Company Profile
Ultragenyx Pharmaceutical Stock Price, News & Analysis
- Ticker:
- RARE
- Exchange:
- NASDAQ
- Sector:
- Biopharma
- Location:
- United States
Stock performance
25.82 USD (+3.57%)
Close
Last updated: August 6, 2026
2.15B USD
Market cap
Why the stock is moving
As of Aug 6, 2026, RARE closed up 3.57% at $25.82. No new same-company headline was identified in the current news window.
Company 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.
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
- 01
Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Source: Ultragenyx Pharmaceutical
- 02
Ultragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update
Source: Ultragenyx Pharmaceutical
- 03
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Source: Ultragenyx Pharmaceutical
- 04
Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
Source: Ultragenyx Pharmaceutical
- 05
Ultragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference
Source: Ultragenyx Pharmaceutical
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Frequently Asked Questions
What is the current stock price of Ultragenyx Pharmaceutical (RARE)?
Ultragenyx Pharmaceutical (RARE) most recently closed at 25.82 USD, a +3.57% move versus the previous session.
What is the market cap of Ultragenyx Pharmaceutical (RARE)?
Ultragenyx Pharmaceutical currently has a market capitalization of 2.15B USD on BioPharmSignal.
What does Ultragenyx Pharmaceutical do?
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...
On which exchange is RARE stock listed?
Ultragenyx Pharmaceutical (RARE) is associated with NASDAQ, United States, Biopharma.
What is GTX-102?
Phase 3 Aspire data for GTX-102 expected in the second half of 2026.
Which conditions is Ultragenyx Pharmaceutical targeting with GTX-102?
Ultragenyx Pharmaceutical is associated with metabolic disease, genetic disease based on its company overview and current catalyst commentary.
How does Ultragenyx Pharmaceutical describe recent progress with GTX-102?
Phase 3 Aspire data for GTX-102 expected in the second half of 2026.
What is Rare-Disease?
Rare-Disease Portfolio Ultragenyx Pharmaceutical develops and commercializes therapies for serious rare and ultrarare genetic diseases.