Gene Therapy Becomes Science Fact

Another Negative-EV “Steal” for Investors

A $5 Billion Opportunity in Biotech

Below is the most recent issue of Biotech Frontiers, from analyst Erez Kalir.  In this issue, Erez provides a recommendation, as well as the “3 Best Buys,” a feature that highlights the three stocks that new investors can purchase now. In addition to hosting Porter & Co. Biotech Frontiers on our website, we also make it available as a downloadable PDF. Subscribers can access this issue as a PDF on the “Issues & Updates” page here. If you have any questions, please give Lance, our Director of Customer Care, and his team a call at 888-610-8895 or internationally at 443-815-4447. Again, thanks for being part of Porter & Co.

By the time Corey Haas was seven years old, the retinal disease he was born with – a rare, genetic condition called Leber congenital amaurosis – had already stolen much of his eyesight. He clung to his parents, walked with a white cane, and learned braille so that he could read. Already legally blind, he was expected to lose his vision entirely.

Then, shortly after his eighth birthday, his parents were able to enroll him in a pioneering gene-therapy trial at Children’s Hospital in Philadelphia. There, physicians injected a corrected version of his defective gene, RPE65, directly into his retina. RPE65 is a critical enzyme found in the retinal pigment epithelium, a layer of cells that supports photoreceptors in the eye. The gene plays a crucial role in the visual cycle by catalyzing a molecular conversion sequence that enables photoreceptors to detect light. Mutations in RPE65 disrupt this cycle, leading to progressive vision loss, as was happening to Corey.

For Corey, replacing his defective REP65 gene with a functional one proved to be a miracle cure. Within weeks of his treatment, he experienced dramatic improvements in his vision. And in less than a year, before his ninth birthday, he could ride a bike, play baseball, and read books in low-light conditions – activities that had been impossible for him before. The experimental therapy that changed Corey’s life is now a therapy called Luxturna that has been approved by the Food and Drug Administration (“FDA”). 

Ever since Austrian monk Gregor Mendel – widely recognized as the “father of genetics” – did his pathbreaking experiments on the mechanics of heredity in pea plants in the 1860s, gene therapy has captivated the imagination of scientists, physicians, and writers. For a long time, from H.G. Wells’ 1896 classic The Island of Dr. Moreau to Michael Crichton’s 2006 Next, gene therapy was more science fiction than science fact. But today, as Corey Haas’s story highlights, medicine is finally beginning to deliver on the promise of using genes to deliver cures. 

Our recommendation this month is a company at the vanguard of this effort. Although it didn’t pioneer the therapy that cured Corey Haas, it’s a leader in gene therapy with several similarly transformative medicines in its pipeline. Best of all, this company trades at a negative enterprise value (“EV”), making it a compelling value – beyond its scientific promise.

Gene Therapy for a Progressive Neurodegenerative Disease