The Nome library.

Article

When one vector isn’t enough: why dual-vector gene therapy matters

The first FDA-approved dual-AAV gene therapy solves a delivery problem that has limited the field for years. Here is why that matters for rare disease families.

Jun 15, 2026

Article

Experimental Therapy Testing: How to know if a therapy is safe and effective before it reaches a patient

For many families, a genetic diagnosis is supposed to be the finish line. Years of appointments. Specialist after specialist. A test that finally puts a name to it.

Jun 1, 2026

Article

ASGCT 2026 Recap: How Personalized Gene Therapies Are Becoming Real for Rare Disease Families

May 21, 2026

Article

FDA's new prior knowledge guidance: why rare disease gene therapy no longer has to start from zero

For many rare disease families, every new therapy feels like building from scratch. FDA's draft guidance on prior knowledge points to a more connected path, where what we learn in one genome editing program can help carry the next one forward.

Jun 9, 2026