Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported granting 17,567 restricted stock units to 11 newly hired non-executive officers. These awards were made under the company's Employment Inducement Plan, effective May 16, 2026, as an inducement for employment in accordance with Nasdaq Listing Rule 5635(c)(4). The restricted stock units will vest over four years, with 25% vesting annually, contingent on continuous employment.
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Ultragenyx Pharmaceutical Inc. announced the grant of 17,567 restricted stock units to 11 new non-executive officers. These awards, approved by the compensation committee, serve as an inducement for their employment in accordance with Nasdaq Listing Rule 5635(c)(4). The restricted stock units will vest over four years, with 25% vesting annually, provided continuous employment.
Fideuram Intesa Sanpaolo Private Banking S.P.A. Invests $1.41 Million in Ultragenyx Pharmaceutical Inc. $RARE
Fideuram Intesa Sanpaolo Private Banking S.P.A. has initiated a new position in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) by purchasing 61,329 shares valued at approximately $1.41 million. Other institutional investors have also increased their stakes in the biopharmaceutical company, which has a "Moderate Buy" consensus rating from analysts. Despite some recent price target cuts and a quarterly earnings miss, institutional interest remains very high, with insiders also actively trading shares.
RARE Maintained by Cantor Fitzgerald -- Price Target Raised to $96
Cantor Fitzgerald maintained an "Overweight" rating for Ultragenyx Pharmaceutical (RARE) and raised its price target from $84.00 to $96.00. Despite this positive analyst outlook, GuruFocus indicates RARE is currently undervalued by about 49.7% but also flags it as a "Possible Value Trap," with a GF Score of 61/100 and recent insider selling of $2.9 million. While the company shows strong growth potential, its financial strength and profitability rank low.
Cantor Fitzgerald Issues Positive Forecast for Ultragenyx Pharmaceutical (NASDAQ:RARE) Stock Price
Cantor Fitzgerald has raised its price target for Ultragenyx Pharmaceutical (NASDAQ:RARE) from $84 to $96, maintaining an "overweight" rating despite the company missing recent quarterly earnings and revenue estimates. This new target suggests a significant upside of over 300% from its current trading price. The stock was trading near its 50-day average, with insiders selling shares, while institutional investors hold a substantial stake.
Cantor Fitzgerald raises Ultragenyx stock price target on therapy outlook By Investing.com
Cantor Fitzgerald has increased its price target for Ultragenyx Pharma (NASDAQ: RARE) to $96 from $84, maintaining an Overweight rating. This adjustment follows increased confidence in GTX-102, a treatment for Angelman syndrome, with its probability of success raised to 60%. Despite recent financial misses for Q1 2026, the company reaffirmed its full-year revenue guidance and recently elected three Class I directors.
Ultragenyx Pharmaceutical’s SWOT analysis: stock faces pivotal trial results
Ultragenyx Pharmaceutical (NASDAQ: RARE) is at a crucial point as it awaits pivotal clinical trial results for setrusumab, its therapeutic candidate for osteogenesis imperfecta. The company recently secured a royalty transaction to improve its financial flexibility, addressing concerns about sustained negative earnings. While the ORBIT trial for setrusumab shows high promise, concerns exist regarding the statistical power of the COSMIC study, which could impact regulatory approval and the stock's valuation.
Ultragenyx (RARE) director Narachi receives RSU and option awards
Ultragenyx Pharmaceutical Inc. director Michael Narachi was granted 7,751 Restricted Stock Units (RSUs) and stock options for 14,058 shares. The RSUs and options vest on the earlier of the company's next Annual Meeting or May 14, 2027, with the options having an exercise price of $25.80 and expiring on May 14, 2036. Following these transactions, Narachi directly holds 40,441 common stock shares.
Ultragenyx Pharmaceutical stock (US74930A1043): Shareholders back incentive plan
Ultragenyx Pharmaceutical shareholders recently approved the company's incentive plan and elected directors in May 2026. This significant governance update reinforces strategic continuity and management alignment for the rare-disease biotech, which is keenly watched by US investors due to its Nasdaq listing and focus on high-value specialty drugs. While not directly impacting earnings or trial data, the clean shareholder vote removes uncertainty and allows attention to revert to execution, clinical development, and product revenue trends.
