Thanks to advances in technology and gene sequencing, gene therapy has gone from science fiction to science fact. By either adding new genes to fight faulty ones, replacing/editing missing/broken prices or actually “turning off” the genes causing problems, gene therapy has the potential to change the game when it comes to biotech stocks and healthcare.
That’s great news, as there are more than 10,000 different disorders and diseases caused by faulty genes.
With the FDA launching several fast-track programs for gene therapies last summer, the biotech stocks that specialize in these cutting-edge treatments could see their drugs hit the market that much faster. And yet, the recent market sell-off has caused many of the biotech stocks looking at gene therapy to crater. Given their game-changing potential, these days many stocks in the sector could be considered bargains.
Some more than others.
Which gene-therapy biotech stocks have great potential to soar over the long haul? Here are three stocks to buy that could do just that.
Gene Therapy Stocks to Buy: CRISPR Therapeutics (CRSP)
Clustered Regularly Interspaced Short Palindromic Repeats is a mouthful to say, which is why scientists have shortened it to just CRISPR. CRISPR is the latest method of gene editing and offers cheaper, simpler and faster slicing and dicing of genes. As its name implies, CRISPR Therapeutics (NASDAQ:CRSP) uses the technique.
CRSP stock is targeting blood diseases such as beta-thalassemia and sickle cell disease- which are caused by the same mutation. The beauty of this is that the biotech has been able to move ahead at the same time for both indications. Partnering with biotech giant Vertex Pharmaceuticals (NASDAQ:VRTX), CRSP’s lead candidate — CTX001 — started phase 1 clinical trials this past February. This trial marks the first time in history that a human trial for a CRISPR-based product has been conducted.
Naturally, a lot is riding on the trial- especially with the drug winning FDA fast track status. If results are even somewhat positive, CRSP stock could surge higher.
But CTX001 isn’t the only drug in CRSP’s arsenal. The firm is working one several oncology products as well as new gene editing therapies for muscular dystrophy and cystic fibrosis. These drugs could provide plenty of upside as well down the road.
In the end, if you’re looking for biotech stocks to buy that are looking at gene therapy, CRISPR Therapeutics could be a major star.
uniQure NV (QURE)
When it comes to biotech stocks, uniQure (NASDAQ:QURE) is gene therapy royalty. That’s because the firm actually created and launched the very first successful gene therapy back in 2012. However, due to the cost of the drug, it was never prescribed. But QURE has turned that successful approval into a platform for further successful development.
This includes the biotech stock’s latest work for hemophilia. QURE has seen great success with its gene therapy program for the blood disorder. After seeing amazing initial results, QURE has moved its top hemophilia medication — AMT-061 — into phase I/IIb trials. This news sent the clinical stage biotech stock up more than 34%. Given its past history of navigating the gene therapy waters to approval, QURE could have another hit on its hand.
Elsewhere, the firm has started trials for the first gene therapy targeting Huntington’s disease and has gene therapies for congestive heart failure in pipelines. All of these are much more “popular” issues and should help QURE actually see prescription growth if successful.
Like most clinical-stage biotech stocks, QURE is a gamble. But it’s a more calculated risk than most given its history and how great its previous results were.
Voyager Therapeutics (VYGR)
When it comes to clinical stage biotech stocks, it pays to look at partnerships. For gene therapy play Voyager Therapeutics (NASDAQ:VYGR), partnerships include biotech giant AbbVie (NYSE:ABBV), neurological specialist Neurocrine Biosciences (NASDAQ:NBIX) and major pharma stock Sanofi (NASDAQ:SNY). All three of those major players have provided VYGR with some major cash infusions to develop its technology and gene therapy applications. Most clinical biotech stocks would kill to have more than $360 million in cash on their balance sheets.
That cash will provide it plenty of working capital to develop its lucrative gene therapy portfolio.
And lucrative it will be. VYGR is targeting Parkinson’s disease, Amyotrophic Lateral Sclerosis (ALS), Huntington’s disease and Alzheimer’s disease. These are some of the hardest diseases to crack and winners here will be massive achievements. It seems that Voyager may just get there.
So far, results for the firm’s tech have been pretty positive, which could explain all the major partnerships. Back in March, Voyager announced that its initial trial for VY-AADC demonstrated improvement in clinical measures for Parkinson’s Disease. This success prompted VYGR to start phase II trials — with results coming in mid-2020. Meanwhile, the firm is moving forward with initial trials with its other partners and developing a robust pipeline. With ample cash, VYGR has plenty of time to get these therapies through testing. Fast track designation from the FDA doesn’t hurt either.
All in all, VYGR represents a great gene therapy play thanks to its leading partners. Clearly, they see the good in the biotech stocks
At the time of writing, Aaron Levitt was long CRSP and VYGR stock