Ultragenyx Pharmaceutical (NASDAQ:RARE) Shares Down 6% - Here's Why
Ultragenyx Pharmaceutical (NASDAQ:RARE) shares dropped 6% in low-volume trading, extending declines below key moving averages, despite the company reporting better-than-expected quarterly results. Analyst sentiment has weakened, leading to several downgrades and price target cuts, resulting in a consensus "Hold" rating, though the average target price remains significantly higher than the current trading price. The biopharmaceutical company focuses on developing therapies for rare genetic diseases and reported a narrower loss and increased revenue in its latest quarter.
(RARE) as a Liquidity Pulse for Institutional Tactics
This article analyzes Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) using AI models to provide institutional trading strategies. Key findings include strong near and mid-term sentiment, a weak long-term outlook, and an exceptional risk-reward setup. The analysis offers specific strategies for position trading, momentum breakout, and risk hedging, along with multi-timeframe signal analysis.
Ultragenyx says EMA validates Sanfilippo gene therapy application
Ultragenyx Pharmaceutical Inc. announced that the European Medicines Agency (EMA) has validated its marketing authorization application for rebisufligene etisparvovec, a gene therapy for Sanfilippo syndrome type A. This validation means the scientific review process can begin in Europe, following the therapy's U.S. approval on September 17. Ultragenyx's stock (NASDAQ: RARE) saw a 3.19% increase following the announcement.
Ultragenyx Announces Marketing Authorisation Application
Ultragenyx announced that the European Medicines Agency (EMA) has validated its Marketing Authorisation Application (MAA) for the investigational gene therapy, rebisufligene etisparvovec, for Sanfilippo syndrome Type A (MPS IIIA). This validation marks a significant regulatory milestone, initiating the formal scientific review process. The company is also engaging with health authorities in other regions, including the UK and Saudi Arabia, to pursue future geographic expansion for this therapy for the rare, progressive inherited disorder.
Ultragenyx Pharmaceutical Inc. (RARE) Options Chain
This article provides the options chain data for Ultragenyx Pharmaceutical Inc. (RARE) on Yahoo Finance. It displays real-time stock prices, basic market information, and a disclaimer regarding delayed options data. Currently, there are no calls or puts listed as "in the money" for the company.
Operating margin % of Ultragenyx Pharmaceutical, Inc. – HAN:UP0
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Return on invested capital % of Ultragenyx Pharmaceutical, Inc. – HAN:UP0
This article provides financial information for Ultragenyx Pharmaceutical, Inc. (UP0) on the Hannover Stock Exchange, specifically focusing on its Return on Invested Capital (ROIC). The content is a data point from TradingView's platform, indicating the company's ROIC percentage over a given period.
Ultragenyx Pharmaceutical (NASDAQ:RARE) Shares Up 5.8% - Time to Buy?
Ultragenyx Pharmaceutical (NASDAQ:RARE) shares rose 5.8% to $15.25, though the stock remains below its 50-day and 200-day moving averages. The company recently beat quarterly expectations, reporting a smaller loss and higher revenue than anticipated. Despite mixed analyst sentiment with an average "Hold" rating and a consensus price target of $34.58, institutional investors hold a significant majority of the stock.
INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RARE
Pomerantz LLP is investigating potential securities fraud claims against Ultragenyx Pharmaceutical Inc. (RARE) on behalf of investors. This investigation follows a significant 44.03% drop in Ultragenyx's stock price after the company announced that its Phase 3 Aspire study for apazunersen in Angelman syndrome failed to meet its primary and key secondary endpoints. Investors are advised to contact the law firm for more information regarding joining the class action.
