Billionaire Stanley Druckenmiller’s Top 10 Stocks Picks with Huge Upside Potential

3. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)

Upside Potential as of April 11: 174.43%

Duquesne Capital’s Stake Value: $3.60 million

Number of Hedge Fund Holders: 57

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is another biopharmaceutical company in Druckenmiller’s portfolio. It develops treatments for rare and ultra-rare genetic diseases, which often have no approved therapies. The leading products include approved treatments like Burosumab (for a bone disorder called XLH), Triheptanoin (for certain metabolic disorders), and Vestronidase alfa (for a rare enzyme deficiency).

In Q4 2024, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) reported strong financial results, with total revenue of $560 million, exceeding previous guidance. For 2025, the company projects continued growth with total revenue between $640 million to $670 million.

In light of this performance, CEO Emil D. Kakkis believes that they “have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products.”

The company is approaching several major catalysts in 2025. One of the major ones is a pending FDA decision for Ultragenyx Pharmaceutical Inc.’s (NASDAQ:RARE) gene therapy to treat Sanfilippo syndrome, which is expected in the second half of the year. The firm is also planning to submit a biologics license application for its Glycogen Storage Disease Type Ia (GSDIa) gene therapy in mid-2025. Additionally, the Phase 3 ORBIT study for setrusumab in osteogenesis imperfecta is progressing toward a second interim analysis in mid-2025. On March 27, JPMorgan raised Ultragenyx’s (NASDAQ:RARE) price target from $104 to $117 and rated the shares as Overweight. It cites a “very favorable” reward/risk profile for the company’s clinical programs.