Almost $3.1 billion was invested in 68 gene therapy companies between 2007 and 2016, a figure that could increase dramatically thanks to recent advances in the science.
On July 12, a U.S. Food and Drug Administration (FDA) panel voted 10-0 to recommend the first gene therapy in the United States. The treatment for children who have B-cell acute lymphoblastic leukemia, a type of blood cancer, could be the first of a multitude of gene therapy solutions for many diseases, including cancers of the breast, lung, ovary, pancreas, and prostate.
The potential promise of gene therapy helping millions of people (14.5 million Americans have cancer reports the National Cancer Institute) has spurred companies and universities to seek new gene therapies, The New York Times article “Companies Rush to Develop ‘Utterly Transformative’ Gene Therapies” says.
This flurry of activity could become an excellent money-making opportunity for investors, although the first two gene therapies in Europe treated only a few people and were a commercial and scientific disappointment.
If the FDA accepts the recommendation of its panel of outside experts, a decision that could made by October, the newest gene therapy treatment would treat far more people than the treatments approved by European health authorities. In short, scientific advances in gene therapy could spur a whole new wave of financial opportunities.
“After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as hemophilia,” Reuters reports. “They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.”
The financial opportunities of gene therapy have become so promising that large pharmaceutical companies like Bristol-Myers Squibb and Pfizer are now competing or partnering with gene therapy companies.
Of the 68 gene therapy companies, a number that doesn’t count the pharmaceutical companies, slightly more than half are American and about one-third are European, reports CrunchBase. Twelve of those companies have gone public.
Gene Therapy Companies to Watch
If you’re thinking about investing in a gene therapy, you should, of course, seek information about the 68 gene therapy companies, what gene therapy drugs and treatments they’re studying, the progress of their research, and their business plans.
First, let’s breakdown the science behind it all.
Basically, all cells have four parts, including the DNA part that contains genes. Missing or malfunctioning genes can cause diseases, so inserting healthy genes into the cells could cure those diseases.
Now, let’s look at some companies that are involved in gene therapy research and might be an investment opportunity for you. They’re listed alphabetically.
* Audentes Therapeutics: This company is researching four gene therapy treatments. One of the treatments would be for life-threatening arrhythmias, one for reducing patients’ risk of neurological damage, and two for muscles.
* AveXis: The price of shares in the company’s stock has increased nearly 70 percent in 2017, reports Investing News Network. It is researching a gene treatment therapy for spinal muscular atrophy.
* BlueBird Bio: The Massachusetts company has raised more investment money than any other gene therapy company, according to CrunchBase. It also has already tested a gene therapy drug on sickle cell disease.
* Editas Medicine: This gene therapy company is researching “transformative” medicines for cancer as well as diseases of the blood, eye, liver, lung, and muscles, reports Investing News Network.
* Intellia Therapeutics: The company’s stock price is up 29 percent in 2017. It plans on testing its gene therapy drugs in people with hepatitis B and transthyretin amyloidosis.
* Kite Pharma: The Santa Monica, Calif., company plans to ask the FDA to approve its gene therapy treatment for lymphoma.
* Novartis: This Swiss pharmaceutical company’s gene therapy treatment for B-cell acute lymphoblastic leukemia was the one that was approved by the FDA panel in July. It also plans to ask the FDA to approve the same treatment for adults who have a type of lymphoma.
* Spark Therapeutics: The Philadelphia company might have a gene therapy treatment for vision loss caused by two specific diseases approved as early as this coming January, so it’s worth examining further.
* UniQure: The Netherlands-based company developed a gene therapy that was approved by the European Union. The therapy treats a genetic disease that clogs the blood with fat.
Remember to do your own research before putting down an investment, and keep a sharp eye on scientific reports to gauge each company’s performance.
Regards,
Ethan Warrick
Editor
Wealth Authority
