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Yongin, South Korea and Austin, TX – GC Biopharma and Speragen announced a joint externally led patients-focused drug development meeting hosted by the SSADH Association. It was attended by the US FDA and other stakeholders to address the patient experience with SSADHD (Succinic Semialdehyde Dehydrogenase Deficiency), a disorder caused by mutations in the ALDH5A1 gene, which is inherited in an autosomal recessive manner.

The purpose of the meeting is for patients affected by SSADHD and their families to provide clear perspectives to researchers, academics, industry partners, and the FDA on living with their condition and what defines a meaningful treatment. In the meeting, 113 participants, including patients, families, KOLs, members of the FDA, GC Biopharma, and Speragen, were gathered from all over the world. The participants were communicating with the FDA about the impact of SSADHD on an individual's daily life, along with the types of treatment that benefit patients' lives, including perspectives on how well available therapies are working.

Based on the discussion, the SSADH Association will submit the Voice of the Patient report to the FDA by the end of this year. GC Biopharma and Speragen also plan to develop a clinical outcome assessment for developing ERT (Enzyme Replacement Therapy) for SSADHD to meet the patient's unmet needs.

"The meeting was a major step towards our ultimate goal of providing new treatment options to the patients and their families," said EC Huh, Ph. D., President of GC Biopharma. "We will continue our work to provide rare disease treatments and expand our R&D pipelines."

"We are so proud of the patients and families for sharing their experiences and impacts from living with SSADHD. It took great vulnerability and courage to be so open about their struggles," said Alice McConnell, CEO of Speragen.

About SSADHD

Succinic semialdehyde dehydrogenase deficiency (SSADHD) is a disorder that can cause a variety of neurological and neuromuscular problems. The signs and symptoms can be extremely variable among affected individuals. They may include mild to severe intellectual disability, developmental delay (especially involving speech), hypotonia, sleep disturbances, difficulty coordinating movements (ataxia), and/or seizures. Some affected individuals may also have decreased reflexes (hyporeflexia), nystagmus, hyperactivity, and/or behavioral problems. SSADH deficiency is caused by mutations in the ALDH5A1 gene and is inherited in an autosomal recessive manner. Management is generally symptomatic and typically focuses on treating seizures and neurobehavioral issues. Like so many rare diseases, there is no treatment for SSADHD worldwide.

About GC Biopharma

GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers lifesaving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century.

About Speragen

Speragen was co-founded by a mother of two children affected by SSADHD. Speragen knows first-hand the challenges of navigating life with a rare disease: from the daily challenges, the diagnostic odyssey, accepting the new reality post-diagnosis, finding a team of knowledgeable clinicians who can provide care, and finally, seeing the chasm between the current state of treatment and the possibility of a first-in-class drug becoming available.

Speragen comes from the Latin word "spera" meaning "hope." It was formed to help solve these issues more rapidly by bridging the gulf from hope to realizing therapy.

This press release may contain forward-looking statements which express the current beliefs and expectations of GC Biopharma's management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties, and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.

GC Biopharma Contacts

Seunghyun Baek
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Yelin Jun
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Sohee Kim
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Hansaem Kim
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Speragen Contacts

Alice McConnell
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