Genetics

GenSight Biologics, a France-based biotech company, is developing a new gene therapy specifically for a debilitating eye disease condition. Currently in phase III trials, the treatment is designed to both prevent and restore vision loss in patients.

Gary Ruot is going blind. Thanks to a genetic condition called Leber’s Hereditary Optic Neuropathy (LHON), the 17 year old is losing his vision, just like thousands of others who carry the mutation in their genome. There is currently no approved form of treatment available for LHON—but that might not be the case for much longer. GenSight Biologics (NASDAQ:GNST, EPA:SIGHT), a France-based biotech company, is developing a new gene therapy specifically for this condition: GS010.
Currently in phase III trials, the treatment is designed to both prevent and restore vision loss in those with LHON. If approved, GS010 has the potential to be a big money maker for the company: GenSight expects to market the one-time treatment for between $500,000 and $1 million, according to UKBusiness Insider.
The treatment involves injecting a virus carrying specific genetic material into the eyeball. In theory, the virus will prevent optic nerve cells from atrophying—and perhaps even restore those already damaged.
That means Ruot, a participant in the trial, may one day see as well as he did before. So too might thousands of others across the globe. While only 35,000 people are diagnosed with LHON worldwide, many more carry the genetic mutation—which means they could develop the condition at any time, or pass it down to their children.
The market potential is therefore substantial for GenSight, particularly considering that no competitor products are currently available. “This is a devastating disorder that has no proven therapy of any great efficacy,” Nancy Newman, a principal investigator in the GenSight trial, told MIT Technology Review.
Approval would also be a big win for gene therapy in general. Research in this area slowed after 1999, when an 18 year old participating in an experimental trial passed away.
Nevertheless, interest has resurged as of late: there has been a lot of recent excitement over the potential of genetics in personalized medicine, thanks to technologies like CRISPR-Cas9.


To date, however, not a single gene therapy has received FDA approval—and it’s this that may give some investors pause. There’s also no guarantee that GS010 will meet its endpoints in the two ongoing phase III trials.
So while GenSight’s research is certainly exciting, the potential payoffs, for both patients and investors, remains to be seen.
Don’t forget to follow us@INN_LifeScience for real-time news updates.

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

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