LogicBio Announces Extension Of Collaboration With Children's Medical Research Institute
"I am very pleased to announce the expansion of our collaboration with CMRI, which over the past two years has already led to many important advances in our liver-targeting AAV vectors research," said
New data highlighting the advantages of next-generation AAV capsids developed through the collaboration have been presented at multiple leading scientific conferences including the
The Company believes that these novel AAV capsids have broad potential applications in gene therapy and for gene editing platforms such as GeneRide™, LogicBio's proprietary gene editing technology that harnesses a cell's natural DNA repair process, known as homologous recombination, to insert a corrective copy of a gene at a precise spot in a patient's genome.
"The expansion of our research collaboration with CMRI comes at a very exciting time for LogicBio as we are poised to enroll the first patients in our SUNRISE Phase I/II clinical trial for LB-001 in the treatment of methylmalonic acidemia,"
"The approval of our IND by the FDA for the SUNRISE trial to treat very young patients is a reflection of the quality of our pre-clinical data and the design of our clinical trial," said Dr.
LB-001 was granted Fast Track designation by the FDA in
Upcoming LogicBio investor conference presentations:
- The 39th annual
J.P. Morgan Global Virtual Healthcare Conferenceon Thursday, January 14, 2021at 4:30 PM ET.
- The 2021 HC Wainwright BioConnect 2021 Conference, which will take place from
January 10-14, 2021. Mr. Chereau'spresentation will be available on demand to conference attendees for the duration of the event.
LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering platforms. LogicBio's proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment in the phase I/II SUNRISE clinical trial is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.
LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort have demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.
This press release contains "forward-looking" statements within the meaning of the federal securities laws, including with respect to the Company's upcoming development milestones and opportunities, the collaboration's research efforts and the timing of patient enrollment in the phase I/II SUNRISE clinical trial. 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