Compare Stocks

Comparative Price Performance Over Time

Compare Stocks - Price & Volume

CompanyCurrent Price50-Day Moving Average52-Week RangeMarket CapBetaAvg. VolumeToday's Volume
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
$99.99
$97.65
$45.50
$100.00
$6.17B0.551.56 million shs107 shs
ArQule, Inc. stock logo
ARQL
ArQule
$20.00
$20.00
$3.15
$20.45
$2.42B2.495.27 million shsN/A
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
$10.71
-1.0%
$12.08
$9.70
$14.57
$3.16B0.753.04 million shs5.09 million shs
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
$2.80
+8.1%
$2.76
$0.50
$3.22
$389.35M1.682.81 million shs6.56 million shs
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
$45.11
+3.3%
$47.95
$31.52
$54.98
$3.71B0.65763,528 shs836,693 shs
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Price Performance

Company1-Day Performance7-Day Performance30-Day Performance90-Day Performance1-Year Performance
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
0.00%0.00%0.00%0.00%0.00%
ArQule, Inc. stock logo
ARQL
ArQule
0.00%0.00%0.00%0.00%0.00%
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
-1.02%+1.42%-7.11%-14.80%-6.79%
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
+8.11%-4.44%-5.72%+7.07%+5.26%
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
+3.25%+6.62%-1.61%+3.44%+3.44%

MarketRank™

CompanyOverall ScoreAnalyst's OpinionShort Interest ScoreDividend StrengthESG ScoreNews and Social Media SentimentCompany OwnershipEarnings & Valuation
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
N/AN/AN/AN/AN/AN/AN/AN/A
ArQule, Inc. stock logo
ARQL
ArQule
N/AN/AN/AN/AN/AN/AN/AN/A
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
3.8931 of 5 stars
3.50.00.04.32.70.81.9
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
3.717 of 5 stars
3.51.00.04.22.61.70.6
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
4.4475 of 5 stars
4.52.00.03.82.42.50.6

Analyst Ratings

CompanyConsensus Rating ScoreConsensus RatingConsensus Price Target% Upside from Current Price
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
N/AN/AN/AN/A
ArQule, Inc. stock logo
ARQL
ArQule
N/AN/AN/AN/A
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
3.00
Buy$20.0086.74% Upside
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
3.00
Buy$5.5096.43% Upside
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
2.91
Moderate Buy$90.33100.25% Upside

Current Analyst Ratings

Latest RARE, ARNA, FOLD, HRTX, and ARQL Analyst Ratings

DateCompanyBrokerageActionRatingPrice TargetDetails
4/23/2024
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
Capital One Financial
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Initiated CoverageOverweight$6.00
4/22/2024
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
Royal Bank of Canada
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Initiated CoverageOutperform$77.00
4/16/2024
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
Wedbush
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Reiterated RatingNeutral ➝ Neutral$48.00
4/11/2024
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
Needham & Company LLC
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Reiterated RatingBuy ➝ Buy$5.00
4/5/2024
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
Cantor Fitzgerald
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Reiterated RatingOverweight ➝ Overweight$107.00
3/18/2024
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
JPMorgan Chase & Co.
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Lower Price TargetOverweight ➝ Overweight$21.00 ➝ $19.00
3/18/2024
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
JPMorgan Chase & Co.
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Boost Price TargetOverweight ➝ Overweight$88.00 ➝ $92.00
3/13/2024
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
Needham & Company LLC
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Boost Price TargetBuy$4.00 ➝ $5.00
2/29/2024
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
Cantor Fitzgerald
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Reiterated RatingOverweight ➝ Overweight$22.00
2/27/2024
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
JPMorgan Chase & Co.
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Boost Price TargetOverweight ➝ Overweight$84.00 ➝ $88.00
2/27/2024
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
Cantor Fitzgerald
Subscribe to MarketBeat All Access for the recommendation accuracy rating
Reiterated RatingOverweight ➝ Overweight$107.00
(Data available from 4/24/2021 forward. View 10+ years of historical ratings with our analyst ratings screener.)

