Neurenati Announces its First Scientific and Clinical Advisory Board Meeting with KOLs to Advance the Development of NEU-001, a Promising Cure for Hirschsprung Disease

MONTRÉAL--(BUSINESS WIRE)--Neurenati, an innovative biotechnology company dedicated to developing cutting-edge therapies for unmet medical needs in rare pediatric diseases, is excited to announce the successful convening of its inaugural Scientific and Clinical Advisory Board (SCAB) meeting. This pivotal meeting recently brought together leading pediatric surgeons, gastroenterologists, and industry experts in regulatory affairs and drug development to collaboratively shape a strategic roadmap for advancing NEU-001, a fixed combination of glial cell derived growth factor (GDNF) and butyrate, into clinical stage.

The SCAB of Neurenati is composed of Dr Jacob Langer (pediatric surgeon, previously at SickKids Hospital, Toronto), Dr Tomas Wester (pediatric surgeon, Karolinska University Hospital, Stockholm), Dr Anne Dariel (pediatric surgeon, La Timone Hospital, Marseille), Dr Kattayoun Kordy (pediatric gastroenterologist, Founder and CEO Lucien Bio), and Dr Andrew Mulberg (pediatric gastroenterologist and former division deputy director of gastroenterology and inborn errors products, Center for Drug Evaluation and Research, US FDA).

The meeting served as a valuable platform for transatlantic dialogue, to share insights on the preclinical prospects of direct benefit, clinical trial design, and regulatory pathways for NEU-001. The diversity of expertise provided Neurenati with meaningful guidance on best clinical practices in both North America and Europe, a deeper understanding of the patient journey, and—most importantly—confirmed the urgent need to advance therapeutic solutions for individuals affected by Hirschsprung disease. “Hirschsprung disease is an uncommon condition that affects approximately one in 5,000 children. The only effective treatment at this time is a major surgical operation. The innovative work being done by Neurenati could revolutionize the treatment of this disease and could potentially avoid the need for surgery in these tiny children,” said Dr Langer. “Novel non-operative treatment options for Hirschsprung disease would have a significant impact on future management of these neonates,” confirmed Dr Wester.

Looking ahead, Neurenati is committed to leverage a strong collaboration with its scientific and clinical advisory board members as it navigates the path towards IND filing, ensuring that NEU-001 meets the highest standards of safety and efficacy. "We are thrilled to have gathered such an esteemed group of thought leaders who are passionate about advancing the field of medicine," said Meriam Kabbaj, DPharm, PhD, Chief Development Officer of Neurenati. "Their contributions will be instrumental in shaping Neurenati's approach as it prepares for the next phase of its clinical program, with a strong focus on delivering a safe and effective therapy to pediatric patients in need."

About Neurenati

Neurenati Therapeutics is a biotechnology company focused on developing innovative therapeutics aimed at addressing unmet medical needs in rare pediatric diseases with a strong commitment to scientific excellence and patient-centered approaches.