William Blair initiated coverage of LogicBio Therapeutics (NASDAQ:LOGC) with an “outperform” rating and $26 fair value estimate. The stock closed at $15.11 on Nov. 12.
LogicBio’s GeneRide platform is a promoter-less, nuclease-free genome-editing technology that harnesses homologous recombination to introduce site-specific integration of a gene to treat rare genetic diseases.
By using a novel set of liver-specific adeno-associated viral vectors and a therapeutic transgene that is preceded by a “self-splicing” 2A-peptide coding sequence, “GeneRide has shown meaningful transduction in preclinical models of liver diseases (targeting the albumin locus), with durable efficacy and reducing potential off-target effects that have become risk factors to consider with other gene therapy and gene-editing modalities,” writes analyst Raju Prasad.
He said GeneRide’s potential for providing specificity in genome editing without off-target effects has been validated across several preclinical models of liver disease, including hemophilia B, Crigler-Najjar syndrome type 1, and alpha-1 antitrypsin deficiency.
“Overall, we believe that the company has taken several measures to address potential safety issues associated with a one-time gene editing methodology that de-risks its platform and has the long-term potential to be curative for rare genetic diseases,” he added.