Sales & Book Value

CompanyAnnual RevenuePrice/SalesCashflowPrice/CashBook ValuePrice/Book
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
$50K123,305.67N/AN/A$10.94 per share9.14
ArQule, Inc. stock logo
ARQL
ArQule
$25.76M93.85N/AN/A$0.72 per share27.78
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
$399.36M7.92N/AN/A$0.55 per share19.47
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
$127.04M3.31N/AN/A($0.23) per share-12.17
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
$434.25M8.55N/AN/A$3.35 per share13.47

Profitability & Earnings

CompanyNet IncomeEPSTrailing P/E RatioForward P/E RatioP/E GrowthNet MarginsReturn on Equity (ROE)Return on Assets (ROA)Next Earnings Date
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
-$616.43M-$10.13N/AN/AN/AN/A-69.83%-62.35%N/A
ArQule, Inc. stock logo
ARQL
ArQule
-$15.48M-$0.16N/AN/AN/A-805.61%-33.10%-26.87%N/A
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
-$151.58M-$0.51N/A31.50N/A-37.96%-119.46%-20.40%5/8/2024 (Estimated)
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
-$110.56M-$0.86N/AN/AN/A-87.02%N/A-50.61%5/9/2024 (Estimated)
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
-$606.64M-$8.33N/AN/AN/A-139.70%-369.54%-44.74%5/2/2024 (Estimated)

Latest RARE, ARNA, FOLD, HRTX, and ARQL Earnings

DateQuarterCompanyConsensus EstimateReported EPSBeat/MissGap EPSRevenue EstimateActual RevenueDetails
3/12/2024Q4 2023
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
-$0.15-$0.07+$0.08-$0.07$30.98 million$34.23 million
2/28/2024Q4 2023
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
-$0.06-$0.11-$0.05-$0.11$115.11 million$115.08 million
2/15/2024Q4 2023
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
-$1.65-$1.52+$0.13-$1.52$119.38 million$127.39 million    

Dividends

CompanyAnnual PayoutDividend Yield3-Year Dividend GrowthPayout RatioYears of Consecutive Growth
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
N/AN/AN/AN/AN/A
ArQule, Inc. stock logo
ARQL
ArQule
N/AN/AN/AN/AN/A
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
N/AN/AN/AN/AN/A
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
N/AN/AN/AN/AN/A
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
N/AN/AN/AN/AN/A

Debt

CompanyDebt-to-Equity RatioCurrent RatioQuick Ratio
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
0.05
8.59
8.59
ArQule, Inc. stock logo
ARQL
ArQule
0.08
8.27
8.27
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
2.42
2.88
2.52
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
N/A
2.37
1.84
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
N/A
2.61
2.49

Ownership

Institutional Ownership

CompanyInstitutional Ownership
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
83.77%
ArQule, Inc. stock logo
ARQL
ArQule
74.63%
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
N/A
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
80.01%
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
97.67%

Miscellaneous

CompanyEmployeesShares OutstandingFree FloatOptionable
Arena Pharmaceuticals, Inc. stock logo
ARNA
Arena Pharmaceuticals
44861.66 million60.17 millionOptionable
ArQule, Inc. stock logo
ARQL
ArQule
36120.87 millionN/AOptionable
Amicus Therapeutics, Inc. stock logo
FOLD
Amicus Therapeutics
517295.38 million289.18 millionOptionable
Heron Therapeutics, Inc. stock logo
HRTX
Heron Therapeutics
126150.33 million140.71 millionOptionable
Ultragenyx Pharmaceutical Inc. stock logo
RARE
Ultragenyx Pharmaceutical
1,27682.30 million76.70 millionOptionable

