Uncategorized
STAT+: Capsida says it still doesn’t know what caused gene therapy death
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.
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.
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.
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.