4 Gene Therapy Companies to Watch

- December 3rd, 2018

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

In 2018, investors have remained eager to find new opportunities in the life science sector, with gene therapy companies piquing the interest of savvy traders.

The work done in this sector is some of the most innovative, with therapies designed to directly treat patients with serious ailments like cancer or Alzheimer’s disease.

Below the Investing News Network takes a closer look at four gene therapy companies with interesting developments in 2018 and beyond. The stocks below are listed from largest to smallest in order of market cap. Numbers were current as of December 3, 2018 at market close, and all companies had market caps below $500 million at that time.

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1. Nightstar Therapeutics (NASDAQ:NITE)

Market cap: $436.97 million; current share price: $15.32

Nightstar Therapeutics 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 the company has faced a downward trend in its share price.

The company is focused on genetic therapies for rare diseases, a promising niche area of genetic work. Its most advanced candidate, NSR-REP1, is being evaluated to treat choroideremia, which is a rare genetic retinal disorder with no current treatments.

In March, the company began a STAR Phase 3 trial to study NSR-REP1 in patients with choroideremia. In its Q3 report, Nightstar said completion of enrollment for this trial is anticipated in the first half of 2019. Its other anticipated 2019 milestones include updates on its work regarding NSR-RPGR for X-linked retinitis pigmentosa.

2. Voyager Therapeutics (NASDAQ:VYGR)

Market cap: $355.62 million; current share price: $11.65

Voyager Therapeutics is a clinical-stage gene therapy company focused on treating severe neurological diseases. It is doing this by advancing the field of adeno-associated virus gene therapy.

The company announced a collaboration with AbbVie (NYSE:ABBV) in February under which 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 in correlation with neurological diseases.

In November, Voyager shared an update on VY-AADC, a candidate for treating advanced Parkinson’s disease. The company said that one-time treatment with VY-AADC demonstrates durable improvements in motor function at 18 months and beyond from an ongoing Phase 1b trial. Also recently, Voyager presented preclinical data for its Huntington’s disease and amyotrophic lateral sclerosis programs.

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3. Kadmon (NYSE:KDMN)

Market cap: $270.95 million; current share price: $2.48

Kadmon is a biopharmaceutical company with a pipeline focused on inflammatory and fibrotic diseases. It has a portfolio of small-molecule Rho-associated coiled-coil kinase (ROCK) inhibitors, with its lead ROCK inhibitor candidate being KD025. 

KD025 is being studied in ongoing Phase 2 clinical trials in chronic graft-versus-host disease (cGVHD), idiopathic pulmonary fibrosis and moderate to severe psoriasis. The company’s latest results came in October, when it announced the dosing of the first patient in a pivotal clinical trial of KD025 in cGVHD.

In its Q3 report, released in November, the company also highlighted that the US Food and Drug Administration has granted breakthrough therapy designation to KD025 for the treatment of cGVHD. 

4. Tocagen (NASDAQ:TOCA)

Market cap: $254.6 million; current share price: $13.65

Rounding out this list of gene therapy companies is Tocagen, which works in the development of genetic therapies with the goal of treating cancer.

Its lead product candidate, Toca 511 & Toca FC, is being investigated for the treatment of recurrent and metastatic cancers with significant unmet medical needs. Tocagen also has an anti-PD-L1 product candidate, Toca 521, that is currently in preclinical development.

The last few months have been busy for Tocagen. In August, it announced that a Toca 5 pivotal Phase 3 trial evaluating Toca 511 and Toca FC in patients with recurrent high-grade glioma would continue without modification. Then, in September, it completed enrollment of 380 patients in an ongoing Toca 5 Phase 3 trial about three months ahead of schedule. Planned second interim and final analyses for Toca 5 are anticipated in 2019, the company said in November.

Which gene therapy companies do you think are the most auspicious in 2018?

This is an updated version of an article originally published by the Investing News Network in 2016.

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

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

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