First-in-Human – Methylmalonic Acidemia treatment trial.

Early data from Phase 1/2 clinical trial in pediatric patients with methylmalonic acidemia showed measurable levels of a biomarker indicating site-specific gene insertion and protein expression.
methylmalonic acidemia

Cilinical Trial of methylmalonic acidemia treatment.

LogicBio Therapeutics, Inc., a clinical-stage genetic medicine company, today announced clinical trial results demonstrating the first-ever in vivo genome editing in children. Early data from the company’s Phase 1/2 SUNRISE clinical trial showed measurable levels of albumin-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression. The SUNRISE trial is evaluating the safety, tolerability, and preliminary efficacy of LB-001, the company’s investigational, single-administration genome editing therapy, in pediatric patients with methylmalonic acidemia.

These results follow a recommendation from the independent Data Safety Monitoring Board overseeing the SUNRISE trial to continue the study without modification. The DSMB’s recommendation was based on an evaluation of the safety data from the first two patients enrolled in the trial. Per the FDA-cleared protocol, albumin-2A detection together with the DSMB continuation recommendation enables LogicBio to begin enrolling two patients in the higher dose cohort (with ages ranging three to twelve years old) and two patients in the lower age (six months to two years old) cohort at the lower dose of LB-001.

“We are very excited to have achieved this significant milestone in the field of genetic medicine,” said Fred Chereau, president, and chief executive officer of LogicBio. “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.”

SUNRISE is a first-in-human, open-label, multi-center, 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. LB-001 is designed to non-disruptively insert a corrective copy of the MMUT gene into the albumin locus to drive lifelong therapeutic levels of MMUT expression in the liver. LB-001 is based on the company’s proprietary GeneRide technology, which uses homologous recombination, a natural DNA repair process, to enable precise editing of the genome without the need for exogenous nucleases and promoters that have been associated with an increased risk of immune response and cancer.

“MMA 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 pediatric patients is achievable, we are one step closer to bringing a safe and effective genetic medicine to children suffering from MMA and, potentially, other early onset genetic diseases where early intervention is critical to achieve optimal health outcomes,” said Daniel Gruskin, MD, chief medical officer of 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.”

The Company remains on track to present additional interim data by the end of 2021.

methylmalonic acidemia

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