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 ·
6h

We're excited to be sponsoring the @JettFoundation’s #CampPromise!

This barrier-free camp empowers people with DMD & other neuromuscular diseases. We're proud to support the #DuchenneCommunity.

Learn more: https://ow.ly/1tU550Wiiwp

#RareDiseases #MuscularDystrophy https://x.com/NSPharmaInc/status/1939740386261406185/photo/1

nspharmainc NS Pharma @nspharmainc ·
25 Jun

Ready for a new challenge where you can make a real impact?

We’re looking for a driven individual to join us as our Director of Launch Excellence and Brand Planning!

Think you might be the right fit? We’d love to hear from you!

Apply now: https://ow.ly/HIRy50WbJ1G

#PharmaJobs https://x.com/NSPharmaInc/status/1937873725115826503/photo/1

nspharmainc NS Pharma @nspharmainc ·
23 Jun

Meet Demet Pilge, our new Associate Director of HCP Marketing!

Demet brings 20+ years of marketing experience across Psychiatry, Neurology, Oncology, ENT & Respiratory. In her free time, she enjoys traveling with her family and unwinding with piano.

Welcome, Demet!

#NewHire https://x.com/NSPharmaInc/status/1937148974487453786/photo/1

nspharmainc NS Pharma @nspharmainc ·
20 Jun

We're excited to be in Minneapolis for the #HopeInFocus Conference!

This inspiring event brings together families, researchers & professionals from around the world to share insights & explore advances in #InheritedRetinalDiseases.

Also attending? Let's connect!

#RareDisease https://x.com/NSPharmaInc/status/1936061920462700915/photo/1

nspharmainc NS Pharma @nspharmainc ·
18 Jun

Viva Las Vegas! We’re en route to #PPMD2025, the largest global gathering of Duchenne and Becker families.

Be sure to stop by booth #4 and say hello. Our team would love to meet you! 💙

#Duchenne #BeckerMuscularDystrophy https://x.com/NSPharmaInc/status/1935337215875314166/photo/1

nspharmainc NS Pharma @nspharmainc ·
16 Jun

Fresh insights on heart health in #Duchenne!🫀

New research underscores the value of early cardiac treatment in DMD, helping to preserve heart function and delay cardiomyopathy-related complications.

Check out the full article: https://ow.ly/4GxH50Wa41R

#Deramiocel #Cardiac https://x.com/NSPharmaInc/status/1934611881789943822/photo/1

nspharmainc NS Pharma @nspharmainc ·
13 Jun

We’re delighted to be supporting the upcoming Akari Foundation Workshop this weekend! 💙

Workshops like this one provide vital resources, connections and support for families navigating life with Duchenne!

See you there!

#AkariFoundation #DuchenneAwareness #RareDisease https://x.com/NSPharmaInc/status/1933524762900509106/photo/1