PFE - 5 Gene Therapy Stocks for Investors
For life sciences investors, the gene therapy market is one of the most interesting plays today.
The US National Library of Medicine defines gene therapies as specialized treatments using genes for the prevention or treatment of disease. Gene therapies are designed to treat 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.
Investing in GeneticsCurious about this emerging market?
Find out what new genetics investors need to know.Looking at market size, the global gene therapy market 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 (AAVs) have become vital in gene therapy. AAVs 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. The gene therapy stocks listed below all have market caps of US$2 billion and under, and all numbers and figures were current as of May 3, 2021. Here’s a closer look at these gene therapy companies.
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, its focus area 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.
The company’s gene therapy product, Zynteglo, 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.
Investing in GeneticsCurious about this emerging market?
Find out what new genetics investors need to know.Spark’s SPK-8001 is in a Phase 1/2 trial to treat hemophilia A; the company’s fidanacogene elaparvovec has been transferred to Pfizer (NYSE: PFE ) and began a Phase 3 trial in June 2018.
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. The company’s 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 (HNSCC). Based on data from a Phase 2 clinical trials, Tipifarnib granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA).
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.
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 MRT5500 undergoing phase 1/2 clinical trials under a collaboration agreement with Sanofi Pasteur, the vaccine division of multinational pharmaceutical company Sanofi (NASDAQ: SNY ).
Investing in GeneticsCurious about this emerging market?
Find out what new genetics investors need to know.In April 2021, Translate Bio announced that peer-reviewed publication of favorable preclinical results for its Covid-19 vaccine candidate MRT5500 will appear in the journal npj Vaccines. Clinical data from the phase 1/2 trial is anticipated in Q3 2021.
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, used to develop treatments for rare or orphan dermatological indications.
The company’s pipeline includes products targeting the treatment of monogenic and congenital skin diseases, including dystrophic epidermolysis bullosa (DEB) and autosomal recessive congenital ichthyosis (ARCI).
In March 2021, Krystal Biotech announced the completion of enrollment in the GEM-3 study of B-VEC 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 immuno-gene therapies derived from its Immulytic™ platform 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 cancer 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 commenced GMP manufacturing in its completed 63,000-square-foot state-of-the-art facility in Massachusetts, which 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.
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Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.
Investing in Genetics