5 Most Promising Mid-Cap Healthcare Stocks According to Hedge Funds

4. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 10 most promising mid-cap healthcare stocks according to hedge funds.

On May 21, Cantor Fitzgerald analyst Kristen Kluska maintained an Overweight rating on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and raised the price target from $84 to $96. This valuation adjustment is based on the growing institutional confidence in the underlying investment case for GTX-102, which is used to treat Angelman syndrome.

Due to highly favorable testing parameters, Kluska says that the clinical asset now shows a significantly higher likelihood of eventual success. This optimistic assessment is heavily driven by the specific structural design of its Phase 3 trial. Specifically, the comprehensive evaluation utilizes the Bayley-4 cognitive raw score alongside the MDRI metric to measure efficacy.

By organizing the clinical architecture in this manner, it can successfully establish two entirely independent pathways to achieve formal statistical success, thereby mitigating developmental risk and strengthening the broader product portfolio.

On May 8, the price target on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) was reduced by Wedbush from $27 to $26, which still yields almost 14% upside at the current level. The firm maintained a Neutral rating on the stock. Although the revenue underperformance is upsetting, management reaffirmed all aspects of the financial year 2026 revenue projection. The firm also anticipates that Crysvita will correct its course during the rest of the year.

Wedbush also expects the company to turn profitable sometime in 2027. Predominantly, Ultragenyx has two PDUFAs for DTX401 and UX111, both of which offer a chance for profitable PRVs, while Wedbush is awaiting Phase 3 ASPIRE results for GTX102.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops novel therapies, with a focus on identifying, acquiring, and commercializing products for rare and ultra-rare genetic diseases. It has a strong emphasis on gene therapy and covers various stages of clinical trials. The company is currently going through high cash burn with the aim of turning profitable from 2027 onwards.