Articles

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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

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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 FDA's draft guidance on prior knowledge points to a more connected path, where what we learn in one genome editing program can help carry the next one forward.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

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KIZ-Mutation Retinitis Pigmentosa: A Textbook Case for Gene Replacement Therapy

Retinitis pigmentosa gene therapy is not one-size-fits-all. Here is how to tell which genes are built for gene replacement, using KIZ, one cause of inherited retinitis pigmentosa, as a worked example.

Jun 3, 2026

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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

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

May 21, 2026

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How AI Can Cut the Cost of Personalized Gene Therapy

May 5, 2026

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Who Owns the Cure? The Case for Patient Control in Rare Disease Drug Development

Apr 21, 2026

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Gene Replacement vs. Gene Editing: Are they the same thing?

Apr 12, 2026

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N-of-1 Therapy Explained: What are the steps and timelines?

Apr 12, 2026

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Patient-Derived Cells: The Essential Foundation for Personalized Drug Development.

Apr 12, 2026

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The New York Times Wrote About Rare Disease Gene Therapy. Here Is What They Left Out

Apr 12, 2026

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Landscape Review: The State of Gene Therapy and ASO Development

Apr 12, 2026