Broadening Gene Therapy's Reach With Novel Capsids

Weak cell specification and required high dosing are just some of the limitations of current capsids used for gene therapy approaches. As the potential for gene therapy is realized in a broader set of disorders, the need for more specific capsids will increase.

LogicBio Therapeutics is tackling this challenge with their novel capsid discovery platform, SAVVY, that they are developing in partnership with the Children's Medical Research Institute in Australia. In addition, the company is working with multiple partners to develop novel therapies for several indications using SAVVY and their proprietary gene editing technology, GeneRide.

The company recently announced that the first patient has been dosed with LB-001 in the phase 1/2 SUNRISE trial (NCT04581785) for methlymalonic acidemia (MMA), which utilizes the GeneRide technology platform. The technology removes the need for exogenous nucleases and promoters associated with an increased risk of immune response and cancer. LB-001 is designed to insert a functioning copy of MMUT to the albumin locus to achieve sufficient, lifelong MMUT expression in the liver via engineered recombinant adeno-associated virus vectors (rAAV-LK03).

To learn more about the platforms and therapeutic targets they could serve, GeneTherapyLive spoke with LogicBio chief scientific officer, Mariana Nacht, PhD.