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 26, 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 26, 2025

Positive Data Showing Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Released at 2025 MDA Conference

Social media

nspharmainc NS Pharma @nspharmainc ·
23 May

Say hello to Latoya Martin, our new Patient Engagement Lead for the North Central Region! 🎉

Latoya comes to us with 15+ years of experience in #Healthcare reimbursement, access education and patient support across specialty and #RareDiseases.

Welcome to the team!

#NewHire https://x.com/NSPharmaInc/status/1925914750849872020/photo/1

nspharmainc NS Pharma @nspharmainc ·
22 May

NS Pharma is honored to sponsor the 2025 @CureDuchenne #FUTURES2025!

Don’t miss the chance to connect with our Patient Services team. They're here to listen, guide and support you—every step of the way!

See you there!

#DuchenneAwareness #Duchenne #PatientSupport https://x.com/NSPharmaInc/status/1925552226233426229/photo/1

nspharmainc NS Pharma @nspharmainc ·
21 May

We’re just over a week away from the start of the @JettFoundation's National Challenge—and we’re lacing up in support!

Ready to join the challenge? Sign up here: https://ow.ly/pyXU50VWe4g

#JettChallenge #TeamJett #DuchenneAwareness https://x.com/NSPharmaInc/status/1925189843988201629/photo/1

nspharmainc NS Pharma @nspharmainc ·
19 May

Our team had a wonderful time at #ASGCT2025 last week!

It was an honor to present our latest research on the promising potential of NS-089/NCNP-02 (Brogidirsen).

If you missed Dr. Aoki’s presentation, you can view it here: https://ow.ly/MbIX50VUVjA 👈

#GeneTherapy #CellTherapy https://x.com/NSPharmaInc/status/1924465920056938916/photo/1

nspharmainc NS Pharma @nspharmainc ·
16 May

We’re heading to Birmingham, AL, for the @JettFoundation's #FamilyWorkshop tomorrow!

Are you also attending? Make sure you stop by and say hello to our team - Patty, Yashly and Laura would love to meet you!

#DuchenneAwareness #JettFoundation #RareDiseases https://x.com/NSPharmaInc/status/1923377898716598391/photo/1

nspharmainc NS Pharma @nspharmainc ·
15 May

Happy 16th Birthday to our incredible #BrandAmbassador, Jordan! 🎂🎈

We hope today brings you all the joy and laughter you deserve—you’ve earned it!

Help us celebrate this incredible young man - drop your birthday wishes for Jordan below! 💙

#Birthday #ExonSkipping #Duchenne https://x.com/NSPharmaInc/status/1923030734324810226/video/1

nspharmainc NS Pharma @nspharmainc ·
15 May

Today is #MPSAwarenessDay!💜

MPS is a group of rare, progressive genetic disorders and we’re working hard to advance new potential #GeneTherapies for MPS I and MPS II in partnership with @REGENXBIO!

Learn more about #MPS here: https://impsn.ca/mpsday/

#MPSDAY2025 #RareDiseases https://x.com/NSPharmaInc/status/1923015523757092916/photo/1