3 Gene Therapy Stocks Investors Can Bet On In Rest Of 2018

Innovation holds the key in the pharma/biotech sector and most companies have been diverting their resources to develop gene therapy candidates in their goal of finding a cure for difficult and incurable diseases. In the first half of 2018, most pharma/biotech bigwigs and also a number of smaller companies scurried for a lucrative catch in the space either through licensing deals or acquisitions. 

In particular, the FDA approval in December 2017 of Luxturna, a new gene therapy for the treatment of children and adult patients with an inherited form of vision loss that may result in blindness, sparked interest in the space. 

What is Gene Therapy?

Gene therapy enables to mitigate the adverse effects of a malfunctioning disease-causing gene. The therapy intends to add a functional gene in a person's system, replacing the malfunctioning one to cure diseases. The gene is delivered to a cell though a carrier known as a vector. The therapy has a different approach from the traditional drug therapies as the underlying cause can be treated with it, unlike symptoms.

Gene therapy is set to become one of the most promising spaces in the volatile biotech sector, which continues to grapple with competitive pressure and pricing woes. The promising therapy is being evaluated for varied diseases such as hemophilia, Duchenne muscular dystrophy (DMD), Parkinson's disease and cancer among others.

Recent Activity in the Space

The year started with the acquisition of AveXis, Inc by Swiss giant Novartis (NVS - Free Report). AveXis’ lead product candidate, AVXS-101, is being evaluated as a one-time gene replacement therapy for spinal muscular atrophy, a disease that results in early death or life-long disability with considerable healthcare costs. Novartis is trying to develop a gene therapy portfolio. In January 2018, Novartis signed a license and commercialization agreement for the development and commercialization of voretigene neparvovec (brand name Luxturna in the United States) outside the United States with Spark Therapeutics.

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