[Video] New Gene Therapy to Cure Blindness?
Dear Wall Street Daily Reader,
Several weeks ago, I alerted you to the resurgence of interest in gene therapy stocks by providing you with three compelling and undeniable data points.
Of course, not everyone is persuaded by data. Some investors need to “see” the trend before they’ll believe it and buy it.
Well, ask and ye shall receive. Because in the case of gene therapies, you can literally see the potential…
I Can See Clearly Now
Remember, gene therapy is the cutting-edge area of biotech that involves one-time treatments designed to replace faulty genes with healthy ones. By doing so, the potential exists to cure hundreds of genetic diseases including muscular dystrophy, hemophilia… and blindness!
That’s right, in the last two weeks, Paris-based GenSight Biologics reported the first partial recovery of vision for a patient with a degenerative eye disease that causes blindness, known as retinitis pigmentosa (RP).
Don’t believe it? Watch this video and you can see it:
Per the write-up in Nature Medicine, the investigators said, “The patient spontaneously reported identifying crosswalks and he could count the number of white stripes. Subsequently, the patient testified to a major improvement in daily visual activities, such as detecting a plate, mug or phone, finding a piece of furniture in a room or detecting a door in a corridor.”
It’s worth noting that the restoration of vision involved two parts: administering the gene therapy via a single intravitreal injection to a patient’s worse-seeing eye, and a pair of special detecting goggles that sends pulses of light to the retina in real time, essentially amplifying the signal.
For anyone following the RP space, you might know that another disruptive company exists, Second Sight Medical Products, Inc. (EYES). This company has demonstrated an ability to restore partial vision to RP patients using special goggles, too.
But unlike GenSight, Second Sight exclusively uses a medical device. It combines a retinal or cortical implant with special goggles — so instead of trying to repair the genetic defect, it’s using technology to bypass it.
Obviously, a formerly blind patient couldn’t care less how their vision is restored.
From an investment standpoint though, a single, injectable gene therapy that corrects the underlying genetic mutation holds the potential to be safer and much more profitable.
More Data to Come
As we all know, biotech investing is all about the data. And a single, positive patient response isn’t enough to win drug approval, or for that matter, prove that a drug or therapy works.
But nonetheless, it is a promising sign.
In GenSight’s case, the man in the video above was one of six patients enrolled in its Phase 1/2 dose escalation clinical trial, whereby the company administers increasing doses of the gene therapy.
The fact that the company witnessed a partial response so early (i.e., at such a low dose) is all the more encouraging. It bodes well for the expansion of the study into additional patients at higher doses, which is set to resume now after being put on hold because of the pandemic.
Bottom line: Keep an eye on (no pun intended) gene therapy companies like GenSight as well as others that I’ve brought to your attention, including uniQure N.V. (QURE) and REGENXBIO Inc. (RGNX).
All it takes is a few more positive patient outcomes and the sector promises to become red-hot once again — and ignite some of the market’s next high-flyers.
Ahead of the tape,
Editor and Founder, Trend Trader Daily