EnCapsid Therapeutics Inc., the pioneer in synthetic virology, has obtained results to show that its modular and versatile capsid modification technology can be used to engineer AAV vectors with enhanced therapeutic properties. By precisely introducing novel chemical functionalities onto capsids and subsequently conjugating performance-enhancing moieties, combined with the Company’s patented strategies to maintain the viral integrity during the process, EnCapsid developed engineered AAV vectors that outperformed expectations in animal models. The conclusion of the results was that the engineered AAVs created by using the EnCapsid technology showed increased infectivity and reduced immunogenicity in animal models.
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The results obtained from a third party that conducted the mouse studies show:
EnCapsid-AAV vectors, equipped with novel attachment handles, do not adversely affect the function of the virus.
EnCapsid-AAV modifiable vectors conserve tropism, increase effectivity and significantly reduce immunogenicity compared to wild-type virus.
EnCapsid-AAV vectors can be homogeneously labeled at predefined sites with activity-altering molecules, which significantly enhances its in vivo potency through increasing its circulation time.
“Until now, engineering new AAV vectors for gene therapy has been largely restricted to genetic approaches. The EnCapsid technology adds a new dimension to AAV engineering, where desired functions can be introduced into any existing AAV vector through precise chemical modification of the capsid with a wide variety of activity-modulating groups,” stated Abhishek Chatterjee, Professor at Boston College and Co-Founder of EnCapsid.
“By modifying AAVs, EnCapsid is ushering in the era of synthetic virology, which will deliver on the promise of gene therapy by enabling targeted, safer, delivery vehicles,” stated John Boyce, President, CEO and Co-Founder of EnCapsid as well as Co-Founder of Tiger Gene. “Over the next year, EnCapsid will build out its therapeutic pipeline utilizing engineered naturally occurring AAVs, as well as creating novel synthetic AAVs with properties to deliver a superior treatment alternative for patients,” Boyce concluded.