LogicBio Therapeutics Announces SUNRISE Phase 1/2 Clinical Design for LB-001 for the Treatment of Methylmalonic Acidemia in Pediatric Patients
– LogicBio’s First IND Clearance Leveraging GeneRide, an In Vivo Homologous Recombination-based Genome Editing Platform –
– Enrollment to Start with Patients as Young as 3 Years Old, De-escalating Down to 6 Months old –
– Webcast and Conference Call Today at
“We’re pleased to announce our plans for developing LB-001 in pediatric patients with MMA. This is an important milestone for patients, their families, our company and for the genetic medicines space more broadly as we believe this is the first Investigational New Drug application (IND) clearance for an in vivo gene editing program harnessing homologous recombination,” said
SUNRISE - Phase 1/2 Clinical Trial of LB-001 in Pediatric Methylmalonic Acidemia (MMA)
The SUNRISE trial is a multi-center, open-label, Phase 1/2 clinical trial designed to assess the safety and tolerability of a single intravenous infusion of LB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. Six leading centers in
LB-001 Webcast and Conference Call Information
LogicBio will host a webcast and conference call on
The event will be broadcast live and available under the investor relations section of LogicBio’s website at https://investor.logicbio.com/. The dial-in details for the call are +1 833-519-1335 or +1 602-585-9978, Conference ID: 1151546. A replay of the webcast will be available on the LogicBio website for one month following the call.
LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery 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 is expected to begin in early 2021. In addition, LogicBio has a collaboration with
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 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.
LogicBio is headquartered in Lexington, Mass. 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, including those related to the Company’s plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Company’s research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; and its plans for LB-301 in Crigler-Najjar. 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. In particular, the impact of the COVID-19 pandemic on the Company’s ability to progress with its research, development, manufacturing and regulatory efforts, including the Company’s plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Company’s common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in
Associate Director, Investor Relations
Source: LogicBio Therapeutics