A place to learn about building a new type of biotech that aims to better serve patients.

White Paper · Sep 2026 · PDF

The Coming Wave of Allelic Series

An allelic series is when one gene shows up in people at several different strengths,It is the most convincing evidence human genetics can offer that a drug target is real. Our new white paper argues that the examples were there all along, waiting on data that has only recently arrived.

White Paper

How Do You Measure a Disease You Can't See?

Approved treatments are changing the course of ATTR-CM, a once-untreatable heart disease. But the thing that matters most is invisible, and a blood measurement of TTR can point two ways at once. So we built a way to quantify what really matters.

Video · Oct 2026

Every Minute Counts: The Story of Noa

When 10-month-old Noa Greenwood was diagnosed with a rare genetic disease, her parents, Lee and Lori were confronted with an idea of a future they'd never prepared for.

Opinion · Sep 2026

Opening a Door in D.C., Closing the Gap for Patients

The door is open. We invite advocates, people living with rare conditions, regulators, policymakers, researchers, and others working across the rare disease community to engage with us in this space, challenge us, and help us move with greater urgency.

Opinion · Sep 2026

The Genetic Testing Gap

The symptoms can look very different, but for many patients they trace back to the same overlooked cause: a rare genetic disease that hasn’t yet been named.

Opinion · Jul 2026

How a mother’s intuition helped uncover her son’s rare disease

Our patient advocacy team has gotten to know Arielle & her son Sebastian's challenges of living with ADH1 quite well. We were excited she was able to share her experiences with a broader audience on CNBC Cures.

Opinion & Video · May 2026

Scaling Innovation for Rare Diseases

Our CEO, Neil Kumar joined a panel moderated by CNBC Cure's Becky Quick to discuss how rewriting the rules will facilitate more rapid, cost-efficient, and scalable progress for the global rare disease community.

Podcast · May 2026

Neil Kumar on the Off-Target Effects podcast

Neil reflects on what it takes to serve patients well, build enduring capabilities, and create a sustainable biotech business in areas where others have historically been less focused.

Opinion · 2026

What makes a drug launch successful?

Having been fortunate to bring multiple medicines to market across several therapeutic areas, I have realized that the success of a launch is rarely attributed to one factor.

White Paper · 2025 · PDF

Unlocking the feasibility of rare disease drug development

Millions of people are affected by rare diseases worldwide, yet fewer than 10% have approved treatments. This is largely because the high cost of drug development makes developing medicines infeasible for diseases with small patient populations.

Opinion · 2025

Congress must renew a popular, bipartisan FDA program benefitting sick kids

Thousands of children’s lives have been saved by a cost-free government initiative that expired last December. Congress needs to renew this program quickly because too many babies are dying needlessly.