What gene therapy companies 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 today.
In simple terms, the US National Library of Medicine defines gene therapies as ways to prevent or treat diseases by using genes. Gene therapies have the potential 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, like 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 completely rid the patient of the disease.
Looking at market size, the global gene therapy market was worth US$536.45 million in 2018, and is estimated to grow at a compound annual growth rate of 33.9 percent to reach US$5.5 billion by 2026.
As a report from Grandview 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 virus (AAV) has become vital in gene therapy. AAV is a naturally occurring virus that has been proven to be well tolerated in clinical trials.
There is of course a range of gene therapy companies that investors can get acquainted with. The gene therapy stocks listed below all have market caps over US$500 million. All numbers and figures were current as of March 3, 2020. Here’s a closer look at these gene therapy companies.
1. Roche (OTCQX:RHHBY)
Market cap: US$297.13 billion; current share price: US$40.57
Roche’s business platform combines both pharmaceuticals and diagnostics, with the latter expanding when the company acquired Spark Therapeutics at the end of 2019.
Spark Therapeutics’ gene therapy treatments are geared towards a range of genetic diseases, such as blindness, hemophilia, lysosomal storage disorders and neurodegenerative conditions.
The company currently has nine investigational gene therapies, three of which are in clinical trials. Its first investigational candidate in the US, SPK-7001, aims to treat choroideremia, which is a retinal disease that primarily affects males and eventually manifests into complete blindness; it often presents in the early stages of childhood as night blindness. SPK-7001 is currently in a Phase 1/2 open label trial.
Spark’s SPK-8001 is in a Phase 1/2 trial to treat hemophilia A, and the company’s fidanacogene elaparvovec has been transferred to Pfizer (NYSE:PFE) and began a Phase 3 trial in June 2018.
2. Bluebird Bio (NASDAQ:BLUE)
Market cap: US$7.79 billion; current share price: US$141.25
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.
3. Audentes Therapeutics (NASDAQ:BOLD)
Market cap: US$1.69 billion; current share price: US$38.08
Audentes Therapeutics describes itself as a genetics medicine organization that is working on products for rare neuromuscular diseases. Through its AAV gene therapy technology platform, Audentes Therapeutics’ programs are being developed between three modalities: gene replacement, vectorized exon skipping and vectorized RNA knockdown.
The company currently has six candidates that it is developing in its pipeline for diseases such as X-linked myotubular myopathy, Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy.
4. Orchard Therapeutics (NASDAQ:ORTX)
Market cap: US$1.19 billion; current share price: US$13.85
Orchard Therapeutics’ gene therapy treatments are also intended for rare diseases. Its portfolio includes strimvelis, which has been approved by the European Medicines Agency to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID).
The company’s other programs are for primary immunodeficiencies, neurometabolic disorders and hemoglobinopathies. Its three registrational studies are for ADA-SCID, metachromatic leukodystrophy and Wiskott-Aldrich syndrome. In clinical programs, Orchard has programs for X-linked chronic granulomatous disease, transfusion dependent beta-thalassemia and mucopolysaccharidosis type 1.
5. Voyager Therapeutics (NASDAQ:VYGR)
Market cap:US$903.87 million; current share price: US$24.54
Voyager Therapeutics is a clinical-stage gene therapy company focused on treating severe neurological diseases. It is doing so by advancing the field of AAV gene therapy.
The company’s pipeline has programs for Parkinson’s disease, a form of amyotrophic lateral sclerosis (ALS), Huntington’s disease, Friedreich’s ataxia and Alzheimer’s disease, as well as other neurodegenerative diseases.
This is an updated version of an article originally published by the Investing News Network in 2016.
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Securities Disclosure: I, Nicole Rashotte, hold no direct investment interest in any company mentioned in this article.