NGGT Announces Positive Data on NGGT002 for Treating PKU

NGGT Inc., known as Next Generation Gene Therapeutics, recently made an important announcement regarding their gene therapy candidate NGGT002, currently under investigation for the treatment of Phenylketonuria (PKU). PKU is a rare genetic disorder caused by a mutation in the phenylalanine hydroxylase (PAH) gene, leading to high levels of phenylalanine in the blood, which can result in severe intellectual and behavioral impairments, skin issues, movement disorders, and seizures if untreated.

The company disclosed promising findings from their investigator-initiated trial (IIT) conducted in China. These findings were presented at the National PKU Alliance biennial conference in July. The data indicate that NGGT002 is both safe and effective in treating PKU. In this study, five out of six patients who received a high dose of NGGT002 displayed normal plasma phenylalanine levels just three weeks after treatment. Remarkably, the first patient administered with NGGT002 maintained normal phenylalanine levels even after 40 weeks post-treatment.

Dr. Yiting Liu, Vice President of Translational Research at NGGT, expressed optimism about the results, highlighting the potential of NGGT002 as a lasting and effective treatment for PKU. The initial results over a span of 40 weeks are promising, and the company looks forward to further validating these findings through their ongoing Phase I/II clinical trials.

Dr. Lixin Jiang, CEO and Co-Founder of NGGT, emphasized the challenges faced by PKU patients, particularly regarding dietary restrictions, and noted how effective and durable reduction of phenylalanine levels could significantly benefit patients. Given the impressive safety and efficacy results from the IIT, the company is excited about the future outcomes of their current clinical trials in the U.S. and China.

NGGT has initiated Phase I/II clinical trials in both countries to further assess the safety and efficacy of NGGT002 in adult patients with PKU. These trials, identified as NCT06332807 in the U.S. and NCT06061614 in China, will dose the first patients based on the effective dosage established in the IIT study.

The U.S.-based clinical trial is an open-label, multiple-center, dose escalation, and cohort expansion study. It aims to evaluate NGGT002, an rAAV-based vector carrying a functional copy of the human PAH gene, in adults with classic PKU. Participants in the trial will receive a single administration of NGGT002 and will be monitored for efficacy over one year and for safety over five years.

NGGT Inc., headquartered in Walnut Creek, California, is a clinical-stage biotechnology company focused on developing gene therapies for various genetic diseases, including retinal, metabolic, and neurodegenerative disorders. The company is advancing its therapeutic development pipeline with several proprietary AAV (Adeno-Associated Virus) products. Among these are NGGT001 for Bietti's Crystalline Dystrophy, NGGT006 for familial hypercholesterolemia, and NGGT007 for Wet-AMD, alongside NGGT002 for PKU.

NGGT continues to innovate in the field of gene therapy, aiming to provide effective treatments for rare genetic diseases. The company's recent achievements with NGGT002 mark a significant step forward in their mission to improve the lives of patients suffering from PKU.