Milestone Sanfilippo nod helps Ultragenyx regroup after tough Angelman defeat
Ultragenyx recently secured FDA approval for Fayuvi, a treatment for Sanfilippo syndrome type A, and Genglycos, its first gene therapy, providing a boost after two significant asset failures. CEO Emil Kakkis highlighted the personal significance of the Sanfilippo approval, a disease class he has worked on since the start of his career. Despite the regulatory successes, the company faces challenges, including a Phase 3 failure in Angelman syndrome, leading to planned expense reductions, though they maintain hope for their osteogenesis imperfecta program.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Stock Has Consensus Price Target of $34.58 According to Analysts
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) has received a consensus "Hold" rating from analysts, with an average 12-month price target of $34.58. The company recently surpassed quarterly earnings expectations, reporting an adjusted loss of $0.90 per share against an anticipated $1.22 loss, and revenues of $214 million, a 28.5% increase year-over-year. Despite mixed recent analyst actions, institutional investors hold a significant 97.67% of the company's shares.
Ultragenyx Gets A ‘Much-Needed Win,’ Says Cantor – Sees $400M Opportunity After FDA Approves Neurodegenerative Disease Therapy
Ultragenyx received a "much-needed win" with FDA approval for Fayuvi, its neurodegenerative disease therapy for Sanfilippo syndrome Type A, leading to price target hikes from Cantor Fitzgerald and Citi. This approval also granted Ultragenyx a Priority Review Voucher, which Cantor estimates could generate $400 million if monetized, providing non-dilutive capital after a recent setback in another program. The stock saw a positive reaction, with analysts projecting significant upside and retail sentiment turning extremely bullish.
H.C. Wainwright reiterates Ultragenyx stock rating on FDA approval
H.C. Wainwright has reiterated a Buy rating and a $25.00 price target for Ultragenyx Pharma following the FDA's approval of its gene therapy, FAYUVI, for Sanfilippo syndrome Type A. This approval marks a significant milestone as it's the first FDA-approved treatment for this rare and fatal disease, and the second gene therapy approval for Ultragenyx. Despite this success, Ultragenyx faces challenges as it remains unprofitable and is rapidly burning through cash, according to InvestingPro Tips, although its stock is considered undervalued.
A Hedge Fund Rolls the Dice on Ultragenyx
Shares of Ultragenyx Pharmaceutical Inc. rose significantly after the FDA approved Fayuvi, its gene therapy for Sanfilippo syndrome Type A. Investment firm Sofinnova Investments, a large shareholder, had filed a Freedom of Information Act (FOIA) request with the FDA about Ultragenyx’s manufacturing compliance ahead of the approval decision. This action highlights how investment professionals use FOIA requests as a strategic tool to assess regulatory risks and market uncertainties surrounding drug approvals.
Novo’s turnaround plan, weight loss wins, Ultragenyx’s Sanfilippo approval, IPO blitz
This article covers Novo Nordisk's strategy to reach $23 billion in revenue by 2035, including workforce reductions and increased manufacturing of Wegovy, despite initial investor skepticism. It also highlights positive weight loss drug developments from Viking Therapeutics and Roche, and Ultragenyx's FDA approval for a Sanfilippo syndrome gene therapy. The piece concludes by noting the continued surge in biotech IPOs.
Ultragenyx Pharmaceutical Inc. (RARE) Stock Price, News, Quote & History
Ultragenyx Pharmaceutical Inc. (RARE) stock closed at $14.50, down 1.83% at the end of the trading day. The company, focused on rare and ultra-rare genetic diseases, has a market cap of $1.43 billion and its stock has seen significant declines over the past year. Analysts have an average 1-year target estimate of $28.05 for RARE.
Ultragenyx Pharmaceutical Inc. (RARE) Investigation: Bronstein, Gewirtz & Grossman, LLC Encourages Investors to Contact the Firm to Learn More About the Investigation
Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) following a significant stock price drop. The investigation stems from the company's announcement that its Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome failed to meet its primary and key secondary endpoints. Investors who purchased Ultragenyx securities are encouraged to contact the firm to assist with the investigation.
