and fatigue, but is treatable with antibiotics. Left untreated, though, it can progress and cause more serious symptoms including facial palsy, an irregular heartbeat, and nerve pain. Tarsus Pharmaceuticals is developing a human formulation of the antiparasitic lotilaner, to prevent Lyme disease. We spoke to Bobby Azamian, CEO of Tarsus, about Lyme disease, the case for the company’s experimental prophylactic drug TP-05, and why you may already have been using a form of the drug to protect a four-legged member of your household.

In February, the U.S. Food and Drug Administration approved the first tumor-infiltrating lymphocyte, or TIL therapy, for solid tumors. The approval was hailed as a milestone that points the path forward for a new class of cell therapies for solid tumors. We spoke to Jason Bock, CEO of CMTC, the joint venture between Resilience and MD Anderson Cancer Center, about the emerging area of TIL therapies, the significance of the first approved therapy in the class, and how they may address existing limitations of CAR-T therapies.  

Lipids have long been used as drug delivery vehicles, but HighField Biopharmaceutics is repurposing lipids as drugs to fight cancer. The company’s technology platform is seeking to change the immunotherapy landscape with new treatments that are better targeted, more potent, and less toxic. We spoke to Don Wyatt, chief business officer for HighField Biopharmaceuticals, about the tumor microenvironment, HighField’s platform technology, and how it is developing lipid-based medicines to overcome challenges of existing immunotherapies.