Innovation has always been the mantra when it comes to biotech stocks. And as such, the biotech sector has been the key driver for new medicines and therapies over the last decade or so. But we haven’t seen anything like this before. Gene therapy is quickly becoming the technique du jour for curing orphan and rare diseases.
Over 10,000 diseases and disorders are caused by faulty genes. That is, either whole or part of a gene is defective or missing from birth, or that a gene has mutated during adult life. In a nutshell, gene therapy is fixing those problems by either adding new genes to fight the faulty ones, replacing/editing missing/broken prices or actually “turning off” the genes causing problems. The tech to do this has finally hit the critical mass stage and since last summer, the FDA has approved three gene therapy applications.
That could increase further as the FDA recently unveiled a fast-track program for gene therapies for the various orphan and rare diseases. That program comes with fewer requirements for trials.
For the biotech stocks operating in the sector, this fast-track process can be a godsend and quicken their ability to launch this lifesaving- and expensive- medicines. With that, here are five gene therapy biotech stocks playing the future of medicine.
Gene Therapy Biotech Stocks To Buy: Sarepta (SRPT)
There’s no denying that Sarepta Therapeutics (NASDAQ:SRPT) is very expensive — even when looking at biotech stocks. SRPT has managed to return more than 140% year-to-date. That was on top of the 102% it returned in 2017. That torrid run has been certainly justified.
SRPT won approval for its RNA-targeted Duchenne muscular dystrophy (DMD) therapy — Exondys 51 — last year. Muscular dystrophy is a serious ailment that affects roughly 200,000 kids each year. Before Exondys 51, there really wasn’t many reliable treatment options for the disease. While it doesn’t fully cure the condition, it does slow or prevent the progression of DMD. That can improve the quality of life for many patients. And so far, sales of the drug have reached blockbuster status.
The drug generated $154.6 million in sales for the full-year of 2017. Those are some pretty insane numbers for an orphan drug. And with that momentum, Sarepta believes that Exondys 51 should make it around $305 million this year. With a price tag of $300,000 per year, it can easily clear that.
But what is really exciting for SRPT is that the biotech stock is working on an experimental gene therapy muscular dystrophy that could potentially cure the disease outright. Early results from a small trial have been more than amazing. SRPT is still risky, but with one drug under its belt and another potentially coming down the pipeline, investors may want to take notice of the gene therapy play.
Gene Therapy Biotech Stocks To Buy: Gilead Sciences (GILD)
Not all gene therapy biotech stocks are high flyers, some are the bread and butter stocks in the sector. That includes Gilead Sciences (NASDAQ:GILD). GILD is no slouch in the biotech space and features plenty of drugs that generate real cash flows/profits for the firm. It also has one of the few approved gene therapies on the market.
After buying Kite Pharma for a cool $11.9 billion, GILD gained access to Yescarta — which is chimeric antigen receptor T-cell (CAR-T) therapy. Basically, CAR-T inserts genes into a patient’s own T-cells. That turns those cells into cancer-killing machines. And it works. In patients who have exhausted all treatment options, Yescarta has produced some real major results for lymphoma patients, with 51% of those given a single infusion and no traces of the cancer months later.
It really is a life saver.
And given its $373,000 price tag, it’s a saver for GILD as well. The biotech has struggled to fill holes in its line-up. But Yescarta could do it. There are enough end-stage cancer patients that provide GILD with around $2 billion in revenues annually from the drug. For Gilead, that could help it get over the pipeline hump and have it regaining its leadership position among biotech stocks.
Gene Therapy Biotech Stocks To Buy: Nightstar (NITE)
If Gilead is one of the sure thing biotech stocks in the gene therapy space, then Nightstar Therapeutics (NASDAQ:NITE) would be on the opposite side of the equation. NITE is very much a clinical-stage drug manufacturer. But that doesn’t mean that NITE isn’t worth buying. There’s plenty of promise in those clinical trials.
NITE’s main focus is on gene therapies for patients who suffer from rare inherited retinal diseases that would otherwise progress to blindness. The biotech has several candidates in various trials. It’s lead product targeting a condition called choroideremia recently begun phase 3 trials. Choroideremia is a rare disease that has no current treatments.
With that in mind and the promise of its already successful trails, the FDA gave NITE a Regenerative Medicine Advanced Therapy (RMAT) designation to the drug. The RMAT designation provides an expedited pathway for the advancement and approval of the drug. Nightstar will get to work with the FDA and the organization will basically give it the info on how to get approved.
It’s not a guarantee for approval, but its pretty darn close. So it’s no wonder why NITE stock jumped 26% when the announcement was made. However, given its pipeline of other therapies, decent sized balance sheet, and slow cash burn, NITE has plenty of potential to keep going.
Gene Therapy Biotech Stocks To Buy: uniQure (QURE)
There’s a good chance you’ve never heard of Dutch-based uniQure (NASDAQ:QURE). But the firm is gene therapy royalty. That’s because QURE created and launched the very first successful gene therapy back in 2012. The problem is and was, the cost for the drug was so expensive — over a $1 million per treatment — that it was only prescribed once. Needless to say, QURE sold its rights to the drug back in 2016 and took a beating on the creation expenses.
But as they say, when life hands you lemons, you make lemonade. And that’s just what uniQure is doing.
Having already created a successful gene therapy gives QURE a big head start over the competition. The firm has three drugs in development. Late-stage products for hemophilia have continued to advance and show positive results. Additionally, the biotech has plans to begin this year the first gene therapy clinical study targeting Huntington’s disease.
These targeted diseases — as well as gene therapies for congestive heart failure — are more “popular” than its previous attempt. That should help actually drive prescription volumes if/when they get approved. And with hefty price tags, QURE should be able to finally overlook the failure of its earlier gene therapy flop.
Investors certainly have. Shares of the biotech stock have nearly doubled this year. But those gains could be peanuts as the firm’s drugs advance through trials.
Gene Therapy Biotech Stocks To Buy: bluebird bio (BLUE)
When it comes to clinical-stage biotech stocks, de-risking is key. A great way to do that is to find those early stage drug producers that have partnered with larger drug companies. And that’s what you get with bluebird bio (NASDAQ:BLUE).
BLUE touts mega-biotech Celgene (NASDAQ:CELG) as a major backer to develop CAR-T therapies. It also announced a recent partnership with Regeneron Pharmaceuticals (NASDAQ:REGN). In addition to this partnership — which continues to see great results from early trials — bluebird features several other gene therapies in various stages of trials. This includes its compound LentiGlobin to treat transfusion-dependent beta-thalassemia (TDT). That drug was granted accelerated assessment by European regulators and bluebird expects it to submit marketing paperwork by the end of the year for approval. Analysts expect LentiGlobin to reach approval stages in the U.S. as well.
With that, BLUE has the potential to evolve from a developer into a commercial-stage biotech stock relatively soon. Given its partnerships with CELG, it usually trades at a premium to rivals. But after an interesting capital raise — because it really didn’t need the cash — shares have traded down to bargain levels given its long-term potential.
For investors looking to snag one of the best gene therapy biotech stocks, now could be a great time.
As of this writing, Aaron Levitt held a long position in GILD, BLUE and CELG