Tafamidis: A revolutionary drug for treating neurodegenerative and heart diseases

Hear from 3 of our newest PhD recipients as they reflect on their experience as students in Scripps Research’s Skaggs Graduate School. The students are among the 49 who were awarded doctoral degrees at the institute’s May 15th Commencement.

Current therapies for inflammatory bowel disease (IBD) primarily focus on suppressing inflammation. But what if we could repair one of the underlying drivers of disease itself? Researchers at Calibr-Skaggs are developing CLF065, a potential first-in-class regenerative medicine designed to restore epithelial barrier function—the protective intestinal lining that regulates what passes from the gut into the body. CLF065 is designed with a dual mechanism of action: supporting barrier integrity and stimulating regeneration of the gut lining. A Phase 2 clinical trial has recently launched in chronic pouchitis, a painful inflammatory condition that can develop in some people with IBD following surgery. In addition, an Investigational New Drug (IND) application has been submitted to support a Phase 2 trial in Crohn's disease. Learn how regenerative medicine could help shift the treatment paradigm from managing symptoms to repairing the tissue damage at the root of disease.

During the first weeks of pregnancy, the developing placenta in a mother’s womb undergoes a dramatic change. Individual cells merge, forming a continuous barrier that will spend the next nine months ferrying oxygen and nutrients to the growing fetus while keeping the mother's immune system at bay. When this structure fails to form properly, pregnancies are at higher risk of complications, including preeclampsia and restricted fetal growth. Now, scientists at Scripps Research have identified a key player in the placenta’s transformation—a molecule called galectin-3 that binds to sugar molecules on specific cell surface proteins, likely holding placental cells tightly together to fuse.

Some of the most devastating diseases are driven by proteins that cannot be targeted with conventional drugs. Scientists are now discovering ways to control disease by recruiting the cell’s own machinery to tackle this challenge. In this Front Row lecture, Associate Professor Michael Erb took the audience inside the cell to explain how his team is developing “proximity” drugs. Erb’s innovation has implications to change how scientists and cells join forces to fight against breast, prostate and blood cancers. Learn how proximity drugs can eliminate proteins, rewire essential cellular pathways, and enable researchers to target previously “undruggable” proteins.