An Epic biotech week Notes: Gene Therapy
I have broken down my genetics watch list into 3 separate lists for Gene Therapy, Genetic Editing and RNA technologies.
QURE – This company has had a very wild ride. It started when their Hemophilia B data bested that of ONCE. That sent the stock into a frenzy as it was undervalued. It was the underdog after all. Then it peaked after the frenzy drove it to over $70 on an announcement they were looking for a buyer. It went to the complete opposite extreme finally bottoming in the $30's again. Now I think it is more fairly valued for the limited data it has from many of its programs. I do not believe a big cap biotech will go after such early stage assets. Overall, I think its a very solid pick for a gene therapy company for anyone interested.
RGNX – This is a company I have been in and out of for years. It got way to carried away on the frenzy into their first data read outs for wet AMD. Since then, the company has come back down to earth and I got back in. They have a very big indication available to them with wet AMD and Diabetic Retinopathy. They had some challenges lately and of no fault of their own. Their partner for Zolgensma got jammed up by the FDA. Now they have an issue with one of the companies that supplies their injectors. I think its held up very well in spite of all the challenges.
KRYS – This is probably one of my favorites of late. The data from their topical gene therapy for skin disorders has exceeded all my expectations. They have the additional bonus of being cheap on valuation compared to other companies in this space.
BOLD- I really like this company, but I felt it was way too expensive. I sold completely out of it and replaced it with RGNX. I still think it is very expensive for the limited development it has so far. Their DMD program could really drive the value, but it is still in preclinic. It is now up against a bunch of competitors. The programs it has in later stage of development are very small indications.
FOLD – This on got onto my list recently. It had been an old school enzyme replacement therapy company. They recently jumped into the gene therapy game. This is now the company I had hoped BioMarin would have become. A blending of the old school ERT that works with the promise of the future in gene therapy. They don't have much data on their gene therapy programs yet. I don't think the company is too expensive. It trades at about 11x its 2019 projected revenues. That isn't bad based on its sales, and it offers a nice pipeline on top of that.