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STAT·May 12·2 min read
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STAT+: Capsida says it still doesn’t know what caused gene therapy death

Some in the gene therapy field lament lost time while Capsida kept silent about the brain receptor it was targeting when things went wrong.

May 12, 2026·read at STAT ↗

STAT PlusBiotech Capsida says it still doesn’t know what caused gene therapy death Trial tested the first of a new wave of brain-targeted treatments Manage alerts for this article Email this article Share this article By Jason MastMay 12, 2026 General Assignment Reporter Jason Mast[email protected]Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05. Capsida Biotherapeutics said Tuesday that it still had no answers in its investigation into the death of a child in a gene therapy trial last September.

Its scientists’ efforts, it said, have been stymied because the hospital where the study was conducted has declined to share tissue samples from an autopsy. Advertisement The therapy, known as CAP-002, was the first of a wave of new gene therapies designed to deliver genes deep into the brain. Scientists around the world engineered viruses that could slide through the blood-brain barrier that walls off our most vital organ from the rest of the body.

Companies spun up promising treatments for devastating rare genetic diseases and common conditions like Alzheimer’s and Parkinson’s. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%!

2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, gene editing, Gene therapy, rare diseases, STAT+ Submit a correction requestReprints Jason Mast General Assignment Reporter Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05.

Newsletter Your morning rundown of the science, politics, and money driving biotech today Recommended The Readout May 11, 2026 STAT Plus: Astellas treatment offers new hope to a devastated rare disease community Health May 11, 2026 STAT Plus: Five years after disaster, a rare disease community gets new chance at treatment Advertisement Exclusive May 11, 2026 STAT Plus: Medicare is spending far less than expected on new Alzheimer’s drugs Biotech May 8, 2026 The biotechs wear Prada The Readout May 8, 2026 STAT Plus: The biotech news you need to read today Subscriber Picks

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Reporting by STAT.

read at STAT ↗
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companies & drugs in this story

companies
Ultragenyx Pharmaceutical Inc.4ASTELLAS
drugs
Rare Disease2
topics
Gene Therapy (AAV)5Rare Disease4CRISPR/Cas9Hematology

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