企業プロフィール
Ultragenyx Pharmaceutical 株価・ニュース・カタリスト
- ティッカー:
- RARE
- 取引所:
- NASDAQ
- セクター:
- Biopharma
- 所在地:
- United States
株価パフォーマンス
26.13 USD (-1.36%)
終値
最終更新: 2026年8月17日
2.15B USD
時価総額
株価変動の要因
As of Aug 17, 2026, RARE closed down 1.36% at $26.13. No new same-company headline was identified in the current news window.
企業概要
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.
今後のカタリスト
- 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.
最近のニュース
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類似銘柄
事業成熟度、治療領域、カタリスト傾向が近い同業銘柄です。