5 Gene Therapy Stocks for Investors

- May 12th, 2021

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

For life sciences 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 has the ability to 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 in order to effectively treat the disease.


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Looking at the market, the global gene therapy space was worth US$2.26 billion in 2020, and is estimated to grow at a compound annual growth rate of 20.4 percent from 2021 to 2028 to reach US$10 billion.

As a report from Grand View Research states, the goal of gene therapy is to pinpoint and treat rare conditions and diseases that don’t have many treatment options. In particular, adeno-associated viruses have become vital in gene therapy. Adeno-associated viruses are naturally occurring viruses that have been proven to be well tolerated in clinical trials.

There is of course a range of gene therapy companies that might pique investors’ interest, but looking at the biggest players is often a good place to start. With that in mind, here’s a look at the top gene therapy stocks by market cap as of May 3, 2021. Read on to learn more about them

1. Bluebird Bio (NASDAQ:BLUE)

Market cap: US$2 billion; current share price: US$29.60

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, which is a metabolic disorder that affects boys between the ages of three and 12; multiple myeloma, which is cancer of cells in the blood; sickle cell disease, which is a type of inherited anemia that causes serious problems due to decreased oxygen flow in the blood; and transfusion-dependent beta-thalassemia, which can lead to serious genetic blood diseases.

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

2. Kura Oncology (NASDAQ:KURA)

Market cap: US$1.77 billion; current share price US$26.74

Clinical-stage biopharmaceutical company Kura Oncology is researching and developing medicines for the treatment of cancer. Its drug candidate pipeline includes targeted therapies that block cancer from growing and spreading by interfering with specific genes or proteins necessary for tumor growth.

One such drug candidate is Tipifarnib, a farnesyl transferase inhibitor for HRAS mutant solid tumors and chronic myelomonocytic leukemia. HRAS is a gene implicated in the development and progression of head and neck squamous cell carcinoma. Based on data from a Phase 2 clinical trials, Tipifarnib has been granted breakthrough therapy designation by the US Food and Drug Administration.

3. Translate Bio (NASDAQ:TBIO)

Market cap: US$1.73 billion; current share price: US$23.03

Translate Bio is a mRNA therapeutics company developing a new class of therapeutic drugs to treat or prevent debilitating or life-threatening diseases caused by protein or gene dysfunction. The company has a proprietary mRNA therapeutic platform that it uses to create mRNA that encodes functional proteins.


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Translate Bio’s lead product candidate, MRT5005, is designed for the treatment of cystic fibrosis, and is being evaluated as an inhaled treatment in an ongoing Phase 1/2 clinical trial.

The company also has a COVID-19 vaccine candidate called MRT5500; it is undergoing Phase 1/2 clinical trials under a collaboration agreement with Sanofi Pasteur, the vaccine division of multinational pharmaceutical company Sanofi (NASDAQ:SNY). In April, Translate Bio announced the peer-reviewed publication of favorable preclinical results for MRT5500 in the journal npj Vaccines. Clinical data from the Phase 1/2 trial is anticipated in the third quarter.

4. Krystal Biotech (NASDAQ:KRYS)

Market cap: US$1.71 billion; current share price: US$77

Krystal Biotech is a gene therapy company with in-house manufacturing capabilities and a novel, redosable gene therapy platform referred to as the Skin TARgeted Delivery platform, or STAR-D platform. It is used to develop treatments for rare or orphan dermatological indications.

The company’s pipeline includes products targeting monogenic and congenital skin diseases, including dystrophic epidermolysis bullosa (DEB) and autosomal recessive congenital ichthyosis.

In March 2021, Krystal Biotech announced the completion of enrolment in a GEM-3 study of its B-VEC product for DEB. The company expects to report top-line results from the study in Q4 2021.

5. Replimune (NASDAQ:REPL)

Market cap:US$1.7 billion; current share price: US$36.75

Replimune is a biotechnology company developing oncolytic immunogene therapies derived from its Immulytic platform. These therapies are designed to improve the direct cancer-killing effects of selective virus replication and the potency of the immune response to the tumor antigens released. The approach is expected to be highly synergistic with other treatments across a broad range of cancers.

The company’s product pipeline includes RP1, which is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor-killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response through the expression of a GALV-GP R- fusogenic protein and GM-CSF.

In 2020, Replimune started manufacturing processes at its 63,000 square foot facility in Massachusetts. This will support late-stage development and full commercialization of all of its products.

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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6 responses to “5 Gene Therapy Stocks for Investors

    1. Hi Dale,
      It will be interesting to see if Propanc (OTCQB:PPCH) receives orphan designation for PRP, its therapy candidate for pancreatic cancer. The company filed that application in early September of this year. You’re right, it could be one to keep an eye on too!

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