Selecta Biosciences validates tech that could improve gene therapies

By The Science Advisory Board staff writers

January 6, 2021 -- Selecta Biosciences has achieved preclinical data validating its ImmTOR platform to enhance the efficacy, safety, and durability of adeno-associated viral (AAV) vector gene therapies. The company plans to present these data at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in May.

In the study, the company found that co-administration of an AAV vector and ImmTOR, a selective immune tolerance compound, in nonhuman primates enabled higher and more durable transgene expression, as well as robust inhibition of anti-AAV8 immunoglobulin G (IgG) and neutralizing antibodies.

The researchers administered a single intravenous infusion of a widely used reporter gene transgene -- recombinant adeno-associated serotype eight capsid directing expression of a transgene encoding secreted embryonic alkaline phosphatase (AAV8-SEAP). It was administered alone or co-administered with ImmTOR.

These findings suggest the potential for dosing lower levels of AAV gene therapies, thereby improving patient safety and lowering costs. Moreover, currently AAV gene therapies cannot be redosed due to the formation of neutralizing antibodies to the AAV vectors. The study found that ImmTOR mitigated the formation of neutralizing antibodies, potentially allowing for redosing.

Selecta, in partnership with Asklepios BioPharmaceuticals, expects to initiate a phase I clinical trial of MMA-101 and ImmTOR for patients with methylmalonic acidemia in the first half of 2021, with preliminary data expected by the end of 2021.

Regulatory Roundup: Gene therapies, bispecific antibodies lead new approvals
Welcome to Regulatory Roundup, a new Science Advisory Board feature that tracks recent regulatory activities in the life sciences industry. Recent approvals...
New COVID-19 immunogen could help with early diagnosis
A new assay that detects the unique SARS-CoV-2 orf8 protein in the sera of COVID-19 patients in the early stage of the infection is a strategic step in...

Copyright © 2021

Science Advisory Board on LinkedIn
Science Advisory Board on Facebook
Science Advisory Board on Twitter