NIPPON SHINYAKU CO

Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.

REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027.

The FDA has granted Orphan Drug Designation to NS-229, a selective JAK1 inhibitor being developed by NS Pharma for eosinophilic granulomatosis with polyangiitis (EGPA).

Durham-based Atsena Therapeutics has raised $150 million in an oversubscribed Series C financing led by Bain Capital to advance gene therapies for inherited blindness conditions.

REGENXBIO and Nippon Shinyaku have entered an exclusive partnership to develop and commercialize RGX-121 for MPS II and RGX-111 for MPS I.

Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.

Capricor Therapeutics' deramiocel has been granted Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations by the EMA for Duchenne muscular dystrophy (DMD).

The FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL).

Atsena Therapeutics and Nippon Shinyaku have entered an exclusive license agreement to commercialize ATSN-101 in the U.S. and Japan, a gene therapy for Leber congenital amaurosis (LCA1).

Data from the Galactic53 trial, a Phase II study of viltolarsen in Duchenne muscular dystrophy (DMD), has been published in Scientific Reports, highlighting its effects on pulmonary function.