Rare drug-specialized bio venture Novelpharma announced on the 31st that it participated as a partner sponsor in the ‘Sanfilippo Community Conference Advance 2023 (ADVANCE 2023, Sanfilippo Community Conference)’ and presented preclinical research results and clinical preparation status.
‘ADVANCE 2023’ is an international event hosted by the Cure Sanfilippo Foundation, the largest Sanfilippo syndrome patient association in the United States. Global pharmaceutical and bio companies currently developing new drugs gather to share the status of new drug R&D for Sanfilippo syndrome, industry trends, and to build networks.
The event was held online for two days starting on the 29th (local time). Industry professionals from around the world, including Novelpharma, GC Green Cross, JCR Pharmaceuticals, Denali Therapeutics, Orchard Therapeutics, Ultragenyx, and authorities in the rare disease field gathered in one place.
Dr. Dongkyu Jin, founder of Novelpharma, gave a speech on the second day. He introduced the innovative new drug for Sanfilippo syndrome type A (MPS IIIA), which has been jointly developed with GC Green Cross since 2020, and presented the research and development and clinical preparation status as well as clinical plans.
A company official said, "The ‘ADVANCE 2023’ event was held amid deep interest from Sanfilippo syndrome patients and their families, who currently have no treatment options," adding, "At this event, we disclosed preclinical results and clinical plans for the Sanfilippo syndrome type A treatment currently under development, attracting significant attention from patients and experts." He further added, "We plan to apply for clinical approval from the U.S. Food and Drug Administration (FDA) early next year and accelerate global clinical trials."
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