Key facts: Ultragenyx Pharmaceutical, Inc. FAYUVI Approved; FDA Decision Sept 19, 2026
Ultragenyx (RARE) has received full FDA approval for its FAYUVI (UX111) gene therapy for pediatric MPS IIIA, a one-time IV AAV9 treatment priced at $3.95 million. This approval follows a resubmission after a 2025 FDA rejection due to manufacturing issues. The decision, expected by September 19, 2026, will enable rapid deployment of the therapy to treatment centers.
How Investors Are Reacting To Ultragenyx Pharmaceutical (RARE) First-In-Disease Gene Therapy Approval For FAYUVI
Ultragenyx Pharmaceutical received full FDA approval for FAYUVI (UX111), a first-in-disease gene therapy for pediatric Sanfilippo syndrome Type A, alongside a Priority Review Voucher. This approval, coupled with another recent gene therapy approval for GENGLYCOS, expands Ultragenyx's commercial gene therapy portfolio. While these approvals improve the near-term outlook, investors should consider the company's high cash burn and potential for dilution, as the speed of adoption and reimbursement will be crucial for reaching profitability goals.
Ultragenyx (RARE) Surges 12.6%: Is This an Indication of Further Gains?
Ultragenyx (RARE) shares rose 12.6% after the FDA granted full approval to Fayuvi (rebisufligene etisparvovec-hopf) for treating pediatric MPS IIIA, marking their second gene therapy and sixth FDA approval. The company is expected to report a reduced quarterly loss and increased revenues, with positive earnings estimate revisions suggesting potential for continued stock appreciation.
Ultragenyx's $4 million gene therapy wins FDA approval for a rare brain and nervous system disorder
Ultragenyx has received FDA approval for its $4 million gene therapy designed to treat a rare brain and nervous system disorder. The one-time infusion therapy aims to correct the genetic cause of Sanfilippo syndrome, a condition affecting approximately one in every 70,000 births. This approval marks a significant milestone for the company in addressing rare genetic diseases.
FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder
The FDA has approved Ultragenyx's gene therapy Fayuvi for mucopolysaccharidosis type IIIA (MPS IIIA), a rare neurodegenerative disorder, marking the first treatment for the underlying disease. This approval follows a previous rejection due to manufacturing issues and provides a significant boost for Ultragenyx despite a recent late-stage clinical setback. Fayuvi has shown positive cognitive outcomes in young children with MPS IIIA, offering a new therapeutic option for affected families.
Ultragenyx sets $3.95 million price for rare disease gene therapy
Ultragenyx Pharmaceutical has priced its new gene therapy, Fayuvi, at $3.95 million for the treatment of Sanfilippo syndrome Type A, a rare and fatal childhood neurological disease. The U.S. FDA approved the one-time treatment, which aims to clear toxic buildup in the brain and nervous system, following trials where treated children maintained or improved cognitive function. J.P. Morgan analysts project peak worldwide sales for Fayuvi to reach $200 million to $250 million.
Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Pharmaceutical Inc. announced that the FDA has granted standard full approval for FAYUVI™ (rebisufligene etisparvovec-hopf) for pediatric patients with Sanfilippo syndrome Type A. This marks the first-ever FDA-approved treatment for this fatal neurodegenerative disease and Ultragenyx's second gene therapy approval. The company will support patient access through its UltraCare® program, with commercial product expected to be available within 30-60 days.
Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Pharmaceutical Inc. has received FDA approval for FAYUVI™ (rebisufligene etisparvovec-hopf), the first-ever gene therapy for Sanfilippo syndrome Type A (MPS IIIA). This approval marks a significant milestone for the devastating neurodegenerative disease, offering a potential treatment option where none existed before. Ultragenyx plans to make FAYUVI available through Qualified Treatment Centers, supported by its UltraCare® program to assist families with access.
