Dive Brief:<\/p>\n
Capsida Biotherapeutics has suspended a recently begun clinical trial of an experimental gene therapy after the first participant in the study died following treatment. Dive Insight:<\/p>\n Capsida shared few specifics Wednesday, noting that it is working to understand the \u201croot cause\u201d of the patient\u2019s death. It\u2019s unclear whether Capsida\u2019s treatment was directly to blame.<\/p>\n The news, however, follows patient deaths over the past year in clinical trials of gene therapies for two types of muscular dystrophy<\/a> and a rare neurological disorder<\/a>, and will therefore likely draw significant scrutiny.<\/p>\n Capsida received a green light<\/a> from the FDA to begin its clinical trial just four months ago, in May. Its gene therapy, which uses a specially engineered virus to reach the brain, is designed to treat developmental and epileptic encephalopathy related to mutations in the syntaxin-binding protein 1, or STXBP1, gene.<\/p>\n The company needed to custom design its viral vector, as the types of the naturally occurring viruses gene therapy developers typically co-opt for their therapies\u2019 delivery don\u2019t adequately saturate brain neurons.<\/p>\n Capsida aimed to enroll around 12 children with STXBP1-related disorders into its trial<\/a>, which had opened recruitment at Weill Cornell Medicine in New York and the Children\u2019s Hospital of Philadelphia in Pennsylvania.<\/p>\n People with STXBP1-related disorders<\/a> usually experience seizures beginning in their first year of life. But symptoms can range wider, including development delays, decreased muscle tone, and difficulty walking and speaking. There is also a risk of sudden death in epilepsy.<\/p>\n Typically, children who are diagnosed with the condition will be treated with anti-seizure medicines to control their convulsions.<\/p>\n Capsida\u2019s therapy, dubbed CAP-002, aims to make up for deficient levels of a vital protein encoded by the STXBP1 gene that helps pass messages between nerves.<\/p>\n \u201cOn behalf of everyone at Capsida Biotherapeutics, we extend our heartfelt sympathies to the patient\u2019s family and loved ones,\u201d Capsida CEO Peter Anastasiou said in a statement emailed to BioPharma Dive. \u201cWe are working with the appropriate parties and will share information with the medical and patient communities as soon as we can.\u201d<\/p>\n \u201cWhile we do not have … answers yet, we are in close contact with the study sponsor and investigators and will share verified updates as the formal safety review progresses,\u201d the STXBP1 Foundation, a nonprofit patient advocacy group,\u00a0added in a separate community note<\/a>.<\/p>\n
\nCapsida disclosed the death in a letter Wednesday<\/a> to the patient community for the rare neurodevelopmental disease its gene therapy is designed to treat. In it, the biotechnology company noted it has informed the Food and Drug Administration and will soon provide regulators a full report of the patient\u2019s death.
\n\u201cWe understand this devastating news will raise questions and uncertainty, and we are working with urgency to gather information and find answers,\u201d Capsida wrote in its letter.<\/p>\n