Ultragenyx Pharmaceutical
RARE
Market cap $2.79B

29.51 USD

-1.53

4.93%

At close Jul 11, 4:00 PM EDT

After hours

28.40

-1.11

3.76%

1 day

-4.93%

5 days

-25.50%

1 month

-21.33%

3 months

-13.56%

6 months

-31.63%

Year to date

-28.29%

1 year

-32.76%

5 years

-63.99%

10 years

-75.20%

About: Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.

Employees: 1,294

Healthcare Biotechnology

0

Funds holding %

of 7,312 funds

0

Analysts bullish %

of 4 analysts

Fund manager confidence

Based on 2025 Q1 regulatory disclosures by fund managers ($100M+ AUM)

40% more funds holding in top 10

Funds holding in top 10: 5 [Q4 2024] → 7 (+2) [Q1 2025]

20% more repeat investments, than reductions

Existing positions increased: 125 | Existing positions reduced: 104

1.27% more ownership

Funds ownership: 94.76% [Q4 2024] → 96.02% (+1.27%) [Q1 2025]

4% more call options, than puts

Call options by funds: $43.9M | Put options by funds: $42.2M

5% less funds holding

Funds holding: 310 [Q4 2024] → 296 (-14) [Q1 2025]

13% less capital invested

Capital invested by funds: $3.68B [Q4 2024] → $3.22B (-$463M) [Q1 2025]

25% less first-time investments, than exits

New positions opened: 33 | Existing positions closed: 44

See Fund Rankings

Research analyst outlook

4 Wall Street Analysts provided 1 year price targets over the past 3 months

Low target

$64

117%

upside

Avg. target

$65

119%

upside

High target

$65

120%

upside

4 analyst ratings

4 positive

100%

0 neutral

0%

0 negative

0%

Wells Fargo Tiago Fauth	120%upside $65	Overweight Maintained	10 Jul 2025
Guggenheim Debjit Chattopadhyay	117%upside $64	Buy Reiterated	20 Jun 2025
William Blair Sami Corwin	120%upside $65	Outperform Initiated	28 May 2025
Morgan Stanley Jeffrey Hung	120%upside $65	Overweight Maintained	9 May 2025

See Analyst Rankings

Financial journalist opinion

Based on 12 articles about RARE published over the past 30 days

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat a rare genetic disorder, the company said on Friday.

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Proactive Investors

Ultragenyx shares plunge after bone disorder trial misses key interim goal

Shares of Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) slumped around 26% on Thursday afternoon after the company and partner Mereo BioPharma said their late-stage study of setrusumab in osteogenesis imperfecta failed to meet a key interim goal. Ultragenyx is developing setrusumab as a potential first-in-class treatment for osteogenesis imperfecta, a rare genetic bone disorder with no approved therapies in the US.

Ultragenyx shares plunge after bone disorder trial misses key interim goal

Zacks Investment Research

RARE, MREO Fall as Osteogenesis Study Likely to Continue Till Year End

Ultragenyx Pharmaceutical and Mereo BioPharma shares tumble as investors await key phase III UX143 osteogenesis data now set for release at year-end.

RARE, MREO Fall as Osteogenesis Study Likely to Continue Till Year End

Investors Business Daily

Ultragenyx, Mereo Hammered For Their Surprise Miss In Brittle Bone Disease

Ultragenyx and Mereo stocks crashed Thursday after their treatment for brittle bone disease missed its mark in a second interim look.

Ultragenyx, Mereo Hammered For Their Surprise Miss In Brittle Bone Disease

Zacks Investment Research

Ultragenyx (RARE) Surges 5.1%: Is This an Indication of Further Gains?

Ultragenyx (RARE) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings estimate revisions for the stock doesn't suggest further strength down the road.

Ultragenyx (RARE) Surges 5.1%: Is This an Indication of Further Gains?

Ultragenyx, Mereo Shares Dive As Brittle Bone Disease Drug Misses Early Win

Ultragenyx Pharmaceutical Inc. RARE and Mereo BioPharma Group plc MREO stocks are plunging on Thursday.

Ultragenyx, Mereo Shares Dive As Brittle Bone Disease Drug Misses Early Win

Methode Electronics, Ultragenyx Pharmaceutical And Other Big Stocks Moving Lower In Thursday's Pre-Market Session

U.S. stock futures were lower this morning, with the Dow futures falling around 100 points on Thursday.

Methode Electronics, Ultragenyx Pharmaceutical And Other Big Stocks Moving Lower In Thursday's Pre-Market Session

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

Data from Orbit and Cosmic studies expected around the end of the year Data from Orbit and Cosmic studies expected around the end of the year

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

Zacks Investment Research

RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome

Ultragenyx Pharmaceuticals' GTX-102 earns FDA Breakthrough status after early data shows sustained progress in Angelman syndrome patients.

RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome

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