News CLN5

Dear Patient Advocacy Organizations,
Neurogene Inc., a biotech company committed to developing life-changing genetic medicines for patients and their families affected by rare neurological disorders, is excited to share an important update on our development program for CLN5, a subtype of Neuronal Ceroid Lipofuscinosis (NCL), or Batten disease. The clinical trial for the investigational AAV (adeno-associated virus) gene therapy, NGN-101, is now enrolling participants at the clinical trial site, University of Rochester Medical Center in Rochester, NY, USA.
The information below is available here: NCT05228145
This investigational gene therapy clinical trial is titled: A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease. This first- in-human gene therapy clinical trial is the first of its kind. Enrolled participants will receive NGN-101 to the brain [through intracerebroventricular (ICV) administration] and the eye [through intravitreal (IVT) administration] to address both the neurodegeneration and vision loss associated with CLN5 Batten disease.
About the Phase 1/2 Gene Therapy Clinical Trial for CLN5 Batten Disease:
• This is a prospective, open-label clinical trial
o Thisisaclinicaltrialwhereallparticipantswillknowtheyarereceivingtheinvestigationalgenetherapy
product and will be followed over time.
• This gene therapy clinical trial will enroll children who are 3 to 8 years old with genetically confirmed Neuronal
Ceroid Lipofuscinosis subtype 5 (CLN5) disease (a subtype of Batten disease)
o Inthistypeofclinicaltrial,itisimportanttohavetheparticipantssimilarinageandstageofdisease
progression to better understand the safety and clinical effects of the investigational gene therapy
• The investigational gene therapy, NGN-101, will be given as a single intracerebroventricular (ICV) dose into the
brain and a single intravitreal (IVT) dose into one eye
o Bothdoseswillbegivenduringthesameprocedure
• Each participant will be followed for safety and efficacy 5 years after dosing
o Forapproximatelythefirst6monthsafterthegenetherapyisgiven,theparticipantwillresidecloseto the clinical trial site to enable the clinical trial doctor to monitor and care for the participant
o Aftertheinitialsafetymonitoringperiod,therewillbetelephoneandin-personvisitswiththegene therapy clinical trial site in decreasing frequency for regular assessments over a 5-year period
• There is a comprehensive travel and expense policy in place covering trial-related costs and expenses
o Trial-relatedcostsandexpensesarepaidbyNeurogene;moredetailsofthespecificpolicycanbe
provided by the clinical trial site
• Although the clinical trial site is in the US, patients from around the world will be considered for enrollment
and are encouraged to contact Neurogene for more information at 877-237-5020 (US+1)
o Travelandaccommodationswillbearrangedforparticipantsintheclinicaltrial
o Theclinicaltrialstaffwillprovidemoredetails
• More information about the clinical trial, including inclusion/exclusion (eligibility) criteria, can be found at (see link above), from the Patient Advocacy & Engagement team at Neurogene, and from the staff at the clinical trial site

• Participation in this trial is voluntary and does not guarantee results
Educational Resources for Clinical Trials:
• Gene Therapy Clinical Trial Process – American Society of Gene & Cell Therapy (ASGCT)
Contact Neurogene for more information and to be connected with the clinical trial site:
• Families, physicians, and healthcare providers are encouraged to contact the Neurogene Contact Center at 877- 237-5020 (US+1) for more detailed information
• Email: Families interested in more information about the clinical trial can email Neurogene at:
• Email: Healthcare Providers interested in more information can email Neurogene at:
• Neurogene can arrange to have a professional translator available if needed
Additional information about Neurogene studies:
• Neurogene is also conducting a natural history study that is enrolling patients with CLN5 Batten disease
• Patients who do not participate in the Phase 1/2 Gene Therapy Clinical Trial for CLN5 Batten disease may be
eligible for the Natural History Study
• A natural history study is not an interventional clinical trial, and no investigational product is given
• This study is critically important research to better understand the natural course of CLN5 Batten disease
• More information can be found at:
Educational Resource for Natural History Studies:
• Understanding Cell & Gene Medicine for your Community cell-gene-medicine/
Additional information about Neurogene and the clinical trial can be found here: Sincerely,
The Patient Advocacy & Engagement Team at Neurogene

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