RARE, ARNA, FOLD, HRTX, and ARQL Headlines

SourceHeadline
Analysts Offer Insights on Healthcare Companies: Neurocrine (NBIX) and Ultragenyx Pharmaceutical (RARE)Analysts Offer Insights on Healthcare Companies: Neurocrine (NBIX) and Ultragenyx Pharmaceutical (RARE)
markets.businessinsider.com - April 23 at 12:46 PM
Ultragenyx Pharmaceutical (NASDAQ:RARE) Coverage Initiated by Analysts at Royal Bank of CanadaUltragenyx Pharmaceutical (NASDAQ:RARE) Coverage Initiated by Analysts at Royal Bank of Canada
americanbankingnews.com - April 23 at 3:26 AM
Forecasting The Future: 11 Analyst Projections For Ultragenyx PharmaceuticalForecasting The Future: 11 Analyst Projections For Ultragenyx Pharmaceutical
markets.businessinsider.com - April 22 at 9:44 AM
Ultragenyx Pharmaceutical (NASDAQ:RARE) Receives New Coverage from Analysts at Royal Bank of CanadaUltragenyx Pharmaceutical (NASDAQ:RARE) Receives New Coverage from Analysts at Royal Bank of Canada
marketbeat.com - April 22 at 8:29 AM
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
globenewswire.com - April 19 at 4:30 PM
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Holdings Raised by Federated Hermes Inc.Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Holdings Raised by Federated Hermes Inc.
marketbeat.com - April 18 at 12:23 PM
Analysts’ Opinions Are Mixed on These Healthcare Stocks: Accolade (ACCD), Ultragenyx Pharmaceutical (RARE) and Bristol-Myers Squibb (BMY)Analysts’ Opinions Are Mixed on These Healthcare Stocks: Accolade (ACCD), Ultragenyx Pharmaceutical (RARE) and Bristol-Myers Squibb (BMY)
markets.businessinsider.com - April 18 at 9:38 AM
Ultragenyx Pharmaceuticals (RARE) "Neutral" Rating Reaffirmed at WedbushUltragenyx Pharmaceutical's (RARE) "Neutral" Rating Reaffirmed at Wedbush
americanbankingnews.com - April 17 at 4:44 AM
Ultragenyx Pharmaceutical (NASDAQ:RARE) Trading Up 1.9%Ultragenyx Pharmaceutical (NASDAQ:RARE) Trading Up 1.9%
marketbeat.com - April 16 at 1:40 PM
Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease StudyUltragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study
zacks.com - April 16 at 12:35 PM
Wedbush Reiterates Neutral Rating for Ultragenyx Pharmaceutical (NASDAQ:RARE)Wedbush Reiterates Neutral Rating for Ultragenyx Pharmaceutical (NASDAQ:RARE)
marketbeat.com - April 16 at 11:00 AM
Ultragenyx Eyes Pivotal Study After Mid-Stage Win in Angelman SyndromeUltragenyx Eyes Pivotal Study After Mid-Stage Win in Angelman Syndrome
biospace.com - April 16 at 9:55 AM
Mirae Asset Global Investments Co. Ltd. Has $5.18 Million Stock Position in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)Mirae Asset Global Investments Co. Ltd. Has $5.18 Million Stock Position in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)
marketbeat.com - April 16 at 5:04 AM
Ultragenyx Pharmaceutical (RARE) Set to Announce Earnings on WednesdayUltragenyx Pharmaceutical (RARE) Set to Announce Earnings on Wednesday
americanbankingnews.com - April 16 at 2:52 AM
Why Ultragenyx Pharmaceutical Shares Are Trading Lower By Around 9%? Here Are Other Stocks Moving In Mondays Mid-Day SessionWhy Ultragenyx Pharmaceutical Shares Are Trading Lower By Around 9%? Here Are Other Stocks Moving In Monday's Mid-Day Session
msn.com - April 15 at 6:23 PM
Ultragenyx says Angelman therapy is working, but safety questions remainUltragenyx says Angelman therapy is working, but safety questions remain
biopharmadive.com - April 15 at 1:23 PM
Ultragenyx Pharmaceutical (NASDAQ:RARE) Shares Gap Down to $47.00Ultragenyx Pharmaceutical (NASDAQ:RARE) Shares Gap Down to $47.00
marketbeat.com - April 15 at 12:53 PM
Ultragenyx Reports Positive Interim Phase 1/2 Data With GTX-102 - Quick FactsUltragenyx Reports Positive Interim Phase 1/2 Data With GTX-102 - Quick Facts
markets.businessinsider.com - April 15 at 8:22 AM
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
finance.yahoo.com - April 15 at 8:22 AM
Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman SyndromeUltragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome
globenewswire.com - April 12 at 4:22 PM
Ultragenyx Pharmaceutical Inc.: Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual MeetingUltragenyx Pharmaceutical Inc.: Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting
finanznachrichten.de - April 12 at 9:58 AM
Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual MeetingUltragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting
globenewswire.com - April 12 at 8:00 AM
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Position Lessened by Peregrine Capital Management LLCUltragenyx Pharmaceutical Inc. (NASDAQ:RARE) Position Lessened by Peregrine Capital Management LLC
marketbeat.com - April 11 at 7:49 AM
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Given Consensus Rating of "Moderate Buy" by AnalystsUltragenyx Pharmaceutical Inc. (NASDAQ:RARE) Given Consensus Rating of "Moderate Buy" by Analysts
marketbeat.com - April 9 at 4:41 AM

