# Ultragenyx Pharmaceutical (RARE) Stock Price, News & Catalysts
Canonical URL: https://biopharmsignal.com/company/RARE
## Company Profile
- Ticker: RARE
- Exchange: NASDAQ
- Sector: Biopharma
- Location: United States
- Tags: Rare Disease
## Stock Data
- Close: 26.86 USD
- Market cap: 2145095298.72 USD
- Last updated: 2026-08-12
## Business Overview
### Rare-Disease Portfolio
Ultragenyx Pharmaceutical develops and commercializes therapies for serious rare and ultrarare genetic diseases. Its portfolio spans antisense oligonucleotides, enzyme and small-molecule medicines, and adeno-associated virus gene therapies.
### GTX-102 for Angelman Syndrome
GTX-102, or apazunersen, is an antisense oligonucleotide designed to reactivate the normally silenced paternal copy of UBE3A in neurons. It is being evaluated in the Phase 3 Aspire study for patients with a full maternal UBE3A deletion and the Phase 2/3 Aurora study across other genotypes and age groups.
### DTX401 for GSDIa
DTX401, or pariglasgene brecaparvovec, is an AAV8 gene therapy under FDA review for glycogen storage disease type Ia. The program is designed to restore glucose-6-phosphatase activity and improve metabolic control through one-time treatment.
### UX111 for Sanfilippo Syndrome
UX111, or rebisufligene etisparvovec, is an AAV9 gene therapy under FDA review for Sanfilippo syndrome type A. The resubmitted application seeks accelerated approval in a progressive pediatric neurodegenerative disease.
### DTX301 for OTC Deficiency
DTX301, or avalotcagene ontaparvovec, is an AAV8 gene therapy for ornithine transcarbamylase deficiency. The Phase 3 program has demonstrated a reduction in 24-hour plasma ammonia, supporting continued development toward registration.
## Why The Stock Is Moving

As of Aug 12, 2026, RARE closed down 0.37% at $26.86. No new same-company headline was identified in the current news window.

## Future Catalysts

- Phase 3 Aspire data for GTX-102 expected in the second half of 2026.
- Completion of enrollment in the Phase 2/3 Aurora study expected in the second half of 2026.
- FDA action on the DTX401 BLA expected by August 23, 2026.
- FDA action on the UX111 BLA expected by September 19, 2026.
- Additional DTX301 clinical data expected in the first half of 2027.

## Recent News
- 2026-08-04 20:05 EST — [Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update](https://biopharmsignal.com/news/RARE/ultragenyx-reports-second-quarter-2026-financial-results-and-corporate-update-629023e6) — Source: Ultragenyx Pharmaceutical
- 2026-07-28 20:05 EST — [Ultragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update](https://biopharmsignal.com/news/RARE/ultragenyx-to-host-conference-call-for-second-quarter-2026-financial-results-and-b9eb5649) — Source: Ultragenyx Pharmaceutical
- 2026-07-24 20:30 EST — [Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)](https://biopharmsignal.com/news/RARE/ultragenyx-reports-inducement-grant-under-nasdaq-listing-rule-5635-c-4-65f2b9fb) — Source: Ultragenyx Pharmaceutical
- 2026-07-08 13:08 EST — [Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact](https://biopharmsignal.com/news/RARE/ultragenyx-releases-2025-impact-report-emphasizing-commitment-to-rare-disease-pa-6c0c6160) — Source: Ultragenyx Pharmaceutical
- 2026-06-03 20:30 EST — [Ultragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference](https://biopharmsignal.com/news/RARE/ultragenyx-to-participate-at-goldman-sachs-47th-annual-global-healthcare-confere-aa3364c0) — Source: Ultragenyx Pharmaceutical