4 Gene Therapy Stocks to Watch

- March 22nd, 2018

What gene therapy stocks to watch should be on investors’ radar? Here are four promising companies to consider looking at in 2018.

At the near-four month mark of 2018, investors remain eager to find new opportunities—and various industries in the life science sector are certainly piquing the interest of savvy investors.

Take the genetics industry, for example, where work done companies in this sector is some of the most innovative, with therapies designed to directly affect patients treatment against serious diseases like cancer or Alzheimer’s disease.

Here, the Investing News Network (INN) takes a closer look at four companies in the genetics space with interesting developments from 2017 that could become catalysts in 2018 and beyond. The companies below are listed in order of market capitalization, from largest to smallest, with all numbers and figures current as of March 22, 2018 at market close.

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


1. Voyager Therapeutics (NASDAQ:VYGR)

Market cap: $561.63 million; current share price: $20.84

This clinical-stage gene therapy company, Voyager is focused on developing treatments for severe neurological diseases. It’s doing this through advancing the field of adeno-associated virus (AAV) gene therapy, which are small viruses with a genome on a single stranded DNA.

A collaboration between Voyager and AbbVie (NYSE:ABBV) was announced in February, where the companies will develop and commercialize vectorized antibodies against tau-related diseases, such as Alzheimer’s disease and other neurological diseases. Tau is a healthy protein in the brain, but with diseased brains the tau is altered which correlates with neurological diseases.

Looking towards the rest of 2018, Voyager has a packed year ahead. One of its biggest challenges comes in the form of a key Phase 2-3 program set to begin patient dosing in the second quarter of the year. This study will evaluate the company’s recently acquired candidate VY-AADC treating advanced Parkinson’s disease.

Partial results and developments from these studies could prove key to continuing Voyager’s momentum gained at the end of 2017 after it recovered from the decline caused by the breakup with Sanofi. Now Voyager’s slate is full, where it will need to prove itself to investors throughout 2018.

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


2. Nightstar Therapeutics (NASDAQ:NITE)

Market cap: $405.77 million; current share price: $13.75

Nightstar publicly launched on the NASDAQ late last year at an asking price of $14 per share. On its first day of trading, the company finished the day valued at $24 per share. However, since that initial high of its IPO, the company has faced a downward trend in its share price, declining 45.33 percent at the end of 2017.

The company is focused on genetic therapies for rare diseases, a promising niche area of genetic work. Their most advanced candidate, NSR-REP1, is being evaluated to treat Choroideremia (CHM), which is a rare genetic retinal disorder.

Steady updates from the observational study on NSR-REP1 would provide a solid look to investors on the development of one of the two trials Nightstar will be working on throughout 2018 and beyond.

Although the company hasn’t had much recent news, in March it did initate the STAR Phase 3 trial to study NSR-REP1 in patients with choroideremia. Which is a rare degenerative genetic retinal disorder, with no current treatments.

3. Kadmon (NYSE:KDMN)

Market cap: $343.67 million; current share price: $4.37

Kadmon is a biopharmaceutical company with a pipeline focused on inflammatory and fibrotic diseases. KD025 is involved in three Phase 2 trials for three indications, it’s a small molecule inhibitor which promoted T regulatory cell function in the preclinical studies to help rebalance immune response.

In February, the company announced positive Phase 2 results from it’s KD025 study for chronic graft-versus-host disease (cGVHD). The results showed about 70 percent of patients showed a positive response to the drug candidate and most were able to reduce or discontinue other medication they were using for the disease.

As part of its 2017 financial report, Kadmon CEO, Konstantin Poukalov said in the press release, “We are in a strong financial position to continue our programs in these and other diseases, with an emphasis on defining pathways to regulatory approval.”

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


4. Tocagen (NASDAQ:TOCA)

Market cap: $246.43 million; current share price: $12.44

Tocagen works in the development of genetic therapies with the goal to treat cancer. As of November 2017, Tocagen’s pipeline included three candidates, with one treating recurrent high-grade glioma at the critical phase 3 stage of development.

To finish the year Tocagen shared with its investors the company’s addition to the highly regarded NASDAQ Biotechnology Index (NASDAQ:NBI), one of the leading exchange-traded funds (ETFs) of the industry. In the company’s Q4 and fiscal 2017 financial results, Tocagen stated it expects to report Toca 6 activity and safety data, complete enrolment for the Phase 3 Toca 5 trial and advance the retroviral replicating vector platform.

The company will present preliminary Phase 1 data from its Toca 6 trial at the American Association for Cancer Research meeting, in April. Prior to that,the company received orphan medical designation for Toca 511 and Toca FC to treat glioma by the European Medicines Agency.

Don’t forget to follow us @INN_LifeScience for real-time news updates.

Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.

2018 Genetics Market Report


Find out what to watch for in the world of genetics investing.


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12 responses to “4 Gene Therapy Stocks to Watch

    1. Hi Dale,
      It will be interesting to see if Propanc (OTCQB:PPCH) receives orphan designation for PRP, its therapy candidate for pancreatic cancer. The company filed that application in early September of this year. You’re right, it could be one to keep an eye on too!

    1. Hi Dale,
      It will be interesting to see if Propanc (OTCQB:PPCH) receives orphan designation for PRP, its therapy candidate for pancreatic cancer. The company filed that application in early September of this year. You’re right, it could be one to keep an eye on too!

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