Ultragenyx Pharmaceutical Inc. (RARE) Stock Price & How to Invest

Ultragenyx Pharmaceutical (NASDAQ: RARE) develops and sells treatments for rare and ultra-rare genetic diseases, conditions with small patient populations and often no approved therapy. Its commercial base includes Crysvita for X-linked hypophosphatemia (its largest product), Dojolvi for long-chain fatty acid oxidation disorders, Evkeeza, and Mepsevii, which together produced about $673 million of revenue in 2025. The company spans multiple modalities, protein-replacement, small molecule, antisense, and gene therapy, and is preparing several late-stage programs for potential approval.

The investment picture is that of a growing but still loss-making specialty biotech. Revenue grew roughly 20% in 2025, yet the company posted a net loss of about $575 million as it funds heavy R&D. Management has reaffirmed 2026 revenue guidance of $730 million to $760 million and reiterated a goal of reaching profitability in 2027, contingent on commercial growth, expense discipline, and monetizing potential priority review vouchers. The stock therefore trades largely on pipeline catalysts and the credibility of that profitability timeline rather than on current earnings.

Ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk. The value concentrated in binary regulatory and clinical events (DTX401, UX111, and especially the GTX-102 Angelman readout) means a single negative outcome could sharply move the stock. Rare-disease revenue depends on small patient counts, payer reimbursement, and diagnosis rates, all of which can disappoint. The company faces well-capitalized competitors in gene therapy and orphan drugs, including BioMarin, Sarepta, and larger players. Finally, the 2027 profitability target is a management objective, not a guarantee, and could slip.

19 analysts publish price targets on RARE, averaging $53.37 against a $24.91 price as of August 2026, or +114.3%. The published targets run from $27.00 to $96.00, a wide spread, and the ratings split 17 buy, 2 hold, 0 sell. A price target is what an analyst published on a date, not a prediction, and sell-side ratings skew positive across the whole market.

Read the full RARE forecast and price target for the target table, the recent rating actions by firm, and how the consensus has shifted.

We give no verdict on Ultragenyx Pharmaceutical Inc.. Both cases are real, which is why the question is contested at all, so here is the strongest version of each.

The case for buying. Near-term gene therapy approvals. Two gene therapies are under FDA review with 2026 decision dates: DTX401 for glycogen storage disease type Ia and UX111 for Sanfilippo syndrome type A. The most optimistic published target, $96.00, assumes this works close to its best case.

The case against. Ultragenyx remains unprofitable, with a net loss of about $575 million in 2025 and roughly $737 million of cash as of December 2025, so continued spending or delayed approvals could pressure the balance sheet and raise dilution or financing risk. The most pessimistic target, $27.00, is roughly what RARE is worth if this bites instead.

Read the full bull and bear case on RARE, including what would have to change to break either one. Walnut is not an investment adviser.

A simple financial snapshot. These are approximations and refresh quarterly; for current figures see Ultragenyx Pharmaceutical Inc.'s investor relations page or your broker.

• Revenue (TTM): ~$670M
• FY2025 revenue: ~$673M (up ~20%)
• Q1 2026 revenue: ~$136M
• FY2025 net loss: ~$575M
• Cash and marketable securities: ~$737M (Dec 2025)
• Market cap: ~$2.6B

Ultragenyx trades at a modest multiple of revenue for a biopharma but has no earnings, so conventional P/E valuation does not apply. The market is effectively pricing the pipeline and the credibility of the 2027 profitability goal. Guidance for 2026 revenue is $730 million to $760 million, which excludes any potential new product launches.

These are the investment theses RARE naturally fits into. Each links to a full theme guide listing every other stock that belongs and the ETFs commonly used as a passive proxy.

Other names that sit close to RARE: same theme, named as a direct competitor, or held beside it in the same funds. Each entry says which. Worth a look if you are thinking about diversification within a thesis rather than concentration on one ticker.

There are three common ways to get RARE exposure. Buy shares (or fractional shares) directly at any major broker. Hold an ETF that includes it, which spreads the position across many companies. Or build it into a focused thematic portfolio, so RARE sits alongside other stocks that express the same thesis.

Walnut takes the portfolio route. Describe a thesis where RARE fits (for example “AI infrastructure” or “dividend-growth large-caps”) and the AI proposes 5 to 6 constituents with target weights. You review the plan and fund it through your own broker when you're ready.

New to this? Start with how to invest in stocks, see how to analyze a stock with AI, or compare the best AI stock analyzers.

Ultragenyx is a real, revenue-generating rare-disease company whose valuation hinges on turning a deep pipeline into approvals and, eventually, profits.

Whether RARE is worth buying today depends more on your time horizon and what you already hold than on any single call. We walk through valuation, what would have to go right, and the risks in is RARE a buy or a sell?, and where the stock could go from here in the RARE stock forecast.

For income investors, whether RARE pays a dividend and how the payout looks is covered in does RARE pay a dividend? And to weigh RARE against a peer, read the full side-by-side comparisons: RARE vs BEAM and RARE vs CRSP.