Researchers will also present data in posters regarding the company's proprietary GeneRideTM platform, an AAV-based, promoterless, nuclease-free genome editing technology. Results from a mouse model indicate that administration of the mLB-001 genome editing therapy leveraging GeneRide improves disease phenotype and survival in methylmalonic acidemia (MMA). In addition, use of the GeneRide vector mLB-301 in a mouse model of Crigler-Najjar syndrome led to a substantial reduction of bilirubin levels, a key biomarker of efficacy in this disease.
'We are encouraged by the robust and diverse data being presented at this year's ASGCT meeting, highlighting the significant potential of both our sAAVy capsid and GeneRide platforms to support development of a new class of genetic medicines to treat serious and rare diseases including methylmalonic acidemia and Crigler-Najjar syndrome,' said
'The data to be presented show the promise of the AAV sL65 capsid as an outstanding delivery vector to overcome the current limitations of traditional AAV vectors including high dosage-related toxicity, high manufacturing costs and low translatability from mouse studies to human trials. Also, our GeneRide data demonstrate the broad applicability of this gene editing technology, in both diseases such as MMA where the liver is damaged and we observed selective advantage of corrected hepatocytes in the mouse model to produce therapeutic levels of MMUT enzyme, and in the mouse model of Crigler-Najjar where selective advantage is not required to see therapeutic benefit,' said
Details of the presentations at ASGCT are as follows:
Oral Presentation
Title: A Novel Liver-Tropic AAV Capsid sL65 Shows Superior Transduction and Efficacy in Humanized Mice and Non-Human Primates
Session Title: Development of AAV Capsid Variants
Presenting Author:
Date and Time:
Digital Poster Presentations
Title: Optimization of GeneRide-Encoding UGT1A1 Vector Improved Efficacy in a Mouse Model of Crigler-Najjar Syndrome
Session Title: AAV Vectors - Preclinical and Proof-of-Concept Studies
Authors:
Date and Time:
Title: Novel Genome Editing Therapy Improves Disease Phenotype and Survival of a Mouse Model of Methylmalonic Acidemia
Session Title: AAV Vectors - Preclinical and Proof-of-Concept Studies
Authors:
Date and Time:
The study abstracts are available on the ASGCT meeting website and can be accessed via the following link: https://annualmeeting.asgct.org/
About sAAVyTM
LogicBio's next-generation sAAVyTM capsid platform is uniquely designed to overcome limitations with older-generation gene therapy capsids by bringing enhanced potency with the potential for increased safety. The company is creating libraries of advanced adeno-associated virus (AAV) capsids and vectors, which are developed through an iterative process with directed evolution, rationale design and machine learning. LogicBio's novel capsid technology supports the development of treatments in a broad range of indications and tissues, including serious diseases of the liver and many diseases that cannot be safely targeted by older-generation capsids.
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