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Which gene therapy stocks should be on investors’ radar? Here are five companies developing a wide range of gene therapy products.

For life science investors, the gene therapy market is one of the most interesting plays available today.

The US National Library of Medicine defines gene therapies as specialized treatments that use genes for the prevention or treatment of disease. Gene therapies are designed to help patients by inserting a gene into a cell rather than treating the patient with drugs or surgeries.

This technique can treat a range of disorders, such as inherited diseases, cancer and viral infections. Unlike other treatment options, gene therapy is intended as a one-time event, although additional dosing may be required.

A Mordor Intelligence report states that the global gene therapy space was worth US$4.12 billion in 2021, and is seen growing at a compound annual growth rate of 25.71 percent from 2021 to 2027 to reach US$14.82 billion.

“The major factors propelling the market's growth include technological advancements, rising investments in research and development, and the growing prevalence of target diseases,” as per the report. “Investment in research and development (R&D) activities are also expected to have a significant effect on the market.”

Mordor Intelligence also notes that a number of gene therapy companies are building technology platforms that incorporate in-house capabilities, strategic collaborations, R&D, licensing, and merger and acquisition activities.

There is, of course, a range of gene therapy companies that might pique investors’ interest. However, the research firm notes that five major players are dominating this market. Here the Investing News Network takes a look at these top gene therapy stocks in order of market cap. All data was current as of April 12, 2022.

1. Novartis (NYSE:NVS,SWX:SOVN)

Market cap: US$207.49 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 also developing a cell and gene therapy technology platform that includes adeno-associated virus-based therapy, CAR-T cell therapy and gene therapy based on CRISPR.

Mordor Intelligence notes, “In December 2021, the company acquired Gyroscope Therapeutics, adding a one-time gene therapy that could transform care for geographic atrophy, a leading cause of blindness.”

More recently, Novartis was granted US Federal Drug Administration (FDA) commercial licensure approval for its second multi-product gene therapy manufacturing facility.

2. Amgen (NASDAQ:AMGN)

Market cap: US$133.25 billion

Biotech pioneer Amgen is focused on taking an “industrial-scale approach to gene-based research” in order to deliver treatments that target high unmet medical needs and improve health outcomes. The company is meeting this goal by developing a strong pipeline of medicines that “aggressively leverage human genetics in its research.”

In early March 2021, Amgen announced the US$1.9 billion acquisition of cancer drug developer Five Prime Therapeutics. The deal included a potentially first-in-class anti-fibroblast growth factor receptor 2b antibody that has subsequently garnered breakthrough therapy designation from the FDA.

3. Gilead Sciences (NASDAQ:GILD)

Market cap: US$77.14 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, 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$563 million in 2020 to US$695 million in 2021, according to Fierce Pharma, which states that the drug “has potential to reach US$1.5 billion in sales if it can win approval in second-line B-cell lymphoma.”

4. Biogen (NASDAQ:BIIB)

Market cap: US$30.63 billion

Based in Cambridge, Massachusetts, Biogen first made a name for itself in the field of neuroscience. The global biotechnology company develops, manufactures and markets therapies aimed at treating serious neurological, neurodegenerative, autoimmune and rare diseases.

Biogen introduced the first approved treatment for spinal muscular atrophy. Today, it commercializes biosimilars of advanced biologics and is researching areas such as: multiple sclerosis and neuroimmunology; Alzheimer's disease and dementia; and neuromuscular disorders. However, Biogen’s launch of its Alzheimer’s disease drug Aduhelm has faced headwinds and the company expects minimal sales of the drug in 2022.

5. Bluebird Bio (NASDAQ:BLUE)

Market cap: US$317.16 million

Bluebird Bio’s therapy treatments are geared at treating genetic diseases and different types of cancer. More specifically, the company’s focus is on conditions such as cerebral adrenoleukodystrophy, multiple myeloma, sickle cell disease and transfusion-dependent beta-thalassemia.

Zynteglo, which is Bluebird Bio’s gene therapy product, has been approved in the EU, Iceland, Liechtenstein and Norway; is the first and only one-time gene therapy treatment for infusion-dependent beta-thalassemia patients.

The company sees its near-term catalysts as “anticipated FDA approvals for its gene therapies for beta-thalassemia and cerebral adrenoleukodystrophy in 2022, and the potential submission of a biologics license application for lovotibeglogene autotemcel gene therapy for sickle cell disease planned in the first quarter of 2023.”

This is an updated version of an article originally published by the Investing News Network in 2016.

Don’t forget to follow us @INN_LifeScience for real-time news updates!

Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.

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