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LogicBio Announces Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

May 4, 2021 at 8:00 AM EDT
- Data demonstrate better in vivo potency and manufacturability of novel AAV sL65 capsid, a new vector for targeting liver diseases
- Additional preclinical data show treatment with mLB-001, based on proprietary GeneRideTM gene editing platform, improved disease phenotype and survival of methylmalonic acidemia in mice
- Efficacy data demonstrated with use of GeneRide vector in mouse model of Crigler-Najjar syndrome

LEXINGTON, Mass., May 4, 2021 /PRNewswire/ -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced several preclinical data presentations, including an oral presentation, will be delivered at the 24th Annual American Society of Gene and Cell Therapy (ASGCT) Virtual Meeting being held May 11-14, 2021. In an oral presentation, Dr. Jing Liao, Associate Director at LogicBio, will discuss results from preclinical studies of the adeno-associated virus (AAV) sL65, the first AAV capsid produced from LogicBio's sAAVyTM platform, for the potential treatment of serious diseases of the liver. Results show high potency in a humanized mouse model and in non-human primates compared to widely used benchmark liver targeting capsids. The sL65 capsid also shows high production yields in suspension HEK293 cells and in bioreactors.

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 Frederic Chereau, President and Chief Executive Officer of LogicBio.

"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 Mariana Nacht, Ph.D., Chief Scientific Officer of LogicBio.

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: Jing Liao, Ph.D., Associate Director at LogicBio
Date and Time: Tuesday, May 11, 2021, 5:30 p.m. - 5:45 p.m. EDT

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: Chih-Wei Ko, Amy Bastille, Shengwen Zhang, Elizabeth McCarthy, Susana Gordo, Jenisha Vora, Dylan Frank, Nikhil Ramesh, Jing Liao, Lauren Drouin, Matthias Hebben, Kyle Chiang, Roshan Padmashali, Ankit Gupta, Vivian Choi, Nelson Chau, Qiang Xiong
Date and Time: Tuesday, May 11, 2021, 8:00 a.m. - 10:00 a.m. EDT

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: Amy Bastille, Xiaohan Zhang, Nikhil Ramesh, Jenisha Vora, Elizabeth McCarthy, Dylan Frank, Chih-Wei Ko, Carmen Wu, Noel Walsh, Jing Liao, Susana Gordo, Lauren Drouin, Matthias Hebben, Kyle Chiang, Nelson Chau, Shengwen Zhang
Date and Time: Tuesday, May 11, 2021, 8:00 a.m. - 10:00 a.m. EDT

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.

About LogicBio Therapeutics, Inc. 

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide™ platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVy™ capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit https://www.logicbio.com/.

Media Contacts:

Bill Berry
Berry & Company Public Relations
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C: 917-846-3862
bberry@berrypr.com

Jenna Urban
Berry & Company Public Relations
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C: 203-218-9180
jurban@berrypr.com

Investor Contacts:

Matt Lane
Gilmartin Group
617-901-7698
matt@gilmartinir.com

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SOURCE LogicBio Therapeutics, Inc.