Bullish case
$60 to $85
More likely if GTX-102 shows strong efficacy in Angelman syndrome, UX143 and DTX401 succeed in Phase 3, commercial revenue from Crysvita and Evkeeza accelerates, and the company manages cash without major dilution.
Ultragenyx Pharmaceutical Inc. research snapshot
RARE AI stock analysis currently reads Ultragenyx Pharmaceutical as a clinical-stage biotech with four approved rare-disease products and a late-stage pipeline that includes GTX-102 for Angelman syndrome, UX143 for osteogenesis imperfecta, and several gene therapy candidates. The analysis is not a certain price prediction. At the July 10, 2026 cutoff, the latest verified close used here was $33.82, market capitalization was about $3.33 billion, and the main question was whether key pipeline readouts, commercial execution of approved products, and cash management can support further upside. This is informational research and not investment advice.
Current price
$33.82
Market cap
$3.33 billion
AI score
55 / 100
Rating
Speculative biotech with pipeline catalysts
Trend status
Recovering from 52-week lows, up ~85% from lows
Data cutoff (updated weekly)
July 10, 2026
Informational use only. This page is not investment advice.
| Dimension | Conclusion | Confidence |
|---|---|---|
| Business quality | Ultragenyx sells four approved rare-disease therapies and has a Phase 3 pipeline spanning Angelman syndrome, OI, MPS IIIA, GSD Ia, and OTC deficiency. Revenue is growing but the business is not yet profitable. | Medium |
| Moat | The moat comes from orphan drug exclusivity, regulatory infrastructure for rare disease development, modality breadth (biologics, gene therapy, ASO), and commercial infrastructure in a niche market. | Medium |
| Management | Founder and CEO Emil Kakkis has led the company since 2010 with a track record of bringing rare disease drugs to market. Key-person risk exists given the founder-led structure. | Medium |
| Financial trend | TTM revenue reached about $670 million driven by Crysvita and Evkeeza sales, but the company reported a TTM net loss of about $609 million. Cash of $413 million provides runway into key catalysts. | Medium-high |
| Valuation | At roughly 5x TTM revenue and an enterprise value of about $2.69 billion, the stock prices pipeline upside. Standard PE valuation is not applicable given negative earnings. | Low-medium |
| Technical trend | RARE has rallied sharply from 52-week lows near $18.29 to the $33.82 level, recovering about 85%. Momentum is positive but the stock remains well below prior highs above $60. | Medium |
| Risk level | Key risks are binary pipeline outcomes (especially GTX-102 for Angelman syndrome), cash burn and potential dilution, commercial execution, competition, and reimbursement pressure. | Medium |
| AI confidence | Medium confidence for commercial revenue data and balance sheet math. Lower confidence for pipeline probability-weighted outcomes because clinical trial results are inherently binary. | Medium data confidence |
| Investment certainty | Low to medium certainty. Pipeline catalysts could drive significant upside or disappointment. Position sizing should account for binary risk. | Low-medium |
RARE AI stock forecast
The RARE AI stock forecast uses scenario ranges around the $33.82 cutoff price. It does not claim that AI can predict a specific future price. The bullish case requires positive Phase 3 data for GTX-102 and other pipeline candidates, continued commercial revenue growth, and controlled cash burn. The base case assumes mixed pipeline outcomes and gradual commercial growth. The bearish case assumes pipeline setbacks, dilutive financing, or commercial disappointment.
$60 to $85
More likely if GTX-102 shows strong efficacy in Angelman syndrome, UX143 and DTX401 succeed in Phase 3, commercial revenue from Crysvita and Evkeeza accelerates, and the company manages cash without major dilution.
$30 to $45
More likely if pipeline readouts are mixed, commercial revenue grows modestly, the company raises capital at moderate dilution, and the stock trades in line with pre-profit biotech peers.
$14 to $22
More likely if GTX-102 or other key pipeline candidates fail, cash burn forces dilutive financing, competition erodes approved product sales, or the biotech sector faces broad risk-off sentiment.
RARE AI technical analysis
RARE AI technical analysis starts from the $33.82 July 10 close used for this July 12 static page. Public technical sources showed the stock above its 50-day moving average after a strong rally from 52-week lows, with RSI in neutral-to-warm territory. Because this page does not fetch request-time market data, traders should confirm levels on a live chart before acting.
