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 / May 14, 2025

FDA Accepts Biologics License Application for Mucopolysaccharidosis II 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 / May 14, 2025

FDA Accepts Biologics License Application for Mucopolysaccharidosis II 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

Social media

Another big step forward for the #MPSII community!

We’re delighted to share that the FDA has accepted the Biologics License Application for RGX-121, a potential first-in-class, one-time #GeneTherapy developed by our partners at @REGENXBIO!

Learn more: https://ow.ly/btLH50VSEmF👈 https://x.com/NSPharmaInc/status/1922667899988193512/photo/1

We're in New Orleans for #ASGCT2025!

Don't miss Dr. Aoki's presentation tomorrow at 5:30pm in Poster Hall I2. They'll be sharing results from the NS-089 clinical trial, offering new insights into potential therapies for #Duchenne muscular dystrophy.

#GeneTherapy #CellTherapy https://x.com/NSPharmaInc/status/1922291545912311886/photo/1

Meet Yashly Garcia, our new Patient Engagement Lead for the South Central Region!🎉

Yashly brings years of patient experience to the team. Outside of work, she also loves to hike, try out new recipes and hang out with her pup, Rowan 🐶

Welcome to the team!

#NSPharma #NewHire https://x.com/NSPharmaInc/status/1921929194910466463/photo/1

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.

#NewHire #GrowingTeam https://x.com/NSPharmaInc/status/1920849518288891947/photo/1

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.

#Welcome to the team!

#NewHire https://x.com/NSPharmaInc/status/1919754533720281572/photo/1

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

Join our growing team at NS Pharma! 🚀

We're excited to announce an opening for an Associate Director of Regulatory Affairs, Strategy.

Want to make a significant impact in the pharmaceutical industry? Apply here: https://ow.ly/5eN550VHEhK

#Hiring #PharmaCareers #JobOpportunity https://x.com/NSPharmaInc/status/1918304715642753287/photo/1