LogicBio Therapeutics (LOGC) Doses First Patient in Phase 1/2 SUNRISE Clinical Trial

LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that the first patient has been dosed with LB-001, the Company's investigational single-administration gene editing therapy based on its proprietary GeneRide™ platform, in the SUNRISE Phase 1/2 clinical trial in pediatric patients with methylmalonic acidemia (MMA). The child received the intravenous infusion of LB-001 at Monroe Carell Jr. Children's Hospital at Vanderbilt.

"Today's landmark announcement represents a significant step forward in gene editing for children suffering from early onset genetic diseases," said Frederic Chereau, president and chief executive officer of LogicBio. "In addition to dosing the first patient, we have now opened several trial sites and identified enough additional patients to fully enroll SUNRISE, subject to screening clearances. We look forward to providing an update on enrollment, dose escalation and age de-escalation in late 2021. In addition, we continue to expect to announce interim clinical data by the end of the year."

"This moment represents the next step in a potential new era for the treatment of MMA," said Kathy Stagni, executive director of the Organic Acidemia Association (OAA). "The OAA community of patients and caregivers are grateful to the researchers at LogicBio who have been dedicated to advancing this promising research and it is exciting to see that we are one step closer to a more hopeful future."

MMA is a rare and life-threatening genetic disorder for which there are currently no treatments addressing the underlying cause of the disease. To manage the symptoms and detrimental effects of MMA, patients must maintain a severely restrictive diet. Even with aggressive management, MMA patients often experience metabolic crises that can cause permanent neurocognitive damage.

The SUNRISE trial is initially enrolling patients 3-12 years old and will potentially enroll infants as young as 6 months once the first two patients meet certain safety parameters and a biomarker indicating genome integration and protein expression is detected.

"Many genetic medicines are unable to target pediatric indications such as MMA, but an early and durable intervention in this vulnerable population has the potential to prevent disease progression and irreversible symptoms, including neurological damage," said Daniel Gruskin, MD, chief medical officer of LogicBio. "Our goal is to provide a safe and durable therapeutic option to treat MMA early enough to make a meaningful difference in patients' lives and eliminate the need for invasive liver transplantation, which is increasingly performed in children suffering from this disease."

"This milestone takes us one step closer to bringing a much-needed treatment option to patients living with MMA. I look forward to helping advance the SUNRISE trial and seeing the results," said Thomas M. Morgan, MD, the principal investigator of the SUNRISE trial at Monroe Carell Jr. Children's Hospital at Vanderbilt.

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