11 Best Gene Therapy Stocks to Buy According to Analysts

7. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM)

Price Target Upside: 58.55% 

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) focuses on developing therapies for rare liver diseases caused by bile acid buildup, including progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). Its flagship product, Livmarli (maralixibat), is approved for treating cholestatic pruritus in pediatric ALGS patients, contributing to the company’s revenue.

Vivet Therapeutics collaborates with the company to develop AAV gene therapies VTX-803 and VTX-802, which treat PFIC subtypes 3 and 2. These therapeutic approaches aim to restore defective MDR3 transporter and bile salt export pump (BSEP) functions, allowing bile secretion to be restored and preventing serious liver damage. Tests on VTX-803 showed significant improvements in PFIC3 biomarker levels, and the drug has earned Orphan Drug Designation from both the FDA and EMA.

Total net product sales for Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) reached $99.4 million during Q4 2024, which reflects a 43% increase from the previous year. Sales of Livmarli increased by 50% during the year, with the product generating $64.1 million in the fourth quarter and $213.3 million in annual sales. During the period, the company spent $140.6 million on R&D expenses and $202.2 million on SG&A expenses. The corporation’s cash position stood at $293 million, with product sales projected to be between $420 million and $435 million for 2025.

The FDA approved the drug CTEXLI (chenodeoxycholic acid) for the treatment of cerebrotendinous xanthomatosis (CTX), providing the drug with seven years of market protection. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) also plans to begin a Phase 2 trial of MRM-3379 to treat Fragile X syndrome, thereby increasing potential expansion opportunities.