RARE - Ultragenyx Pharmaceutical Inc. stock news, filings & ratings
Ultragenyx Pharmaceutical Inc. (RARE) focuses on novel products for rare genetic diseases, with key products like Crysvita, Mepsevii, and Dojolvi. The company recently reported inducement grants of restricted stock units to new non-executive officers and several insider transactions involving grants and sales of shares. Ultragenyx also released its Q1 2026 financial results, reaffirming its 2026 guidance and outlining progress on its clinical pipeline.
Ultragenyx director Suliman Shehnaaz sells $144,188 in stock
Ultragenyx director Suliman Shehnaaz sold 5,740 shares of the company's common stock for $144,188 on May 18, 2026. This transaction occurred as the stock trades at $24.18, down from a previous close of $25. Separately, Shehnaaz also received 7,751 restricted stock units and 14,058 stock options on May 14, 2026, which are set to vest by May 14, 2027.
[Form 4] Ultragenyx Pharmaceutical Inc. Insider Trading Activity
This article details an insider trading activity at Ultragenyx Pharmaceutical Inc. (RARE), specifically a Form 4 filing by director Daniel G. Welch. Welch reported receiving 7,751 shares of common stock as restricted stock units and a stock option for 14,058 shares, with both vesting fully by the company's next Annual Meeting of Stockholders or May 14, 2027. The filing indicates a neutral impact and sentiment regarding these transactions.
[144] Ultragenyx Pharmaceutical Inc. SEC Filing
This article details an SEC Form 144 filing by Ultragenyx Pharmaceutical Inc. (RARE) on May 18, 2026. The filing indicates a proposed sale of 5,740 shares of common stock with an aggregate market value of $144,188.80 through Morgan Stanley Smith Barney LLC. The securities were acquired on May 14, 2026, as restricted stock from the issuer.
Ultragenyx Shareholders Approve Incentive Plan, Elect Directors
Ultragenyx Pharmaceutical shareholders approved an updated 2023 equity incentive plan, elected directors, and ratified auditors at their Annual Meeting on May 14, 2026. This approval supports the company's long-term compensation framework and aligns management with shareholder interests in the competitive rare-disease biotech market. Despite a "Neutral" rating from TipRanks’ AI Analyst Spark due to financial performance concerns, the company has positive technical momentum, reaffirmed 2026 guidance, and upcoming regulatory catalysts.
Ultragenyx shareholders approve director elections and incentive plan at annual meeting
Ultragenyx Pharmaceutical Inc. shareholders approved the election of three Class I directors and the Third Amended and Restated 2023 Incentive Plan at their annual meeting. They also ratified Ernst & Young LLP as the independent accounting firm and approved executive compensation in an advisory vote. Despite these approvals, the company reported a net loss and lower-than-expected revenue in Q1 2026, though it maintained full-year revenue guidance.
Chancery Court Tosses Stockholder Complaint Against Ultragenyx Pharma
The Delaware Chancery Court dismissed a stockholder lawsuit against Ultragenyx Pharmaceutical, which alleged excessive compensation for non-employee directors. The court ruled that a pre-suit demand letter sent by the plaintiff, John Solak, and subsequently rejected by the board, negated his legal options. Vice Chancellor Kathleen S. McCormick determined that the letter functioned as a pre-suit demand under court rules, despite Solak's disclaimer.
UBS Group AG Raises Stock Position in Ultragenyx Pharmaceutical Inc. $RARE
UBS Group AG significantly increased its holdings in Ultragenyx Pharmaceutical Inc. by 22.3% in the fourth quarter, bringing its total to 668,811 shares valued at $15.4 million. This move comes despite Ultragenyx Pharmaceutical reporting weaker-than-expected quarterly earnings and revenue, with the company missing analyst estimates. While analyst sentiment is mixed, the consensus remains a "Moderate Buy" with an average price target of $60.41, though near-term EPS estimates have been trimmed.
