For decades, families affected by rare diseases have heard the same heartbreaking message: ‘There is no approved treatment yet.’ In 2026, that story is finally changing. From the first-ever approval for Menkes disease to a landmark FDA policy shift for gene therapies, the rare disease landscape is transforming at a pace never seen before. Here’s what you need to know.

A Practical lens for Board Members - Will this PI Founder still be CEO by Series B? Most European life sciences funds back companies where the founding CEO is a first‑time scientific leader: a PI out of a top lab, an ex‑pharma director, sometimes a computational founder with limited management experience. Very few investment memos say, “And we expect to replace them before Series B.” Yet that’s exactly what happens in a lot of cases—not always for the wrong reasons, but often

Your first five hires will decide whether your “platform biotech” ever becomes more than a pitch deck. In the first 18 months you’re not building an org chart, you’re building an engine: one person who truly owns the platform, one who runs everything that isn’t science, one who turns data into a scalable system, an early regulatory voice to stop you tripping over yourself later, and a business partner who keeps capital and deals flowing.