Ultragenyx México, S. de R.L. de C.V.

Cell and gene therapy sectors see significant progress, including Estrella Immunopharma's EB103 achieving complete response in B-cell lymphomas, NEXICART-2 trial moving to expansion cohort, Ultragenyx planning higher dose evaluation for UX701, Anixa Biosciences seeking FDA approval for second doses of FSHR-targeting CER T-cell therapy, Aspen Neuroscience collaborating with Cell X Technologies for iPSC automation, GEMMABio receiving $100 million from Fiocruz for accessible gene therapy research, MiNK Therapeutics partnering with Autonomous Therapeutics for metastatic tumor treatments, and BioHub Maryland receiving a $75,000 grant for bioprocessing training.

Ultragenyx plans to submit a protocol amendment for the phase 1/2/3 Cyprus2+ clinical trial evaluating UX701, an AAV vector-based gene therapy for Wilson disease, to include a higher dose cohort and optimized immunomodulation regimen. The new cohort will be added to stage 1, which has already dosed 15 patients. Six patients have fully tapered off standard of care treatment, with stabilization of nonceruloplasmin bound copper levels and improved ATP7b gene function noted. The gene therapy has been well-tolerated with no significant safety events.

FDA grants Breakthrough Therapy designation to setrusumab for osteogenesis imperfecta type I, III, or IV in patients aged 2+; setrusumab, a monoclonal antibody, inhibits sclerostin to enhance bone formation, mineral density, and strength. Positive phase 2 Orbit trial data showed 67% reduction in fracture rate and 22% increase in bone mineral density. Setrusumab previously received Orphan Drug and rare pediatric disease designations.

The Orphan Drugs Market is projected to grow from USD 225.9 Billion in 2023 to USD 493.21 Billion by 2031, driven by rising prevalence of rare diseases, unmet medical needs, favorable regulatory policies, technological advancements, and growing public and patient advocacy. Key challenges include high R&D costs, limited patient populations, pricing and reimbursement issues, and lack of long-term data. Trends include focus on gene therapies, personalized medicine, stakeholder collaboration, and expansion into emerging markets. North America leads the market, followed by Europe and Asia Pacific.

Ultragenyx plans accelerated FDA approval for UX111, a gene therapy for Sanfilippo syndrome, using CSF HS as a biomarker. Data from the Transpher A trial shows reduced HS levels and cognitive benefits in children. The company also faces a lawsuit over HeLa cell use in gene therapy production.

UX111 gene therapy shows rapid, sustained reductions in CSF biomarker levels in MPS IIIA patients treated before advanced neurodegeneration. Safety data from the Transpher A trial indicate no severe adverse events. Neurodevelopmental improvements and dose-dependent biomarker reductions were observed, with long-term follow-up ongoing.

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