Ultragenyx México, S. de R.L. de C.V.

The cell and gene therapy sectors are rapidly advancing, with significant developments like Capricor's BLA submission for DMD cardiomyopathy therapy, Arbor Biotechnologies' FDA clearance for a CRISPR-based therapy trial, Ultragenyx's BLA for MPSIII gene therapy, RESTEM's fast-tracked ULSC therapy for IIM, ViGeneron's rare pediatric disease designation for a retinitis pigmentosa gene therapy, and uniQure's public offering pricing shares at $17.

STAT's biotech newsletter highlights key sector events, including Ultragenyx and Praxis's Phase 3 trials, Trump's FDA transition plan, Eli Lilly's lawsuit involvement over tirzepatide, and Biden's science and tech awards to CRISPR scientists and mRNA vaccine developers.

Ultragenyx is developing setrusumab for Osteogenesis Imperfecta, with Phase 3 study results expected soon. Praxis Precision Medicines is working on ulixacaltamide for essential tremor, also awaiting Phase 3 study results this quarter.

Ultragenyx focuses on developing innovative treatments for rare and ultrarare diseases, aiming to deliver transformative therapies. With a robust clinical pipeline, the company prioritizes patient needs in both development and commercialization to benefit rare disease communities effectively.

UX 111 treatment shows rapid, sustained HS reduction in CSF for Sanfilippo syndrome type A patients, correlating with improved cognitive development. Common adverse events were mild to moderate liver enzyme elevations. FDA accepted CSF HS as a biomarker, enabling BLA submission and potential accelerated approvals for MPS disorders.

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Arcturus Therapeutics, a leader in mRNA medicines, leverages strategic partnerships, a diverse pipeline, and innovative delivery technology to maintain its competitive edge. Facing regulatory hurdles and competition, it focuses on expanding into new therapeutic areas and global markets, aiming for continued growth and development in the biotech industry.

Ultragenyx (RARE) submitted a BLA to the FDA for accelerated approval of UX111, ABO-102, an AAV gene therapy for Sanfilippo syndrome type A treatment.