RARE Stock Hits Fresh 52-Week Low As Sanfilippo Drug Ruling Nears — Failed Angelman Syndrome Trial Weighs On Shares
Ultragenyx Pharmaceutical (RARE) stock hit a new 52-week low as investors await an FDA decision on UX111 for Sanfilippo syndrome type A. The stock's recent decline is also heavily influenced by the earlier failure of its Angelman syndrome drug trial, leading to significant market value loss. Despite these setbacks, the company recently secured FDA approval for Genglycos and aims for profitability by 2027.
Bronstein, Gewirtz & Grossman, LLC Is Investigating Ultragenyx Pharmaceutical Inc. (RARE) And Encourages Investors to Connect
Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of Ultragenyx Pharmaceutical Inc. (RARE) following a significant stock price drop. The investigation stems from Ultragenyx's announcement that its Phase 3 Aspire study for apazunersen did not meet its primary or key secondary endpoints, causing the stock to fall 44.03%. The law firm encourages affected investors to connect with them for a potential class action lawsuit, noting that they operate on a contingency fee basis.
Ultragenyx stock hits 52-week low at 13.78 USD By Investing.com
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) stock has fallen to a 52-week low of $13.78 USD, reflecting challenges in the biotech sector. Despite this, analysts maintain a "Buy" rating with price targets suggesting significant upside potential. Recent developments, including a failed Phase 3 study for GTX-102, have led several investment firms to lower their price targets for the company.
Ultragenyx Pharmaceutical (NASDAQ:RARE) Reaches New 12-Month Low - Time to Sell?
Ultragenyx Pharmaceutical (NASDAQ: RARE) recently hit a new 52-week low of $13.80, trading below its moving averages, despite beating quarterly earnings and revenue expectations. Several analysts have downgraded the stock or cut price targets, although the consensus remains a "Moderate Buy" with a $36.63 target. The biopharmaceutical company, which focuses on rare diseases, remains deeply unprofitable and is expected to post a full-year loss.
Bronstein, Gewirtz & Grossman, LLC Encourages Ultragenyx Pharmaceutical Inc. (RARE) Investors to Inquire about Securities Investigation
Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) investors. This follows a significant 44.03% stock price drop after the company announced its Phase 3 Aspire study for apazunersen in Angelman syndrome failed to meet its primary and key secondary endpoints. The firm is encouraging investors to inquire about assisting the securities investigation.
Squarepoint Ops LLC Sells 106,171 Shares of Ultragenyx Pharmaceutical Inc. $RARE
Squarepoint Ops LLC has reduced its stake in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) by 23.9%, selling over 106,000 shares but still retaining a significant holding. Despite recent analyst downgrades and price target reductions, the company maintains a "Moderate Buy" consensus rating. Ultragenyx Pharmaceutical recently reported better-than-expected quarterly results with a 28.5% revenue increase year-over-year.
Ultragenyx SVP Theodore Huizenga sells $2,252 in shares
Theodore Alan Huizenga, SVP and Chief Accounting Officer at Ultragenyx Pharmaceutical Inc., sold 161 shares of company stock for $2,252 on September 3, 2026, to cover tax withholdings. This transaction occurred as the stock trades near its 52-week low, following a significant decline. The sale comes amidst news that Ultragenyx's Phase 3 study for apazunersen failed to meet its primary and secondary endpoints, leading several financial firms to lower their price targets and ratings for the company.
Why Ultragenyx Pharmaceutical (RARE) Is Down 40.3% After Angelman Trial Failure And Cost-Cut Plan
Ultragenyx Pharmaceutical (RARE) shares dropped 40.3% after its Phase 3 Aspire trial for Angelman syndrome failed to meet primary endpoints, leading to a program review and planned cost reductions. This setback overshadowed the positive news of FDA approval for its GENGLYCOS gene therapy and its qualification for a rare pediatric disease priority review voucher. Investors are now focused on the company's ability to scale approved products and implement cost-cutting measures effectively.
Ultragenyx vows 'significant' cuts after experimental rare-disease drug flunks late-stage clinical trial
Ultragenyx announced "significant" cuts following the failure of its experimental Angelman syndrome drug in a late-stage clinical trial. The news caused the company's stock to drop by 44%. CEO Emil Kakkis expressed disappointment for the patient community, which had invested heavily in the research.
