Clinical Trials
Gene Editing
Gene Therapy

Early Clinical Trial Results Suggest a Step Towards the First In Vivo Genome Editing Opportunity in Children

Georgi Makin
20 October 2021
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LogicBio Therapeutics has announced early data from Phase I/II clinical trials that reportedly demonstrate the first-ever in vivo genome editing in children.

The LogicBio team has been investigating the safety, tolerability and preliminary efficacy of LB-001 – an investigational, single-administration genome editing therapy – in paediatric patients with methylmalonic acidemia.

Early findings from the SUNRISE trial demonstrated measurable levels of albumin-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression.

Results based on safety data from the first two patients prompted the independent Data Safety Monitoring Board to recommend continuation of the trial, and the further enrolment of children as young as six months, as well as dose escalation.

“We are very excited to have achieved this significant milestone in the field of genetic medicine,” Fred Chereau, President and CEO of LogicBio, commented.

“These early data indicate that we can precisely edit hepatocytes in vivo to treat a genetic liver disease with a single intravenous infusion using our proprietary GeneRide™ technology. Today’s announcement is a demonstration that homologous recombination genome editing without the use of nucleases is a potential alternative to genome editing technologies in development that use nucleases, such as CRISPR. The ability to insert the correct version of a gene in a cell’s genome without nucleases is an important step to unlocking the potential of GeneRide™ to treat a larger number of genetic diseases,” Fred continued.

The SUNRISE trial is a first-in-human, open-label, multi-centre, Phase I/II clinical trial, set up to evaluate the safety and tolerability of a single intravenous infusion of LB-001 in children with methylmalonic acidemia.

LB-001 – based on LogicBio’s GeneRide technology – relies on the natural DNA repair process, homologous recombination, to enable precise editing of the genome without the need for various exogenous nucleases and promoters sometimes associated with an increased risk of immune response and cancer.

“Methylmalonic acidemia is a rare, life-threatening genetic disorder for which there are no treatments addressing the underlying cause of the disease. By demonstrating for the first time ever that in vivo, nuclease-free genome editing in paediatric patients is achievable, we are one step closer to bringing a safe and effective genetic medicine to children suffering from methylmalonic acidemia and, potentially, other early onset genetic diseases where early intervention is critical to achieve optimal health outcomes,” explained Daniel Gruskin CMO at LogicBio.

“I would like to thank the patients, their families and the investigators who are participating in this landmark trial. We look forward to continuing to progress the clinical study to better understand the biochemical and clinical effect of this genome editing therapy,” Daniel concluded.

According to a recent press release, LogicBio is on track to present additional interim data by the end of this year.

Source: LogicBio Therapeutics press release