Investigation Alert: Long-Term Investors in Ultragenyx
The Shareholders Foundation announced an investigation into potential breaches of fiduciary duties by directors and officers of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) on behalf of current long-term investors. This investigation follows a lawsuit alleging securities law violations after significant stock declines, which occurred subsequent to the failure of two Phase III studies (Orbit and Cosmic) to meet primary endpoints. Long-term investors in Ultragenyx are encouraged to contact the Shareholders Foundation for options.
RARE: Upcoming gene therapy launches and Angelman data drive growth toward 2027 profitability
Ultragenyx Pharmaceutical Inc. (RARE) has reaffirmed its revenue guidance and aims for profitability by 2027, driven by growth in existing and new products. Key factors include upcoming Phase III data for Angelman syndrome and the launch of two gene therapies, both supported by strong clinical and commercial readiness. This information is based on an audio transcript from the Bank of America Global Healthcare Conference 2026.
State Street (RARE) reports 5.1% ownership, 5,021,441 shares
State Street Corporation has disclosed a passive beneficial ownership of 5,021,441 shares, representing 5.1% of Ultragenyx Pharmaceutical Inc. common stock, as of March 31, 2026. This Schedule 13G filing indicates that State Street, through its various advisory and trust entities, holds shared voting power over 4,769,004 shares and shared dispositive power over all 5,021,441 shares. The report, signed on May 12, 2026, suggests an investment-only intent without plans to influence control.
State of New Jersey Common Pension Fund D Sells 42,451 Shares of Ultragenyx Pharmaceutical Inc. $RARE
The State of New Jersey Common Pension Fund D significantly reduced its stake in Ultragenyx Pharmaceutical Inc. by selling 42,451 shares, decreasing its holdings by 58.9%. This comes amidst mixed analyst sentiment, with several firms trimming price targets and Goldman Sachs downgrading the stock to neutral. The company also reported weaker-than-expected quarterly results and saw notable insider selling.
HC Wainwright Brokers Decrease Earnings Estimates for RARE
HC Wainwright has lowered its Q1 2027 earnings estimate for Ultragenyx Pharmaceutical (NASDAQ:RARE) to $0.27 per share, while maintaining a Buy rating and a $50 price target. This follows Ultragenyx reporting a larger-than-expected Q1 loss of $1.84 per share, missing both EPS and revenue estimates. Analyst sentiment for Ultragenyx is mixed but generally positive, with a "Moderate Buy" consensus and an average price target of $60.41, despite some recent downgrades and target reductions from other firms.
Ultragenyx Pharmaceutical (RARE) Valuation Check After Recent Share Price Momentum And Long Term Pressure
Ultragenyx Pharmaceutical (RARE) has seen recent positive share price momentum but long-term holders remain under pressure, with the stock trading at US$26.13 against a narrative fair value of $52.45. The company is considered undervalued due to its rare disease and gene therapy pipeline, which benefits from increasing diagnoses and regulatory support for orphan drugs. However, potential regulatory setbacks or ongoing net losses could impact this positive outlook.
Number of shareholders of Ultragenyx Pharmaceutical, Inc. – FWB:UP0
This article provides financial information for Ultragenyx Pharmaceutical, Inc. (FWB:UP0) on the Frankfurt Stock Exchange. It focuses on the number of shareholders, displaying period values and changes. The content is primarily a data presentation, indicating the company's financial overview.
GTX-102 data show continued developmental gains in Angelman
Long-term data from a clinical study on GTX-102 (apazunersen) indicate sustained improvements and developmental gains in children with Angelman syndrome. Developed by Ultragenyx Pharmaceutical, GTX-102 is an RNA-based therapy designed to reactivate the paternal UBE3A gene copy. Two ongoing trials, Phase 3 Aspire and Phase 2/3 Aurora, are further evaluating the therapy's safety and efficacy, with Aspire results expected later this year.
Earnings call transcript: Ultragenyx Q1 2026 sees revenue miss, stock rises
Ultragenyx Pharmaceutical Inc. reported a net loss of $1.84 per share, missing analyst expectations, and revenue of $136 million, falling short of the forecasted $160.69 million for Q1 2026. Despite these misses, the company's stock rose 4.51% in after-hours trading, possibly due to reaffirmed full-year revenue guidance of $730-$760 million. The earnings call detailed performance of key products like CRYSVITA and DOJOLVI, ongoing clinical trials for Angelman syndrome treatment GTX-102, and upcoming gene therapy launches for DTX401 and UX111, with a continued focus on achieving profitability by 2027.