Ultragenyx to weigh ‘significant’ cost cuts as Angelman drug fails key study
Ultragenyx plans to implement "significant expense reductions" after its experimental Angelman syndrome treatment, GTX-102, failed to meet primary and secondary endpoints in a Phase 3 study. This setback, which caused Ultragenyx's stock to drop by almost half, forces the company to focus on its approved products and achieving profitability by 2027. The failure also raises questions for other companies developing similar Angelman therapies.
Wellington Management Group LLP Reduces Stock Position in Ultragenyx Pharmaceutical Inc. $RARE
Wellington Management Group LLP significantly reduced its stake in Ultragenyx Pharmaceutical Inc. by 52.6% in the second quarter, selling 1.39 million shares. Despite this, institutional investors still hold 97.67% of the company, and analysts maintain a "Moderate Buy" rating with an average price target of $37.53. The stock, which has fallen 44% and is near its 52-week low, faces pressure due to a failed Angelman syndrome trial, though the company exceeded quarterly earnings and revenue expectations.
Why Did RARE Stock Slump 46% After-Hours?
Ultragenyx Pharmaceutical (RARE) shares plunged 46% after its experimental Angelman syndrome drug failed a late-stage study. This is the second late-stage failure for the company, following a brittle-bone drug failure in late 2025. Despite the setback, retail investors on Stocktwits showed increased bullish sentiment, anticipating a decision on another gene therapy program.
Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
Ultragenyx Pharmaceutical Inc. announced that its Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome did not meet its primary or key secondary endpoints. The company expressed disappointment and plans to evaluate the program's future, while also assessing operational changes to implement expense reductions. Despite this setback, Ultragenyx remains focused on its growing commercial business, including recently approved and pipeline products, to achieve profitability by 2027.
Bank of New York Mellon Corp Makes New Investment in Ultragenyx Pharmaceutical Inc. $RARE
Bank of New York Mellon Corp has made a new investment in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), purchasing 470,697 shares valued at approximately $15.7 million. This investment comes as institutional investors collectively own 97.67% of the company's stock, and analysts maintain a "Moderate Buy" consensus rating despite a wide range of price targets. Ultragenyx Pharmaceutical recently reported better-than-expected quarterly results, with revenues up 28.5% year-over-year, though the company remains unprofitable.
Corient Private Wealth LP Acquires New Shares in Ultragenyx Pharmaceutical Inc. $RARE
Corient Private Wealth LP has acquired 139,094 shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) valued at approximately $4.64 million, making them a new significant institutional investor. Other hedge funds have also increased their holdings in Ultragenyx, with institutional investors and hedge funds collectively owning 97.67% of the company's stock. The biopharmaceutical company recently reported strong quarterly revenue and better-than-expected EPS, leading analysts to maintain a "Moderate Buy" rating with a consensus price target significantly above the current trading price.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Receives Average Rating of "Moderate Buy" from Analysts
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) has received an average "Moderate Buy" rating from nineteen analysts, with an average 12-month price target of $59.44, significantly above its current stock price of $25.21. The company's recent quarterly results exceeded expectations, reporting a narrower loss and higher revenue than anticipated. Institutional ownership is substantial at 97.67%, with major investors like BlackRock increasing their stakes.
Man Group plc Makes New $6.51 Million Investment in Ultragenyx Pharmaceutical Inc. $RARE
Man Group plc has invested $6.51 million in Ultragenyx Pharmaceutical Inc. by acquiring 195,110 shares, representing 0.20% of the company. This comes as Ultragenyx reported better-than-expected Q2 results, with a narrower loss per share and revenue growth of 28.5%. Despite positive analyst sentiment leading to a "Moderate Buy" rating, company insiders have recently sold shares.
