10 Best Cancer Stocks to Invest in for Long-Term Gains

6. Krystal Biotech, Inc. (NASDAQ:KRYS)

Number of Hedge Fund Holders: 30

Total 5-Year Return: 144.11%

Krystal Biotech, Inc. (NASDAQ:KRYS) is a commercial-stage gene therapy company using a proprietary, redosable HSV-1 vector platform to develop treatments for genetic diseases and cancer. The company saw a breakout year in 2024, generating $290.5 million in revenue (up 473% year-over-year), which was driven by the successful U.S. launch of VYJUVEK for dystrophic epidermolysis bullosa (DEB).

High reimbursement rates, strong patient compliance (85%), and gross margins of 95% contributed to $89.2 million in full-year net income. With $749.6 million in cash and investments, the company has transitioned from an R&D-focused biotech to a profitable commercial company.

In oncology, Krystal Biotech, Inc. (NASDAQ:KRYS)’s lead candidate KB707 targets solid tumors through the delivery of immune-stimulating cytokines IL-12 and IL-2. Administered intratumorally or via inhalation, KB707 has shown a 27% objective response rate and 73% disease control rate in early-stage trials for non-small cell lung cancer (NSCLC). Both forms have received FDA Fast Track Designation.

Krystal Biotech, Inc. (NASDAQ:KRYS) is also expanding its pipeline with KB407 (Cystic Fibrosis), KB408 (Alpha-1 Antitrypsin Deficiency), and dermatological and aesthetic programs like KB105 and KB301, with key clinical milestones expected throughout 2025.