-Participating in the 'World Orphan Drug Congress USA 2026' in Boston from June 9 to 11
-Unveiling research achievements of 'DDC-02' targeting rare genetic pediatric brain diseases, including Pitt-Hopkins syndrome
-Discovered via the proprietary AI platform 'JWave,' anticipated to expand global partnership opportunities
May 2026
JW Pharmaceutical announced on the 14th that it will participate in the 'World Orphan Drug Congress USA 2026,' held in Boston, USA, from June 9 to 11, to present the preclinical research results of 'DDC-02' (tentative development code), a novel drug candidate for rare pediatric brain diseases.
DDC-02 is a First-in-Class oral small-molecule compound targeting rare genetic pediatric brain disorders, including Pitt-Hopkins syndrome.
Focusing on the degradation of Wnt signaling typically observed in these conditions, JW Pharmaceutical has been meticulously researching a small-molecule therapeutic capable of normalizing this deficient signaling pathway.
The newly unveiled DDC-02 was discovered utilizing 'JWave,' JW Pharmaceutical's proprietary, integrated AI-based drug discovery platform. Notably, by leveraging the platform's advanced genomic analysis capabilities, the company significantly enhanced research efficiency and precision, successfully elucidating the molecular understanding of the disease and the drug's mechanism of action (MoA).
DDC-02 is currently undergoing comprehensive safety evaluations. Through its upcoming presentation at the congress, JW Pharmaceutical plans to showcase the candidate's robust research milestones and developmental potential to the global market, while actively exploring diverse collaborative opportunities.
“This presentation is a meaningful opportunity to demonstrate the global potential of JW's innovative drug candidate in the field of rare pediatric brain diseases,” an official from JW Pharmaceutical stated. “Through in-depth interactions with leading experts in the rare disease sector, we will expand various collaboration opportunities, including technological partnerships, and accelerate the development of novel drugs that can significantly contribute to improving patients' quality of life.”
■ About the World Orphan Drug Congress USA
The World Orphan Drug Congress USA is a premier global event dedicated to the advancement of therapeutics for rare diseases. It serves as a vital networking and knowledge-sharing platform where key stakeholders across the orphan drug ecosystem—including major pharmaceutical and biotechnology companies, government and regulatory agencies, patient advocacy groups, and investors—convene to discuss the latest industry trends and innovative case studies.