The child dies after receiving new experimental gene therapy
The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after the dosage, said society sponsoring this trial on Wednesday.
The company, Capsida Biotherapeutics, is part of a group of companies and laboratories in engineering of new viruses potentially capable of securely transporting genes deeply in the brain. His first effort was designed to treat STXBP1 encephalopathy, a condition that can cause convulsions, development delays and other symptoms.
The STXBP1 Foundation declared in a note to the members of the community that the company and the investigators were still working to understand what had happened.
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