Ultragenyx Pharmaceutical Inc. research snapshot

RARE AI Stock Analysis

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.

Research quality check

information Richness
B-level information richness. Ultragenyx has approved products with commercial revenue disclosures, active analyst coverage, SEC filings, and visible pipeline timelines. However, as a pre-profit biotech, future returns depend on binary clinical and regulatory outcomes that carry inherent uncertainty for AI-driven analysis.
bias Check
The main AI bias risk is that missing pipeline milestones or trial failures can render most analysis obsolete. This page separates filing-backed commercial data from scenario judgments and emphasizes that clinical-stage biotech requires wide scenario ranges.
ai Confidence
Medium data confidence
investment Certainty
Low to medium. Ultragenyx has commercial revenue and a broad pipeline, but the stock price is driven by pipeline catalysts, clinical trial outcomes, regulatory decisions, and financing needs that are inherently hard to predict.

Quick verdict table

DimensionConclusionConfidence
Business qualityUltragenyx 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
MoatThe 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
ManagementFounder 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 trendTTM 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
ValuationAt 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 trendRARE 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 levelKey risks are binary pipeline outcomes (especially GTX-102 for Angelman syndrome), cash burn and potential dilution, commercial execution, competition, and reimbursement pressure.Medium
AI confidenceMedium 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 certaintyLow to medium certainty. Pipeline catalysts could drive significant upside or disappointment. Position sizing should account for binary risk.Low-medium

RARE AI stock forecast

RARE AI Stock Forecast Scenarios

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.

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.

Base case

$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.

Bearish case

$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

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.

LevelValueWhy it matters
Current price$33.82Latest verified close used for this page as of the July 10, 2026 data cutoff.
Near support$28 to $30Support planning zone around the 20-day and 50-day moving-average area after the recent rally.
Secondary support$22 to $24The area around prior consolidation before the breakout. A break below would weaken the recovery narrative.
Near resistance$38 to $40The 52-week high zone and a psychological resistance level. Breakout attempts need volume confirmation.
50-day moving averageAbout $29 to $30RARE traded above the 50-day moving average around the cutoff, supporting the short-term recovery trend.
200-day moving averageAbout $32 to $34The stock was testing or near the 200-day moving average, a key level for confirming the trend change.
MomentumRSI neutral-to-warm, positive MACDRSI was in the 55-65 range after the rally, while MACD showed positive momentum but not yet overbought.
VolumeAbout 1.5 million sharesThe July 10 volume snapshot was below the average of about 2.2 million shares, so breakouts should be confirmed by higher participation.
VolatilityBeta 0.31, elevated biotech volatilityLow beta masks the binary event risk. Position sizing should account for potential 10-20% moves around pipeline data readouts.
InvalidationClose below $28, then $22A 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

RARE AI Trading Strategy Framework

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.

Trend-following setup

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.

Mean-reversion setup

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.

Pipeline catalyst monitor

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

Four-master Research Compression

Business essence

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.

Moat

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.

Munger risk inversion

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.

Management

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.

Industry trend

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.

Valuation and margin of safety

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

RARE Data Table

Every metric below includes a source and last verification date.

MetricValueSourceLast verified
RARE price$33.82 close on July 10, 2026Yahoo Finance quote snapshotJuly 12, 2026
Market capitalization$3.33 billion, verified as $33.82 x 98,488,000 sharesfinancial_rigor.py market cap verification and Yahoo FinanceJuly 12, 2026
TTM revenue$670 millionYahoo Finance and company filingsJuly 12, 2026
TTM net income-$609 million (net loss)Yahoo Finance and company filingsJuly 12, 2026
Cash and cash equivalents$413 millionYahoo Finance balance sheet snapshotJuly 12, 2026
Enterprise value$2.69 billionYahoo FinanceJuly 12, 2026
Price to sales ratio5.03x TTMYahoo Finance and financial_rigor.pyJuly 12, 2026
EPS (TTM)-$6.10Yahoo FinanceJuly 12, 2026
Free cash flow (TTM)-$219.09 millionYahoo FinanceJuly 12, 2026
52-week price range$18.29 to $39.89Yahoo FinanceJuly 12, 2026
Analyst price target$52.32 average, range $25 to $96Yahoo Finance analyst estimatesJuly 12, 2026
Valuation math5.03x P/S, EV/Revenue 4.01x, P/FCF -15.23x (financial_rigor.py)financial_rigor.py valuation verification and Yahoo FinanceJuly 12, 2026

Frequently Asked Questions

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.