Gene editing refers to the process of insertion, deletion or replacement of DNA at a specific site in the genome of an organism or cell. Investors have a few opportunities to gain exposure to companies that operate in this field, two of which analysts at SunTrust Robinson Humphrey hold a bullish stance on, including Crispr Therapeutics AG CRSP, and Editas Medicine Inc EDIT.
SunTrust's Peter Lawson upgraded Crispr Therapeutics' stock from Hold to Buy with a price target boosted from $16 to $45. The analyst upgraded Editas Medicine's stock from Hold to Buy with a price target boosted from $17 to $45.
Crispr: Bullish Ahead Of Data Release
Crispr is expected to release its first clinical data for its beta thalassemia program in the middle of 2018 but the company already released preclinical data, Lawson said in a note. The preclinical data for the edited hematopoietic stem cell transplant, CTX-001, showed that edited patient samples achieved (β+γ)/α ratios of around 70 percent, which is superior to the 50 percent threshold typically seen in asymptomatic patients with beta thal trait.
The company's CTX-001 data was "encouraging" in sickle cell disease as biallelic editing achieved in 76 percent of erythroid colonies from healthy donors. This lead to a fetal a fetal hemoglobin to total hemoglobin ratio of 37 percent in those colonies, which is above the 30 percent that is needed for any clinical benefit.
Editas: Encouraging Data
Similar to Crispr, Editas has already released encouraging preclinical data from its eye program trial LCA10, the analyst said. The data showed productive editing of 50 percent of the CEP290 alleles in non-human primate photoreceptors and in human retinal explant which surpassed the therapeutic target of just 10 percent.
The company's genetic blood disease programs in sickle cell disease and beta-thalassemia are potential Investigational New Drug candidates in 2019. In the interim, Lawson said data from the natural history study for LCA10 are expected to be released in the middle of 2018 and will " help grasp deeper understanding of the disease, the range of visual acuity restoration required to show clinical benefit, guide design the trial assays and potentially identify patients for the Phase 1/2 trial."
Shares of Crispr Therapeutics were trading higher by more than 9 percent Tuesday and hit a new 52-week high of $38.87.
Shares of Editas Medicine also hit a new 52-week high of $36.20 and were trading higher by 0.4 percent on the day.
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