LogicBio Therapeutics Announces Successful Repopulation of Diseased Livers in Mice with Corrected Healthy Hepatocytes in Two New Indications Using GeneRide ™ Genome Editing Technology

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– Data from mouse models in hereditary tyrosinemia type 1 and Wilson’s disease showed that the modified hepatocytes in vivo expanded and significantly repopulated diseased livers, correlating with improved disease burden

Posted: October 21, 2021 at 8:00 a.m. EDT|Update: 1 hour ago

LEXINGTON, Mass., 21 October 2021 / PRNewswire / – LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company, is due today to present new preclinical data on its GeneRide â„¢ platform to the European Society of Gene and Cell Therapy ( ESGCT) Virtual Congress 2021, ongoing October 19-22, 2021. The newly presented preclinical data further validates previous research on methylmalonic acidemia (MMA) and highlights the selective advantage, a key feature of GeneRide technology, in two additional indications characterized by intrinsic liver damage, hereditary tyrosinemia of type 1 (HT1) and Wilson’s disease. The selective advantage allows modified hepatocytes carrying the corrective gene to survive and reproduce better than endogenous mutated hepatocytes and ultimately repopulate part or all of the diseased liver.

Data presented to ESGCT demonstrated mouse models of the three hepatic indications treated with GeneRide vectors to deliver corrective genes. In all of these models, expansion of corrected healthy hepatocytes was correlated with improvement in diseased markers.

In HT1 models with acute liver damage, data showed that hepatocytes corrected by GeneRide repopulated the entire liver within four weeks of administration, replacing diseased hepatocytes with corrected hepatocytes. HT1 mice are deficient in the gene encoding fumarylacetoacetate hydrolase (FAH), which is required to metabolize the amino acid tyrosine, resulting in the accumulation of toxic metabolites. HT1 mice that received the GeneRide-FAH vector were no longer dependent on current standard care for disease and demonstrated normal body growth, liver function, and undetectable levels of succinylacetone, one of the toxic metabolites that accumulate in patients with HT1. . Compared to the current standard of care, treatment with the GeneRide vector resulted in a superior reduction in succinylacetone and lower alpha-fetoprotein levels, a clinically validated biomarker for hepatocellular carcinoma and another risk factor for patients. HT1 untreated.

Wilson’s disease results from a defect in copper transport, leading to toxic copper build-up and tissue damage. In a mouse model of Wilson’s disease, hepatocytes corrected by GeneRide repopulated the liver over time, and the treated mice showed improvements in liver function, hepatomegaly, and urinary copper excretion.

“We are very pleased to present these preclinical data in HT1 and Wilson’s disease. These data demonstrated the repopulation of a diseased liver using our in vivo genome editing technology, resulting in corrected hepatocytes edited by GeneRide. The results in HT1 were particularly encouraging, demonstrating complete repopulation of the liver after treatment. This data further validates our technology and represents an important step as we continue our mission of giving hope in genetic medicine to those affected by devastating diseases, ”said Mariana Nacht, Ph.D., Scientific Director of LogicBio.

Shengwen Zhang, Director of Molecular and Cellular Pharmacology at LogicBio, will give an oral presentation highlighting the successful delivery of corrective genes by GeneRide in HT1, Wilson disease and MMA. A selective advantage and expansion of corrected hepatocytes was observed in these preclinical models, demonstrated by the detection of increasing levels of a labeled albumin protein, albumin-2A, a technological biomarker indicating the insertion of genes and l site-specific protein expression, as well as immunohistochemistry for the corrective protein in liver sections. The results also showed increasing levels of albumin-2A correlated with increased expression of the corrective gene and improved disease burden. The company believes that these data support the development of GeneRide vectors to sustainably treat multiple genetic diseases with liver dysfunction.

Additional posters on display at ESGCT highlight advancements in the company’s Adeno-Associated Virus (AAV) technology platform. A poster detailed the combination of LogicBio’s proprietary plasmids and the optimized transfection process in HEK293 cells in suspension, which resulted in a 10- to 25-fold increase in titers using an LK03 capsid in 50 L bioreactors. separate poster highlighted the recent development of anion exchange-based high pressure liquid chromatography (AEX), allowing LogicBio to use an analytical method to measure the percentage of complete capsids in a given sample of AAV-LK03.

Additional information about the meeting is available on the website ESGCT website.

The oral presentation and posters will be available in the Presentations section of the Company’s website at https://investor.logicbio.com/events-and-presentations/presentations.

About LogicBio Therapeutics

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to treat rare and serious diseases from infancy through adulthood. The company’s genome editing platform, GeneRide â„¢, is a novel approach for the precise insertion of genes exploiting a cell’s natural DNA repair process, potentially leading to higher levels of expression. long-lasting therapeutic proteins. The Company’s gene delivery platform, sAAVy â„¢, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a wide range of indications and tissues. The company is headquartered in Lexington, MA. For more information visit www.logicbio.com, which is not part of this version.

Forward-looking statements

The statements contained in this press release regarding the strategy, plans, outlook, expectations, beliefs, intentions and objectives of LogicBio are forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, as as amended, including, but not limited to, statements regarding validation. previous research; the potential of the GeneRide â„¢ platform; and the company’s belief that preclinical data supports the development of GeneRide vectors to sustainably treat multiple genetic diseases with liver dysfunction. The terms “believe”, “validate” and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the risk that existing preclinical data may not be predictive of results. results of ongoing or subsequent preclinical studies and / or clinical results; the potential direct or indirect impact of the COVID-19 pandemic on our business, operations and the markets and communities in which we and our partners, employees and suppliers operate; manufacturing risks; risks associated with changes in management and key personnel and periods of transition; the actual funding required to develop and commercialize product candidates, including for safety, tolerability, registration, manufacturing or economic reasons; the timing and content of decisions taken by regulatory authorities; the actual time required to initiate and complete preclinical and clinical studies; the competitive landscape; changes in the economic and financial conditions of LogicBio; and LogicBio’s ability to obtain, maintain and enforce patents and other intellectual property protections for the LB-001 and any other product candidate. Other risks and uncertainties include those identified under the heading “Risk Factors” in LogicBio’s annual report on Form 10-K for the fiscal year ended December 31, 2020 and other documents that LogicBio may file with the Securities and Exchange Commission of the United States in the future. These forward-looking statements (unless otherwise indicated) speak only as of the date of this press release, and LogicBio does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.

Investor contacts:
Laurence Watts
Gilmartin Group
(619) 916-7620
[email protected]

Stephen jasper
Gilmartin Group
(858) 525-2047
[email protected]

Media contacts:
Jenna Urban
Public Relations Berry & Company
W: 211-253-8881
C: 203-218-9180
[email protected]

Bill berry
Public Relations Berry & Company
W: 211-253-8881
Telephone: 917-846-3862
[email protected]

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SOURCE LogicBio Therapeutics, Inc.

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