CRISPR gene editing stocks are higher today after an abstract published in Nature reported success in preventing genetic deafness in mice.
The abstract's findings suggest that protein--RNA complex delivery of target gene-disrupting agents in vivo is a potential strategy for the treatment of some types of autosomal-dominant hearing loss.
Scientists think it could work in humans but much there is much work to be done to first ensure the approach is safe.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and CRISPR-associated (Cas) gene editing technology is still in the early stages but offers huge potential in the field. There are three publicly traded biotech companies that are using CRISPR technology.
- Editas medicine (EDIT +7.5%) has a $1.2 billion market capitalization and a ~$835 million enterprise value after a recent equity offering. The lead asset is EDIT-101, which treats Leber Congenital Amaurosis type 10 (LCA10), a rare inherited eye disease. The company is on track for a mid-2018 investigational new drug application.
- CRISPR Therapeutics (CRSP +3.5%) has a $800 million market cap and an enterprise value of $550 million. CRISPR submitted a Clinical Trial Application (CTA) for CTX001 in ß-thalassemia and partnered with Vertex (VRTX) for the program this month. The Phase 1/2 trial of CTX001 is expected to begin in Europe in 2018.
- Intellia Therapeutics (NTLA +2.5%) has a $800 million market capitalization and a ~$430 million enterprise value. Intelia's lead program is partnered with Regeneron (REGN) treating Transthyretin Amyloidosis (ATTR), a neurodegenerative disease. Based on top-line data from Intellia's first exploratory non-human primate (NHP) studies and other research, the company may be able to select a human guide development candidate as early as the first quarter of 2018.
These three biotech stocks are preclinical and therefore highly speculative with an elevated risk/reward profile.
This week, the FDA approved its first gene therapy for an inherited disease. The FDA approved Spark Therapeutics' (ONCE) one-time gene therapy for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.