Number of shareholders of Ultragenyx Pharmaceutical Inc. – NASDAQ:RARE
This article provides current information on the number of shareholders for Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE). It appears to be a stub or data placeholder, indicating the period and value for shareholder figures would be displayed here. The page also includes various links related to financial data, company information, and community features on TradingView.
Ultragenyx Pharmaceutical (NASDAQ:RARE) Cut to "Strong Sell" at Wall Street Zen
Wall Street Zen has downgraded Ultragenyx Pharmaceutical (NASDAQ:RARE) from "hold" to "strong sell" following a weak earnings report where the company missed EPS and revenue expectations. Despite this downgrade, the overall analyst consensus remains a "Moderate Buy" with an average price target of $60.41, significantly higher than its opening price of $26.12. The article also notes recent insider selling and institutional investment activity for Ultragenyx, a biopharmaceutical company focused on rare genetic disorders.
Vanguard Group Inc. Buys 131,087 Shares of Ultragenyx Pharmaceutical Inc. $RARE
Vanguard Group Inc. increased its stake in Ultragenyx Pharmaceutical Inc. by 1.3% in the fourth quarter, purchasing an additional 131,087 shares and bringing its total holdings to 10.26 million shares valued at $236.1 million. The biopharmaceutical company recently reported a wider-than-expected quarterly loss of ($1.84) EPS and revenue of $136 million, missing analyst estimates. Despite this, Wall Street analysts maintain a "Moderate Buy" rating with an average price target of $60.41, although Goldman Sachs downgraded its rating to "neutral" with a $25.00 price target.
Ultragenyx Pharmaceutical (RARE) Is Up 5.8% After Reaffirming 2026 Outlook Despite Wider Losses – What's Changed
Ultragenyx Pharmaceutical (RARE) saw its stock rise by 5.8% after reaffirming its 2026 revenue guidance of US$730 million to US$760 million, despite reporting wider losses in Q1 2026. The company remains focused on achieving profitability by 2027, driven by potential 2026 approvals for two gene therapies and key late-stage data. This outlook is critical for its investment narrative, which relies on its rare disease and gene therapy pipeline transitioning the company from losses to sustainable profitability.
Ultragenyx Pharmaceutical (RARE) Is Up 5.8% After Reaffirming 2026 Outlook Despite Wider Losses – What's Changed
Ultragenyx Pharmaceutical reported wider Q1 2026 losses but reaffirmed its 2026 revenue guidance of US$730-$760 million, maintaining its path to profitability by 2027. The company's investment narrative hinges on its rare disease and gene therapy pipeline, with upcoming approvals and data readouts expected to drive significant revenue. Despite the quarterly miss, management's re-affirmation indicates confidence in clinical and regulatory milestones to achieve longer-term earnings goals.
Ultragenyx Pharmaceutical (RARE) Is Up 5.8% After Reaffirming 2026 Outlook Despite Wider Losses – What's Changed
Ultragenyx Pharmaceutical (RARE) saw its stock rise by 5.8% after reiterating its 2026 revenue guidance of US$730 million to US$760 million, despite reporting wider first-quarter losses. The company aims for profitability by 2027, backed by potential 2026 gene therapy approvals and key late-stage data for its Angelman syndrome program. This update focuses on the company's commitment to its profitability path, balancing recent financial misses with anticipated clinical and regulatory milestones.
RARE SEC Filings - Ultragenyx Pharm 10-K, 10-Q, 8-K Forms
This article provides an overview of Ultragenyx Pharm's (RARE) SEC filings, including annual reports (10-K), quarterly earnings (10-Q), material events (8-K), and insider trading forms. It highlights recent filings detailing stock awards and sales by company executives, FDA's Priority Review for DTX401 gene therapy, and the company's financial performance and strategic restructuring plans. StockTitan offers AI-powered summaries and real-time updates for these regulatory documents.
