LogicBio Therapeutics Provides Update on FDA Review of Investigational New Drug Application for LB-001 for Methylmalonic Acidemia
LB-001 is an investigational pediatric genome editing therapy based on LogicBio’s GeneRide™ technology. GeneRide enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LB-001 is designed to incorporate a functioning version of the faulty human methylmalonyl-COA mutase (MMUT) gene into the genome of MMA patients. LogicBio has demonstrated preclinical proof-of-concept of GeneRide in multiple animal models of the disease, improving survival and reversing disease pathology. In preclinical MMA models, LogicBio has shown that cells into which GeneRide has inserted a transgene demonstrate a selective survival advantage over cells not expressing the transgene. LB-001 has received both orphan drug and rare pediatric disease designations from the
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Forward Looking Statements
This press release contains “forward-looking” statements within the meaning of the federal securities laws, including with respect to the Company’s plans to resolve the FDA’s clinical hold on the IND for LB-001. These are not statements of historical facts and are based on management’s beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Company’s plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Company’s current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Company’s filings with the U.S. Securities and Exchange Commission (
Associate Director, Investor Relations
Ten Bridge Communications
Source: LogicBio Therapeutics