Building healthier futures powered by thoughtful science
We are a highly focused, research-driven biopharmaceutical company working in rare diseases. We're exploring the potential of cutting-edge science as a subsidiary of Nippon Shinyaku Co., Ltd. In Japan.
Advancing rare disease research
Inspired by our own families, as well as the individuals we aim to provide with treatment, NS Pharma is conducting advanced research in rare diseases, including exon-skipping technology, to offer new, effective treatment options for Duchenne. We’re also developing JAK1 inhibition for the potential treatment of eosinophilic granulomatosis with polyangiitis (EGPA).
Therapeutic areas
NS Pharma is working to have an important impact on the lives of patients with rare diseases. Our therapeutic areas encompass neurological diseases and inflammatory diseases.
Scientific approaches
Harnessing the power of innovative science can help bring new treatments to patients with rare diseases. NS Pharma is developing therapies through exon-skipping, cell therapy and JAK1 inhibition technology.
Therapeutic areas
NS Pharma is working to have an important impact on the lives of patients with rare diseases. Our therapeutic areas encompass neurological diseases and inflammatory diseases.
Scientific approaches
Harnessing the power of innovative science can help bring new treatments to patients with rare diseases. NS Pharma is developing therapies through exon-skipping, cell therapy and JAK1 inhibition technology.
From our blog
Press Release / April 18, 2025
FDA Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis
Press Release / March 27, 2025
Positive Data Showing Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Released at 2025 MDA Conference
Press Release / March 6, 2025
FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment
Press Release / April 18, 2025
FDA Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis
Press Release / March 27, 2025
Positive Data Showing Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Released at 2025 MDA Conference
Press Release / March 6, 2025
FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment
A warm #Welcome to Heather Greenberg, our new Associate Director of the Patient Services Hub!
Heather brings 20+ years of expertise in patient access and reimbursement, notably in #RareDiseases! Outside of work, she's a passionate runner and beach lover.
Meet Ross, our new Director of Market Access Marketing!
Ross brings 20+ years of pharma commercial leadership experience across sales, operations, marketing, and market access. When he’s not at work, he enjoys spending time with his family.
We are proud to be joining @REGENXBIO as a Visionary sponsor of the 2025 @Projectalive Building A Future Together MPS II Community Conference this weekend, bringing together members of the Hunter syndrome community for a day of learning, connection and fun. Hope to see you there! https://x.com/NSPharmaInc/status/1918322518185873656/photo/1