Billionaire Buys Stake In Gene Therapy Stock

Ultragenyx may not have crossed your radar but it’s already in billionaire Stan Druckenmiller’s portfolio. What has he spotted in this novel therapies for rare and ultra-rare genetic diseases firm that convinced him to take a position?

Its focus on underserved patient populations, deep pipeline of innovative products, and strong track record in obtaining regulatory approvals make it an attractive long-term investment, but there’s more under the hood to know.

Specialty Focus in Rare and Ultra-Rare Diseases

By targeting serious genetic conditions with limited or no existing treatments, Ultragenyx can command premium pricing and benefit from strong support by payers, patient advocacy groups, and even regulators.

Rare disease therapies that Ultragenyx specializes in often receive orphan drug designations, providing extended market exclusivity, expedited review pathways, and potential tax credits, helping de-risk research and development.

And to get technical a moment, here are some of the drugs already approved. Crysvita (burosumab) has been approved for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). This has been a first-in-class therapy and provides a foundational revenue stream, with further opportunity to grow abroad and generate label extensions.

Dojolvi (UX007) has also been approved for long-chain fatty acid oxidation disorders (LC-FAOD). As one of the few treatment options for this ultra-rare metabolic disease, Dojolvi cements Ultragenyx’s commercial opportunity and adds incremental revenue growth.

Diverse Pipeline of High-Potential Candidates

Ultragenyx’s push into AAV (adeno-associated virus) gene therapy puts it at the forefront of an area with strong long-term growth. Programs like DTX301 (for ornithine transcarbamylase deficiency) and DTX401 (for glycogen storage disease type Ia) offer good upside opportunity if clinical outcomes are positive.

Beyond gene therapy, Ultragenyx is exploring other modalities and diversifying its technological capabilities. It’s also partnered with GeneTx Biotherapeutics for Angelman Syndrome, a severe neurogenetic disorder with no approved treatments. This candidate, if successful, is likely to create a meaningful revenue stream and address significant unmet needs.

Here Comes The Revenues Ramp?

Ultragenyx has already shepherded products from clinical development to regulatory approval, and has also navigated accelerated approvals under orphan drug and rare pediatric designations.

Thanks to a growing sales force and distribution networks, particularly in North America and Europe, the company has built a scalable infrastructure for upcoming launches, potentially accelerating revenue ramp.

Sales from Crysvita and Dojolvi continue to grow and are helping to fund future R&D activities.

Despite not being fully profitable yet, Ultragenyx has historically maintained a strong cash position that enables it to pursue new research programs and acquisitions or partnerships that can enhance its portfolio.

Potential for Significant Upside

As the global rare disease market expands, new gene therapy approvals and label expansions for existing drugs are likely to translate to substantial earnings growth.

Investors may well be overlooking the long-term earnings potential of Ultragenyx’s gene therapy candidates, which is likely to lead to an attractive entry point if clinical outcomes and approvals materialize positively.

Why Is Stan Buying Ultragenyx?

Ultragenyx offers Stan and his Duquesne Family Office exposure to a rare disease-focused biotechnology company with a growing portfolio of commercial products and a promising, diversified pipeline.

Its leadership in gene therapy, established commercial footing, and focus on high-value orphan indications place it well for durable long-term growth.

While biotech investments carry inherent risks, Ultragenyx’s track record of product approvals and ongoing pipeline advancement support a compelling risk-reward profile for investors.