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

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

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

Say hello to Tyler Shepard, our new Director of #PatientAccess on the #PatientServices team!

Tyler has 20+ years of experience in pharma sales, training and access & reimbursement management across a range of therapeutic areas.

We're thrilled to have him on the team!

#Welcome https://x.com/NSPharmaInc/status/1932800451671417259/photo/1

The JettFoundation #NationalChallenge is a wrap—and we’re so proud to have been part of it.

Together, we’ve logged over 15k steps!

A HUGE thanks to everyone who took part and to the Jett Foundation for leading the charge!🙌

#TeamJett #DuchenneAwareness https://x.com/NSPharmaInc/status/1932075215342756203/photo/1

4

This June, we’re putting purpose into motion! 💪

Victoria Ramirez, our amazing Clinical Research Manager is leading the #RelayForLife in Paramus & we couldn’t be prouder.

Let’s show team Victorious some love & support!

Donate here: https://ow.ly/amHv50W48Xt

#CancerSurvivorMonth https://x.com/NSPharmaInc/status/1930988556559790380/photo/1

Did you know, #ExonSkipping helps the body bypass a #GeneticMutation to produce #Dystrophin that’s 84–97% as long as usual.

Whereas, #GeneTherapy inserts an engineered micro-dystrophin gene, creating a protein that’s 32–40% as long.

Learn more: https://ow.ly/LzuM50W2qIh https://x.com/NSPharmaInc/status/1930263647038603288/photo/1

The @JettFoundation #NationalChallenge officially kicked off yesterday and Team NS Pharma is proud to sponsor this critical event!

Doesn't our team look fabulous in their Jett Foundation t-shirts?

How are you going to move for #TeamJett?! 💙

#DuchenneAwareness #RareDisease https://x.com/NSPharmaInc/status/1929538498320621624/photo/1