GC Pharma partners with Speragen to develop first in class drug for a rare disease

South Korean biopharmaceutical firm GC Pharma has partnered with US-based biotech firm Speragen to develop a cure for a rare disease, according to the company, Tuesday.

According to GC Pharma, the two companies will develop an enzyme replacement therapy for a rare disease called succinic semialdehyde dehydrogenase deficiency, which currently has no treatment.

The two companies aim to start preclinical studies this year and phase 1 and 2 clinical trials during the second half of 2023.

Under the partnership, GC Pharma will be responsible for the overall drug discovery and development process, including biomarker research and clinical studies.

Speragen will meet with the US Food and Drug Administration for patient-focused drug development meetings, designed to take the patient into account throughout the entire drug development.

Speragen will be also responsible for recruiting patients for upcoming clinical trials.

“The latest partnership will help the company strengthen its rare disease pipeline for patients,” GC Pharma CEO Huh Eun-chul said.

SSADH is a rare genetic disorder characterized by lack of one of two enzymes involved in the breakdown of gamma-aminobutyric acid, the major inhibitory neurotransmitter in the brain.

Individuals with SSADH deficiency typically present with global developmental delay, hypotonia, epilepsy and ataxia.