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

Social media

nspharmainc NS Pharma @nspharmainc ·
28 Apr

We're in Las Vegas for the 2025 Asembia Summit this week!

Let’s connect and explore what’s next in access, advocacy and rare disease care. We’re ready for the conversation!

#Asembia2025 #PatientAccess #RareDiseaseCare #AXS25 https://x.com/NSPharmaInc/status/1916855684001370505/photo/1

nspharmainc NS Pharma @nspharmainc ·
25 Apr

NS Pharma is thrilled to continue our journey with the #Duchenne community by sponsoring another @CureDuchenne Community Dinner.

📍 Location: True Foods, San Diego
🕒 Time: 6:00PM - 9:00PM PT

Secure your place at the table: https://ow.ly/lgxV50VFw1R

#CureDuchenneCares https://x.com/NSPharmaInc/status/1915767757037019295/photo/1

nspharmainc NS Pharma @nspharmainc ·
23 Apr

We’re thrilled to announce our sponsorship of the upcoming #JettFoundationFamilyWorkshop this Saturday in Iowa City!

This workshop is a fantastic opportunity to connect with specialists, share experiences, and gain insights that empower the #DuchenneCommunity.

See you there! https://x.com/NSPharmaInc/status/1915043616902234170/photo/1

nspharmainc NS Pharma @nspharmainc ·
21 Apr

We’re proud to share that the FDA has granted #OrphanDrug designation to NS-229 for the treatment of #EGPA — a rare autoimmune disease that affects fewer than 200,000 people in the U.S!

Check out the press release: https://ow.ly/zYkj50VEL8x

#RareDisease #FDA https://x.com/NSPharmaInc/status/1914333149225509365/photo/1

nspharmainc NS Pharma @nspharmainc ·
17 Apr

What is #Dystrophin? 🤔

It's a vital protein that plays a key role in supporting muscles as they contract and relax.

Without it, muscles become more prone to damage and gradually weaken, leading to mobility challenges.

Visit http://WeSpeakDuchenne.com to learn more! https://x.com/NSPharmaInc/status/1912869018521817581/photo/1

nspharmainc NS Pharma @nspharmainc ·
15 Apr

Are you ready to take the lead on something big?

We're looking for a dynamic leader to join us as our Executive Senior Director of Regulatory Affairs!

Think you've got the experience and the drive? Apply here: https://ow.ly/euPT50VB1aN

#PharmaJobs #Careers #Hiring https://x.com/NSPharmaInc/status/1912182937035977059/photo/1

nspharmainc NS Pharma @nspharmainc ·
11 Apr

We’re thrilled to welcome Tanya as our new Director of Supply Chain!

With 20+ years of experience in pharma, she brings a wealth of expertise in leading and shaping global clinical and commercial #SupplyChain operations.

#Welcome to the team!

#NewHire #GrowingTeam https://x.com/NSPharmaInc/status/1910694667563258037/photo/1