LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) for LB-001 for Methylmalonic Acidemia (MMA) and Highlights Key Corporate Milestones
– Filed IND for lead GeneRide™ candidate LB-001 in pediatric MMA patients –
– Phase 1/2 trial initiation planned for H1 2020, with preliminary data in H2 2020 –
– Established research collaboration with Takeda for GeneRide in Crigler-Najjar Syndrome (CN) –
LogicBio intends to disclose additional details regarding the planned Phase 1/2 trial, including trial size, endpoints, and timelines, once the
“We founded LogicBio with the mission of bringing genetic medicines to children with rare diseases. Both the IND submission and the nomination of our second indication represent significant steps in advancing our goal,” said
Today, LogicBio also highlighted key recent and upcoming milestones.
- Established collaboration with Takeda to leverage the GeneRide platform in a second indication, Crigler-Najjar Syndrome. LogicBio and Takeda will further research and develop LB-301, an investigational pediatric genome editing therapy based on LogicBio’s GeneRide technology for the treatment of CN. The LB-301 construct, utilizing the modularity of GeneRide, is expected to share several components with LB-001 to facilitate development. Those components include: LK-03 as the capsid; the albumin gene as the target genetic locus for integration; and a 2A peptide sequence to facilitate polycistronic expression and serving as a circulating biomarker. A separate press release with more details can be found on LogicBio’s investor relations website at investor.logicbio.com.
- Initiated Retrospective Natural History Study in MMA. This study is designed to evaluate disease progression in pediatric patients (born since 2010) with severe MMA, with the aim of informing LogicBio’s future development in MMA and its discussions with regulatory agencies.
- Reported positive data on
Next Generation Capsid Developmentprogram at European Society of Gene and Cell TherapyAnnual Meeting. Data were presented from a set of novel, synthetic adeno-associated virus (AAV) capsid candidates tested against references AAV2, AAV8, and LK-03. All capsids showed selective tropism and more potent transduction and gene expression than the reference capsids in human hepatocytes of a chimeric FRG mouse model. LogicBio, working in partnership with the Children’s Medical Research Institute, intends to advance this research and present additional findings at a scientific conference in 2020.
- Doubling available lab and office space to support GeneRide platform development and capabilities expansion. LogicBio expects to move into new headquarters in
Lexington, Mass.in the spring of 2020. LogicBio will be adding vivarium space, internal development capabilities, and will increase its capacity for in-house manufacturing of preclinical material. The expanded lab space will support the continued advancement of a robust pipeline that builds on the modular GeneRide construct. The new facilities will also support continued growth of the capsid program, which aims to develop and license new state-of-the-art viral vectors.
About Methylmalonic Acidemia
Primarily caused by mutations in the MMUT gene, methylmalonic acidemia is a rare, life-threatening, autosomal recessive disease for which there are no approved therapies. The disease, which starts in the first month of life, prevents the body from properly processing certain fats and proteins, resulting in a toxic accumulation of metabolites that can cause life-threatening decompensations in infants and children. This buildup can lead to significant morbidity and mortality, including infections, neurodevelopmental disabilities and chronic kidney disease. The incidence of MMA in
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 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
For more information, please visit www.logicbio.com.
Forward Looking Statements
This press release contains “forward-looking” statements within the meaning of the federal securities laws. 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