New MarketBeat Followers Over Time

Media Sentiment Over Time

Company Descriptions

Arena Pharmaceuticals logo

Arena Pharmaceuticals

NASDAQ:ARNA
Arena Pharmaceuticals, Inc., a biopharmaceutical company, focuses on providing novel medicines with pharmacology and pharmacokinetics to patients worldwide. The company's investigational clinical programs include Etrasimod (APD334) that is in Phase III clinical trial for ulcerative colitis, Phase IIb/III clinical trial for Crohn's disease, Phase II clinical trial for alopecia areata, Phase III clinical trial for atopic dermatitis, and Phase IIb clinical trial for eosinophilic esophagitis; Olorinab (APD371), which is in Phase IIb clinical trial for the treatment of abdominal pain associated with irritable bowel syndrome; APD418 that is in Phase II clinical trial for acute heart failure; and Temanogrel for coronary microvascular obstruction that is in Phase II clinical trial. It also develops ralinepag (APD811), which is in Phase III clinical trial for pulmonary arterial hypertension. The company has collaboration agreements with United Therapeutics Corporation; Everest Medicines Limited; Eisai Co., Ltd. and Eisai Inc.; Boehringer Ingelheim International GmbH; Beacon Discovery, Inc.; and Aristea Therapeutics, Inc. Arena Pharmaceuticals, Inc. was incorporated in 1997 and is based in Park City, Utah. As of March 11, 2022, Arena Pharmaceuticals, Inc. operates as a subsidiary of Pfizer Inc.
ArQule logo

ArQule

NASDAQ:ARQL
ArQule, Inc., a biopharmaceutical company, researches and develops therapeutics for the treatment of cancer and rare diseases in the United States. The company's pipeline includes ARQ 531, an orally bioavailable, potent and reversible dual inhibitor of wild type and C481S-mutant Bruton's tyrosine kinase that is in Phase I trial for patients with B-cell malignancies refractory to other therapeutic options; and miransertib (ARQ 092), a potent and selective inhibitor of the protein kinase B (AKT), a serine/threonine kinase, which is in Phase Ib in combination with the hormonal therapy and anastrozole in patients with advanced endometrial cancer. Its pipeline also comprises ARQ 75, a potent and selective inhibitor of AKT that is in Phase I clinical development for solid tumors harboring AKT, phosphoinositide 3-kinase or phosphatase, and tensin homolog loss mutations. In addition, the company's pipeline includes Derazantinib (ARQ 087), a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family of kinases that is in a registrational clinical trial in intrahepatic cholangiocarcinoma in patients with FGFR2 fusions. ArQule, Inc. has license agreements with Basilea Pharmaceutica Limited and Roivant Sciences Ltd. The company was founded in 1993 and is headquartered in Burlington, Massachusetts.
Amicus Therapeutics logo

Amicus Therapeutics

NASDAQ:FOLD
Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.
Heron Therapeutics logo

Heron Therapeutics

NASDAQ:HRTX
Heron Therapeutics, Inc., a commercial-stage biotechnology company, focuses on enhancing the lives of patients by developing and commercializing therapeutic that enhances medical care. The company's product candidates utilize its proprietary Biochronomer, a drug delivery technology, which delivers therapeutic levels of a range of short-acting pharmacological agents over a period from days to weeks with a single administration. It offers SUSTOL (granisetron), an extended-release injection for the prevention of acute and delayed nausea and vomiting associated with moderately emetogenic chemotherapy, or anthracycline and cyclophosphamide combination chemotherapy regimens; and CINVANTI, an intravenous formulation of aprepitant, a substance P/neurokinin-1 receptor antagonist for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic cancer chemotherapy, as well as nausea and vomiting associated with moderately emetogenic cancer chemotherapy. The company is also developing ZYNRELEF, a dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of the nonsteroidal anti-inflammatory drug meloxicam; and APONVIE, an intravenous formulation of a substance P/neurokinin-1 receptor antagonist indicated for the prevention of postoperative nausea and vomiting in adults. The company was formerly known as A.P. Pharma, Inc. and changed its name to Heron Therapeutics, Inc. in January 2014. Heron Therapeutics, Inc. was founded in 1983 and is headquartered in San Diego, California.
Ultragenyx Pharmaceutical logo

Ultragenyx Pharmaceutical

NASDAQ:RARE
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.