Clive Svendsen, Meritxell Huch, Ameen Salahudeen, and Maksim Plikus will discuss the latest advances in using patient-derived stem cells to create more accurate disease models.
Damaged lysosomes are repaired by a lipid-based signaling pathway dubbed PITT that could be targeted to treat neurodegenerative disease, its discoverers say.
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
Critics point out that cell therapy has yet to top existing treatments. Biotech companies are setting out to change that—and prove that the technology can revolutionize medicine.
This year’s crop of winning products features many with a clinical focus and others that represent significant advances in sequencing, single-cell analysis, and more.
An open mind and collaborative spirit have taken Hans Clevers on a journey from medicine to developmental biology, gastroenterology, cancer, and stem cells.