The Manufacturers Life Insurance Company Invests $12.74 Million in Ultragenyx Pharmaceutical Inc. $RARE
The Manufacturers Life Insurance Company has acquired a new stake of 381,420 shares in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), valued at approximately $12.74 million. This investment represents about 0.39% of Ultragenyx, with institutional investors and hedge funds collectively owning 97.67% of the company's stock. Analysts maintain a "Moderate Buy" rating with an average price target of $59.44, and the company recently exceeded revenue expectations, reporting $214 million for the quarter.
Palo Alto Investors LP Takes $19.92 Billion Position in Ultragenyx Pharmaceutical Inc. $RARE
Palo Alto Investors LP has invested $19.92 million in Ultragenyx Pharmaceutical Inc. ($RARE), acquiring 596,495 shares, making it their 12th largest position and representing 0.61% ownership of the biopharmaceutical company. Ultragenyx Pharmaceutical recently reported better-than-expected quarterly results, and analysts maintain a "Moderate Buy" consensus rating with an average price target of $59.44. Despite insider sales totaling $207,455 over the past three months, institutional investors collectively hold 97.67% of the company's stock.
Ultragenyx Delivers First Gene Therapy FDA Approval for GSDIa
Ultragenyx Pharmaceutical Inc. (RARE:NASDAQ) has received FDA accelerated approval for GENGLYCOS, its first gene therapy, targeting Glycogen Storage Disease Type Ia (GSDIa) in patients aged eight and older. This approval marks a significant shift from symptom management to addressing the underlying genetic cause of the disease, which affects 1,500 to 2,500 patients in the U.S. The approval, based on Phase 3 data showing reduced cornstarch dependency, has led to increased price targets from analysts, with Ultragenyx expecting a commercial launch within 30-60 days at a wholesale cost of $2.7 million.
BlackRock Inc. Makes New Investment in Ultragenyx Pharmaceutical Inc. $RARE
BlackRock Inc. has acquired a new position in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) during the second quarter, purchasing over 10.65 million shares valued at approximately $355.7 million, representing about 10.82% of the company. Other institutional investors have also adjusted their holdings, while company insiders have sold a total of 8,552 shares worth $207,455. Analysts have updated price targets for RARE, with the stock currently holding a "Moderate Buy" rating and an average price target of $59.22.
Algert Global LLC Reduces Position in Ultragenyx Pharmaceutical Inc. $RARE
Algert Global LLC decreased its stake in Ultragenyx Pharmaceutical Inc. by 9.0% in the second quarter, now owning 389,964 shares worth $13.02 million. Other institutional investors, such as Parallel Advisors LLC and Leonteq Securities AG, increased their holdings. Insider activity also saw directors and executives selling shares, though insiders still collectively own 5.20% of the company's stock.
Ultragenyx Pharmaceutical Grants Inducement Restricted Stock Units to 23 New Employees
Ultragenyx Pharmaceutical has granted 45,984 restricted stock units to 23 new non-executive employees as an employment inducement, in accordance with Nasdaq regulations. These units will vest over four years, with 25% vesting annually, provided the employees remain continuously employed. This move aims to attract and retain talent in the biopharmaceutical sector, though it could lead to concerns about equity dilution among existing shareholders.
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Ultragenyx Pharmaceutical Inc. granted 45,984 restricted stock units to 23 newly hired non-executive officers. This inducement award, approved by the compensation committee, vests over four years and is in accordance with Nasdaq Listing Rule 5635(c)(4). Ultragenyx is a biopharmaceutical company focused on rare and ultra-rare genetic diseases.
Ultragenyx gives 23 newly hired officers stock awards over four years
Ultragenyx (NASDAQ: RARE) has granted 45,984 restricted stock units (RSUs) to 23 newly hired non-executive officers. These awards, valued as an inducement for new employment, will vest over four years, with 25% vesting annually, subject to continued employment. The grant was approved under the Ultragenyx Employment Inducement Plan and complies with Nasdaq Listing Rule 5635(c)(4).