RARE Maintained by Wedbush -- Price Target Lowered to $26
Wedbush has maintained a Neutral rating for Ultragenyx Pharmaceutical Inc (RARE), lowering its price target from $27.00 to $26.00 due to a cautious outlook on the biotechnology sector. Despite being considered 43.7% undervalued by GuruFocus's GF Value at $46.83 compared to its current price of $26.34, the stock carries a "Possible Value Trap" warning. Insider activity shows significant sales of $2.8 million in the past three months, indicating mixed signals for potential investors.
Guggenheim Cuts Ultragenyx Pharmaceutical (NASDAQ:RARE) Price Target to $43.00
Guggenheim has reduced its price target for Ultragenyx Pharmaceutical (NASDAQ:RARE) from $52.00 to $43.00, while maintaining a "buy" rating, indicating a potential upside of 63.11%. This adjustment follows Ultragenyx missing its Q1 earnings and revenue estimates. Despite the cut, the stock holds an overall "Moderate Buy" rating from analysts with a consensus target price of $60.41.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q1 2026 Earnings Call Transcript
Ultragenyx Pharmaceutical Inc. discussed its Q1 2026 financial results, highlighting growing revenue projected to exceed $700 million and upcoming approvals for two gene therapies. The company also provided updates on its GTX-102 program for Angelman syndrome, noting positive long-term Phase 1/2 data and the progress of the Phase 3 Aspire study. Additionally, Ultragenyx reaffirmed its 2026 revenue guidance and discussed its path to profitability in 2027, including plans for launching two new products and potential PRV monetization.
Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference
Ultragenyx Pharmaceutical Inc. announced that its CFO, Howard Horn, and Chief of Staff/VP of Investor Relations, Joshua Higa, will participate in a fireside chat at Bank of America's 2026 Healthcare Conference. The event is scheduled for Tuesday, May 12, 2026, at 2:20 PM PT, and a webcast will be available on the company's website. Ultragenyx focuses on developing and commercializing therapies for serious rare and ultra-rare genetic diseases.
Rare disease families find roadmap to drug development at bootcamps
Rare disease families are increasingly taking the lead in driving research efforts to find treatments for their children, often due to the high cost and low return on investment for large pharmaceutical companies. Ultragenyx hosts a "Rare Bootcamp" twice a year, providing a roadmap for these families and patient advocates to navigate drug development. The program connects attendees with researchers, clinicians, and other advocates, offering over 20 sessions covering the full spectrum of drug development.
Ultragenyx Earnings Call: Growth, Gene Therapy and Risk
Ultragenyx Pharmaceutical's Q1 earnings call showcased strong commercial growth, particularly from Crysvita, and progress in its gene therapy pipeline with potential regulatory catalysts on the horizon. Despite substantial Q1 losses, the company reaffirmed its 2026 revenue guidance and aims for profitability by 2027 by leveraging new launches and Priority Review Voucher monetizations, alongside strict cost discipline. The future success of Ultragenyx hinges on timely approvals, successful voucher sales, and effective market uptake of its new therapies to mitigate current cash burn and achieve its profitability goals.
Ultragenyx (NASDAQ: RARE) CMO sells shares to cover RSU taxes
Ultragenyx Pharmaceutical Inc.'s EVP and Chief Medical Officer, Eric Crombez, sold 344 shares of common stock at $24.96 per share on May 5, 2026. This sale was made to cover tax withholdings related to the vesting of restricted stock units (RSUs). Following this transaction, Crombez still directly holds 111,189 shares of Ultragenyx common stock.
Earnings Flash (RARE) Ultragenyx Pharmaceutical Inc. Reports Q1 Revenue $136.0M, Vs. FactSet Est of $158.4M
Ultragenyx Pharmaceutical Inc. (RARE) reported its Q1 revenue at $136.0 million, significantly missing the FactSet estimate of $158.4 million. This earnings flash provides key financial data for the company's first quarter performance.
Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that its Chief Financial Officer, Howard Horn, and Chief of Staff and VP of Investor Relations, Joshua Higa, will participate in a fireside chat at Bank of America’s 2026 Healthcare Conference on May 12, 2026, at 2:20 PM PT. A live and archived webcast will be available on the company's website. Ultragenyx is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases.
Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that its CFO and EVP, Howard Horn, along with Chief of Staff and VP of investor relations, Joshua Higa, will participate in a fireside chat at Bank of America’s 2026 Healthcare Conference on May 12, 2026, at 2:20 PM PT. A webcast of the panel will be available on the company's website. Ultragenyx is a biopharmaceutical company focused on developing and commercializing novel therapies for rare and ultra-rare genetic diseases, with a strategy centered on efficient drug development.
Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference
Ultragenyx Pharmaceutical Inc. will participate in Bank of America's 2026 Healthcare Conference on May 12, 2026. Howard Horn, CFO, and Joshua Higa, Chief of Staff and VP of IR, will represent the company in a fireside chat. A webcast of the panel will be available on Ultragenyx's investor relations website.
Ultragenyx Pharmaceutical Inc. Q1 2026 Earnings Call Summary
Ultragenyx Pharmaceutical Inc. presented its Q1 2026 earnings, highlighting a disciplined country-by-country execution strategy across 35+ countries and preparations for two new gene therapy launches. The company anticipates achieving profitability in 2027 through double-digit revenue growth from its base business and monetization of Priority Review Vouchers, aiming for 2026 revenues of $730 million to $760 million. Key updates include the Phase 3 Aspire study for Angelman syndrome on track for a second-half 2026 readout and strategic expense management.
Universal Beteiligungs und Servicegesellschaft mbH Has $323,000 Position in Ultragenyx Pharmaceutical Inc. $RARE
Universal Beteiligungs und Servicegesellschaft mbH significantly reduced its stake in Ultragenyx Pharmaceutical (NASDAQ:RARE) by 84.6% in the fourth quarter, now holding 14,094 shares valued at $323,000. This comes as Ultragenyx reported disappointing Q1 results, missing EPS and revenue estimates, although product sales and full-year revenue guidance offer some commercial support. Market sentiment for Ultragenyx is mixed, with high institutional ownership from some and notable insider selling and analyst downgrades contributing to near-term volatility.
Ultragenyx Pharmaceutical (NASDAQ:RARE) Posts Quarterly Earnings Results, Misses Estimates By $0.34 EPS
Ultragenyx Pharmaceutical (NASDAQ:RARE) reported Q1 earnings with a loss of $1.84 per share, missing consensus estimates by $0.34. Despite the miss, the biopharmaceutical company reaffirmed its full-year 2026 revenue guidance between $730–$760 million and remains on track for profitability in 2027. The company highlighted solid commercial momentum for its existing rare-disease products and upcoming regulatory catalysts for two gene-therapy BLAs.
Ultragenyx Reports First Quarter 2026 Financial Results and Corporate Update
Ultragenyx reported first-quarter 2026 revenue of $136 million, reaffirming its full-year guidance of $730 million to $760 million. The company is advancing several clinical programs, including GTX-102 for Angelman syndrome, with Phase 3 data expected in the second half of 2026, and has two Biologics License Applications (BLAs) under review by the FDA. Ultragenyx remains on track for profitability in 2027.
Two FDA decisions by September put Ultragenyx gene therapies in focus
Ultragenyx (NASDAQ: RARE) reported Q1 2026 revenue of $136 million and reaffirmed its full-year 2026 guidance of $730–$760 million, maintaining its path to profitability in 2027. The company's gene therapies, DTX401 and UX111, have upcoming FDA PDUFA dates in August and September 2026, respectively, alongside anticipated Phase 3 data for GTX-102 in the second half of 2026. Despite consistent revenue growth and reaffirmed guidance, Ultragenyx's stock has historically experienced negative price reactions following earnings reports, a trend that continued this quarter amid a larger net loss and substantial R&D spending.
Gene therapy reviews and Q1 2026 results at Ultragenyx (RARE)
Ultragenyx Pharmaceutical reported Q1 2026 revenue of $136 million, a slight decrease from the previous year, and a net loss of $185 million. Despite widening losses and high cash burn, the company reaffirmed its 2026 revenue guidance of $730–$760 million and aims for profitability in 2027. Key catalysts include two gene therapy BLAs under FDA Priority Review, DTX401 and UX111, with PDUFA dates in August and September 2026, respectively, and upcoming Phase 3 data for GTX-102 for Angelman syndrome.