| Level | Value | Why it matters |
|---|---|---|
| Current price | $33.82 | Latest verified close used for this page as of the July 10, 2026 data cutoff. |
| Near support | $28 to $30 | Support planning zone around the 20-day and 50-day moving-average area after the recent rally. |
| Secondary support | $22 to $24 | The area around prior consolidation before the breakout. A break below would weaken the recovery narrative. |
| Near resistance | $38 to $40 | The 52-week high zone and a psychological resistance level. Breakout attempts need volume confirmation. |
| 50-day moving average | About $29 to $30 | RARE traded above the 50-day moving average around the cutoff, supporting the short-term recovery trend. |
| 200-day moving average | About $32 to $34 | The stock was testing or near the 200-day moving average, a key level for confirming the trend change. |
| Momentum | RSI neutral-to-warm, positive MACD | RSI was in the 55-65 range after the rally, while MACD showed positive momentum but not yet overbought. |
| Volume | About 1.5 million shares | The July 10 volume snapshot was below the average of about 2.2 million shares, so breakouts should be confirmed by higher participation. |
| Volatility | Beta 0.31, elevated biotech volatility | Low beta masks the binary event risk. Position sizing should account for potential 10-20% moves around pipeline data readouts. |
| Invalidation | Close below $28, then $22 | A close below the 50-day area near $28 would weaken the recovery setup. A break below $22 would challenge the broader uptrend. |
RARE AI trading strategy
The RARE AI trading strategy is a rules-based framework, not personalized advice. It combines pipeline catalyst awareness, technical confirmation, position sizing for binary outcomes, and clear invalidation levels.
Look for RARE to hold above the 50-day area near $28 to $30 and break above the $38 to $40 resistance zone with increasing volume. Wait for pipeline catalyst confirmation before adding to positions.
A failed breakout or daily close below the 50-day area should reduce confidence. Reduce position size before binary pipeline events.
If RARE pulls back toward $28 to $30 without fundamental deterioration, evaluate cash runway, pipeline timelines, and upcoming catalysts before considering entry.
Do not average down solely because the stock is cheap. Biotech stocks can stay depressed if catalysts are delayed, and binary trial outcomes can drive 30-50% moves.
Track GTX-102 Angelman syndrome Phase 3 data, UX143 OI trial enrollment and readouts, DTX401 and DTX301 gene therapy progress, quarterly revenue trends for Crysvita and Evkeeza, cash position, and any financing announcements.
Lower the rating if cash burn accelerates without clear pipeline progress, or if key trial timelines are pushed out significantly.
Investment research summary
Ultragenyx discovers, develops, and commercializes therapies for rare and ultra-rare genetic diseases. Customers are patients, specialty pharmacies, and healthcare systems seeking treatments for conditions with few or no alternatives.
The moat is built on orphan drug exclusivity, regulatory expertise in rare disease drug development, broad modality capabilities (biologics, gene therapy, ASO), and commercial infrastructure in a niche market. Approved products generate recurring revenue but face long-term competition.
The thesis can fail if GTX-102 or other key pipeline candidates show weak efficacy, clinical trials suffer enrollment or safety setbacks, commercial sales disappoint, cash burn forces dilutive financing, or competition erodes approved product economics.
Founder and CEO Emil Kakkis has led the company since 2010 with a focus on rare disease drug development. The team has successfully brought multiple products to market. The main risk is key-person dependence on the founder and the challenge of managing a broad pipeline with limited resources.
Rare disease gene therapy and precision medicine represent a durable secular trend supported by regulatory incentives, improving delivery technologies, and growing payer acceptance. Ultragenyx is positioned across multiple rare disease modalities.
At roughly 5x TTM revenue and an enterprise value of about $2.69 billion, the stock prices meaningful pipeline upside. Without GAAP profitability, traditional valuation metrics have limited relevance. The margin of safety depends on successful pipeline execution and disciplined capital management.
Source-backed data
Every metric below includes a source and last verification date.
| Metric | Value | Source | Last verified |
|---|---|---|---|
| RARE price | $33.82 close on July 10, 2026 | Yahoo Finance quote snapshot | July 12, 2026 |
| Market capitalization | $3.33 billion, verified as $33.82 x 98,488,000 shares | financial_rigor.py market cap verification and Yahoo Finance | July 12, 2026 |
| TTM revenue | $670 million | Yahoo Finance and company filings | July 12, 2026 |
| TTM net income | -$609 million (net loss) | Yahoo Finance and company filings | July 12, 2026 |
| Cash and cash equivalents | $413 million | Yahoo Finance balance sheet snapshot | July 12, 2026 |
| Enterprise value | $2.69 billion | Yahoo Finance | July 12, 2026 |
| Price to sales ratio | 5.03x TTM | Yahoo Finance and financial_rigor.py | July 12, 2026 |
| EPS (TTM) | -$6.10 | Yahoo Finance | July 12, 2026 |
| Free cash flow (TTM) | -$219.09 million | Yahoo Finance | July 12, 2026 |
| 52-week price range | $18.29 to $39.89 | Yahoo Finance | July 12, 2026 |
| Analyst price target | $52.32 average, range $25 to $96 | Yahoo Finance analyst estimates | July 12, 2026 |
| Valuation math | 5.03x P/S, EV/Revenue 4.01x, P/FCF -15.23x (financial_rigor.py) | financial_rigor.py valuation verification and Yahoo Finance | July 12, 2026 |
This RARE AI stock analysis is an informational tool for research and education only. It is not investment advice, a recommendation, or a guarantee of future performance. Forecast ranges are scenarios based on available data as of July 10, 2026 and can be wrong. Biotech investments carry elevated risk due to binary clinical trial outcomes and regulatory decisions.
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