Avrobio moves forward with gene therapy candidate

By The Science Advisory Board staff writers

May 14, 2020 -- Avrobio released preclinical data for AVR-RD-03, lentiviral gene therapy for Pompe disease, at the American Society of Gene and Cell Therapy annual meeting on May 14.

To develop AVR-RD-03, Avrobio leveraged its lentiviral vectors with glycosylation-independent lysosomal targeting-tag technology, which consists of a short peptide sequence linked to a therapeutic protein. The platform is designed to enhance uptake in key tissues and provide a potent transgene promoter to boost protein production.

In the study, Avrobio researchers studied 10 different lentiviral vectors with therapeutic transgenes in mice. Each vector was successful in increasing expression of the GAA, the gene responsible for Pompe disease. The leading vector had minimal off-target effects.

Based on the data, Avrobio will move forward with an investigational new drug (IND)- enabling proof-of-concept study to be completed by the end of the year.

Thermo Fisher to double viral vector manufacturing capacity
Thermo Fisher Scientific is expanding its viral vector development and manufacturing services with construction of a new commercial manufacturing site...
Caribou, MaxCyte enter CRISPR license agreement
Caribou Biosciences and MaxCyte have announced a clinical and commercial license agreement for the development of Carbiou's CAR-T cell therapy program.
Fujifilm Diosynth partners with Oxgene for gene therapy
Fujifilm Diosynth Biotechnologies has partnered with Oxgene to deliver gene therapy products with a 25% reduction in production time, the company reported...
MilliporeSigma expands viral and gene therapy capacity
MilliporeSigma is expanding its BioReliance viral and gene therapy service offering with a second commercial manufacturing facility in Carlsbad, CA. The...

Copyright © 2020 scienceboard.net


Conferences
Microscopy and Microanalysis Meeting
July 31 - August 4
Portland, Oregon United States
Glasgow International Health Festival
August 24-27
Glasgow, Glasgow City United Kingdom
Pharma Competitive Intelligence Conference and Exhibition
September 21-22
Newark, New Jersey United States
BioProcess International (BPI) Conference
September 27-30
Boston, Massachusetts United States
Connect
Science Advisory Board on LinkedIn
Science Advisory Board on Facebook
Science Advisory Board on Twitter