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 in the industry today.
In simple terms, the US National Library of Medicine defines gene therapies as ways to prevent or treat diseases by using genes. It has the potential to treat patients by inserting the gene into a cell rather than treating with drugs or surgeries.
What’s more, this technique also has the possibility of treating a range of disorders, like inherited diseases, cancer and viral infections. Unlike other treatment options, gene therapy is intended as a one-time treatment, but may need additional dosing in order to completely rid the disease.
Looking at its 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 which has been proven to be well-tolerated in clinical trials.
On that note, of course there is a range of gene therapy companies in the space that investors can get acquainted with. The companies listed below were compiled using Crunchbase.com’s gene therapy companies data and are listed in order according to that list. All numbers and figures are current as of July 18, 2019. Here’s a closer look at those gene therapy companies.
1. Spark Therapeutics (NASDAQ:ONCE)
Market cap: US$3.77 billion; current share price: US$98.18
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, intends to treat choroideremia, which is a retinal disease that primarily affects males that 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.
Its 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 being developed to treat 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 was 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, 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 Therapeutics 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, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.