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Neurogene Initiates Natural History Study of Aspartylglucosaminuria (AGU)

Key data now being collected to support endpoint assessment for
future gene therapy clinical trial

NEW YORK–(BUSINESS WIRE)–Neurogene
Inc
., a company founded with a mission to bring life-changing
medicines to patients and families affected by rare neurological
diseases, today announced that it has enrolled the first patient in a
natural history study of aspartylglucosaminuria (AGU), a rare
neurodegenerative lysosomal storage disorder. The goal of the study is
to provide important data to support a future gene therapy clinical
trial. The potential therapy is being investigated under a collaboration
agreement between Neurogene and UT Southwestern Medical Center and is
one of multiple sponsored agreements.

Lysosomal storage disorders are a group of diseases resulting from
defects in lysosomal enzyme function. Lysosomes are cellular organelles
responsible for the digestion of molecules. Defective or absent
lysosomal enzyme function leads to intracellular accumulation of
substrate molecules, which, in turn, lead to toxicity and cell death.
AGU is caused by a deficiency of the aspartylglucosaminidase (AGA)
enzyme, which leads to toxic accumulation of N-acetylglucosamines and
cellular dysfunction. AGU typically presents in childhood with
developmental delay. With disease progression, patients experience
psychomotor regression, worsening gait disturbance, behavioral and
emotional issues, and worsening intellectual disability. People with AGU
have a shortened lifespan, with mortality typically in the fourth decade.

“This is an exciting time for people with AGU and their families,” said
Kimberly Goodspeed, M.D., Assistant Professor at UT Southwestern Medical
Center and lead investigator of the study. “This study will collect the
first prospective data from AGU patients, which will be foundational for
developing a potential therapy for this under-served neurodegenerative
disease.”

“With the UT Southwestern Medical Center team, Neurogene is working to
better understand the progression of AGU and what endpoints could be
used in our future gene therapy trial,” said Effie Albanis, M.D.,
Neurogene’s Chief Medical Officer. “We are taking a streamlined approach
to clinical development to facilitate the fastest possible approval
timeline for a future gene therapy medicine, consistent with our belief
that patients should not be waiting longer than necessary for
life-altering medicines.”

About the natural history study
Neurogene Inc. is sponsoring
a longitudinal, prospective, observational natural history and outcome
measure discovery study into which approximately 20 patients with
genetically confirmed AGU will be enrolled. Patients, who are currently
being enrolled at UT Southwestern Medical Center, will undergo multiple
assessments including MRI of the brain, neuropsychological testing and
ophthalmological evaluation. Each patient will be followed for up to
five years. Kimberly Goodspeed, M.D., Assistant Professor at UT
Southwestern Medical Center, is the lead investigator. For additional
information about the trial, please visit www.clinicaltrials.gov,
NCT03853876.

About Neurogene Inc.
Neurogene Inc. was founded to bring
life-changing medicines to patients and families affected by rare
neurological disorders. We partner with leading academic researchers,
patient advocacy organizations and caregivers to bring therapies to
patients that address the underlying genetic cause of a broad spectrum
of neurological diseases where no effective treatment options exist
today. Our lead programs use AAV-based gene therapy technology to
deliver a normal gene to patients with a dysfunctional gene. Neurogene
is also investing in novel technology to develop treatments for diseases
not well served by gene therapy. For more information, visit www.neurogene.com.

Contacts

Sara Green, Ten Bridge Communications
sgreen@tenbridgecommunications.com
617-233-1714

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