William Blair analyst Raju Prasad initiates coverage on Generation Bio (NASDAQ: GBIO) with a Outperform rating.
The analyst comments "Exploring the clinical potential of nonviral gene therapy. While AAV gene therapy has produced impressive clinical data to date, there are some notable drawbacks to this approach. These include: 1) the size of a transgene that can be used (less than 5 kilobases of genetic cargo); 2) potential for waning of durability of effect as evidenced in BioMarin’s Roctavian long-term data in hemophilia A; 3) nonoptimal transduction efficiency, which can result in the need for higher doses and subsequently require more manufacturing lots; and 4) immunogenicity, including both preexisting neutralizing antibodies against the AAV vector and increasing the need for prophylactic steroid use with initial delivery. Generation Bio’s technology value proposition is a redosable, titratable gene therapy that allows for a larger genetic payload, tissue specificity, and a capsid-free manufacturing process, with the potential to improve on both AAV gene therapy and biologic offerings."
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