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Amicus Therapeutics (NASDAQ:FOLD) announced more positive preliminary data from the CLN6 Batten disease gene therapy program, AT-GTX-501.

Amicus Presents AT-GTX-501 Results

The published results were presented in a poster presentation during the virtual Joint 16e International Congress of Child Neurology and 49e Annual meeting of the Society for Childhood Neurology. The Abigail Wexner Research Institute (AWRI) at Children’s National Hospital is conducting the ongoing Phase 1/2 clinical study. The study is evaluating a single intrathecal administration of At-GTX-501 gene therapy for a type of late childhood neuronal ceroid lipofuscinosis called CLN6 Batten disease. Currently, there are no approved treatments for CLN6 Batten disease, which is a neurological condition that can impair a child’s cognitive and motor functions.

Preliminary safety results are available for 13 children with CLN Batten disease, while interim efficacy results are available for the first 12 children. These are the children who reached the 12-month time point and eight children who reached 24 months after administration of AAV-CLN6 gene therapy.

AT-GTX-501 well tolerated by patients

AT-GTX-501 treatment was well tolerated in children with mild unrelated adverse effects. There was no observed pattern of adverse events related to the immunogenicity of CLN6 or AAV. Gait and speech assessment showed significant effects in reducing disease progression at 12 and 12 months.

Amicus Chief Development Officer Jeff Castelli said he was pleased to share positive interim clinical data for intrathecal AAV gene therapy with the CLN6 community. He added that the data suggests that gene therapy has the potential to treat CLN6 batten disease in children. This rare disease can lead to a progressive decline in motor and cognitive functions and sometimes leads to premature death.

The study’s lead researcher, Emily de loos Reyes, said she was pleased with the progress of the CLN6 clinical trial. Reyes also said collecting natural history data will further inform the results of the AAV-CLN6 gene therapy study. She added that preliminary results indicate that AT-GTX-501 has the potential to slow disease progression in patients with CLN6 Batten disease.