South Korea’s GC Pharma has signed a partnership agreement with U.S. Speragen for joint development of a first-in-class therapy for succinic semialdehyde dehydrogenase deficiency (SSADHD), a rare inherited disorder that commonly affects young children, the company said on Tuesday.
SSADHD disrupts the metabolism of neurotransmitter gamma-aminobutyric acid (GABA) and causes neurological symptoms that include epilepsy and developmental delays.
Currently, there is no treatment available for this disease, and only anticonvulsant medications are prescribed to relieve seizure symptoms.
The two companies aim to develop enzyme replacement therapy using SSADHD proteins.
GC Pharma is entitled to a plasmid-related patent for SSADHD protein production while Speragen is responsible for patient-focused drug development (PFDD) with the U.S. Food and Drug Administration (FDA) and neonatal screening to enroll new patients, according to GC Pharma.
GC Pharma said it will conduct clinical trials and biomarker research based on its enzyme treatment technology with a goal of initiating Phase 1/2 clinical trials in the second half of 2023.
GC Pharma shares closed Tuesday down 0.16 percent at 317,000 won ($275.53).
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