News

The U.S. Food and Drug Administration (FDA) is requesting changes to Ultragenyx Pharmaceutical’s manufacturing process before it will consider accelerated approval of UX111, the company’s experimental gene therapy for Sanfilippo syndrome type A. In a corporate update earlier this week, Ultragenyx stated it “believes the observations are readily…

The European Commission has granted orphan drug status to JR-446, a brain-penetrating enzyme replacement therapy (ERT) for Sanfilippo syndrome type B. The designation, announced in a press release from developer JCR Pharmaceuticals, is aimed at accelerating the clinical development and review of investigational therapies that have the…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to JR-446, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type B, the company has announced. The designation is meant to accelerate the clinical development and review of investigational therapies with the potential…

Spruce Biosciences said it plans to launch a Phase 3 clinical study of tralesinidase alfa as an enzyme replacement therapy for children with Sanfilippo syndrome type B, a move toward seeking accelerated approval of the treatment. The company said it plans to file a biologics license application (BLA) in…

A hematopoietic stem and progenitor cell (HSPC) transplant eased several disease symptoms, including motor problems and enlarged organs, in a new mouse model of Sanfilippo syndrome type C, according to a study. HSPC transplant provides blood cell precursors from a healthy donor, which are then able to develop into…

The U.S. Food and Drug Administration (FDA) has accepted Ultragenyx Pharmaceutical‘s application seeking approval of UX111, a gene therapy for Sanfilippo syndrome type A, and granted it priority review, which will speed the agency’s decision. By being granted priority review, the biologics license application (BLA) will be…

Ultragenyx Pharmaceutical’s experimental gene therapy UX111 slows or halts the brain damage that affects communication and cognitive and motor skills in children with Sanfilippo syndrome type A, according to new trial data. While younger children receiving the one-time therapy saw gains in these functional domains compared with untreated age-matched…

Ultragenyx Pharmaceutical has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of its gene therapy UX111 for Sanfilippo syndrome type A. If approved, UX111 would become the first therapy to be cleared in the U.S. for Sanfilippo, a…

The release of extracellular vesicles (EVs) from microglia may play a key role in driving neurodegeneration in Sanfilippo syndrome. EVs are tiny particles that carry a variety of cargo, such as proteins and DNA, between cells to facilitate cell-to-cell communication. Microglia are a class of immune cells in the…

A Phase 1 clinical trial in Japan that’s testing JCR Pharmaceuticals‘ experimental enzyme replacement therapy JR-441 for treating Sanfilippo syndrome type A has dosed its first patient. The study will test the therapy as a once-weekly infusion into the bloodstream in children and adolescents, ages 1-18 to…