SEOUL -- GC Pharma, a biopharmaceutical company in South Korea, tied up with Speragen, a bio company in the United States, to develop enzyme replacement therapy for a rare disease called "succinic semialdehyde dehydrogenase deficiency (SSADHD). They aim to embark on clinical trials in the second half of 2023.
SSADHD is a rare autosomal recessive disorder caused by an enzyme deficiency in GABA degradation. Symptoms include hypotonia and intellectual disabilities. While SSADH has been studied for decades, knowledge of the disorder and its pathophysiology remains unclear.
The two companies agreed to develop enzyme replacement therapy using SSADH proteins. The goal is to develop the first-in-class of SSADH. GC Pharma said it would conduct clinical trials, biomarker research and the development of drugs based on its technology in enzyme treatment. "This cooperation will strengthen our rare disease pipeline for patients," GC Pharma CEO Huh Eun-chul said in a statement on July 20.
GC Pharm said it 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 secure new patients. The U.S. company has focused on developing therapies for ultra-rare pediatric neurometabolic disorders.
PFDD is a systematic approach to help ensure that patients' experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. The primary goal is to better incorporate the patient’s voice in drug development and evaluation.
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