Gene Therapy Stocks: 5 Biggest Companies

Market cap: US$215.46 billion

One of the largest pharmaceutical companies in the world, Switzerland-based Novartis is focused on treatments for a wide range of diseases, including various cancers, malaria, leprosy and sickle cell disease. Novartis is developing adeno-associated-virus-based and CRISPR-based gene therapies.

Grand View Research states that Novartis' Zolgensma, which targets the underlying cause of spinal muscular atrophy, “is the only gene treatment in this field to have been approved” by the US Federal Drug Administration (FDA).

Additionally, Novartis has an in-licensing agreement with Roche Holdings (OTCQX:RHHBY,SW:ROG) subsidiary Spark Therapeutics that gives the latter commercialization rights within the US for the gene therapy Luxturna, while Novartis retains commercial rights for outside markets. Luxturna is “the first gene therapy that can be directly administered for targeting a genetic disease caused by mutations in a single genetic factor,” notes the research firm.

Market cap: US$140.12 billion

Global biopharmaceutical company Bristol-Myers Squibb has made groundbreaking advancements in the therapeutic areas of oncology, hematology, immunology and cardiovascular disease. Research at Bristol-Myers Squibb is focused on several key areas, including advancing CAR T-cell therapy for the treatment of certain cancers.

In April, Bristol-Myers Squibb strengthened its cell therapy capabilities with the addition of a new US-based manufacturing facility, which will produce viral vectors for both of the company's CAR T-cell therapies.

Market cap: US$103 billion

Global biopharmaceutical company Gilead Sciences has spent three decades advancing breakthrough medicines to prevent and treat serious diseases such as HIV, viral hepatitis and cancer. In 2017, the company acquired stem cell therapy developer Kite Pharma just before Kite was granted approval for its cell-based gene medicine for blood cancer, the CAR T-cell therapy Yescarta.

The second gene therapy approved by FDA, Yescarta was also the first such therapy for certain types of non-Hodgkin's lymphoma. The treatment aids a patient’s immune system in fighting the disease. Sales of Yescarta increased from US$695 million in 2021 to US$1.16 billion in 2022.

Market cap: US$16.97 billion

BioMarin Pharmaceutical’s research and early development is led by Dr. Kevin Eggan, a pioneer in stem cell biology. The company develops and commercializes therapies for serious genetic diseases with unmet medical needs.

BioMarin’s biologics license application for Roctavian, its gene therapy for adults with severe hemophilia, is currently under FDA review. Under consideration is the data from a Phase 3 study, which “is the longest and largest to date for a gene therapy in hemophilia.”

Market cap: US$892.18 billion

Genomic medicine company uniQure develops and markets gene therapy products for patients with severe genetic diseases. The company's adeno-associated-virus-based gene therapy platform targets liver-directed and central nervous system disorders.

uniQure signed a licensing agreement in early 2023 for Apic Bio’s APB-102, a clinical-stage gene therapy for the treatment of patients with amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1. Under the agreement, uniQure will obtain the rights to develop and market APB-102 worldwide.