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 / October 14, 2025

Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress

Press Release / September 19, 2025

FDA Grants Orphan Drug Designation to NS-051/NCNP-04 for the Treatment of Duchenne Muscular Dystrophy

Press Release / September 9, 2025

FDA Grants Fast Track Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis

Press Release / October 14, 2025

Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress

Press Release / September 19, 2025

FDA Grants Orphan Drug Designation to NS-051/NCNP-04 for the Treatment of Duchenne Muscular Dystrophy

Press Release / September 9, 2025

FDA Grants Fast Track Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis

Social media

We are conducting a Phase 2 #ClinicalTrial evaluating NS-229, an investigational JAK1 inhibitor, to learn more about potential new treatment options for adults with #EGPA.

See if you are eligible to join:
https://clinicaltrials.gov/study/NCT06046222#participation-criteria

#PatientRecruitment #NSPharma https://x.com/NSPharmaInc/status/1984260117773115792/photo/1

We’re proud to sponsor the Jett Foundation’s final 2025 Family Workshop in Norfolk, VA!

Join us on Saturday Nov 8 for a day of learning, connection & empowerment supporting the Duchenne community.

Register here: https://ow.ly/gSuH50Xif1a

#Duchenne #NSPharma #CommunitySupport https://x.com/NSPharmaInc/status/1983534797260575194/photo/1

🌟 Welcome Shannon Merkel to NS Pharma as our new Director of #MarketAccess Marketing Communications!

With 20+ years in market access, payer policy & launch excellence - including a decade at Takeda - she brings deep expertise & passion to our team. 💙

#NewHire #NSPharma https://x.com/NSPharmaInc/status/1983172404311425345/photo/1

Excited to share new data at #AANEMinSanFran!

Don’t miss our poster on exon-skipping therapy persistence:

Thu 10/30 - 6:15 PM
Fri 10/31 - 2:45 PM.

Join Mara Wood & Denise Quigley to discuss insights for the #Duchenne community. See you in San Francisco! 💙

#RareDiseases https://x.com/NSPharmaInc/status/1982810230544023727/photo/1

We’re excited to attend the 10th Annual #UPINFamilyDay Conference on Oct 25 at the Loveland Living Planet Aquarium! 🐠

A day of learning, connection & fun for families impacted by neuromuscular disorders.

Register to join us: https://ow.ly/fAV150XgP4w

#RareDiseases #NSPharma https://x.com/NSPharmaInc/status/1981722931592937623/photo/1

At #NSPharma, we’re committed to advancing science & hope for #RareDiseases.

This #HunterSyndromeAwareness Week (Oct 19–25), we stand with families & advocates to raise awareness of #MPSII, a rare genetic disorder that primarily affects boys.

Learn more: https://ow.ly/1Wna50XgOW1 https://x.com/NSPharmaInc/status/1981360732408013020/photo/1

The #AANEMinSanFran Annual Meeting kicks off next week!

We’re joining global experts from Oct 30-31 to explore the latest in NM, MSK, EDX & NMUS. Don’t miss our poster (#294) where we’ll share new insights on exon-skipping for the #Duchenne community. 💙

#RareDiseases https://x.com/NSPharmaInc/status/1980998359566389570/photo/1