Donated Resource Application 2025
First name
Last name
Email
Phone Number
Institution Name
Institution Address
Institution Type
Nonprofit
University
Research Foundation
Hospital
Pharmaceutical/Drug Discovery
Biotechnology/Life Science
Contract Laboratory
Other
Please provide a brief background on the main applicant, including past experience with individualized therapy development and other relevant expertise. Please also describe teaming strategy, including collaborating organizations, where applicable.
Which resource are you requesting?
Please provide a description of the work and the intended scope of the resource’s application. If applicable, include details of funding acquired to facilitate the work. Please avoid sharing any confidential information.
If applicable, please confirm the precise variant and, if possible, provide an annotation verified by Mutalyzer, VariantValidator, or a similar tool. The annotation will help expedite the review process.
We encourage you to share additional insights into key areas of your project. While optional, this will help us understand how we can assist you. If applicable, please outline your answers in a document and upload it using the feature below.
Mutation details, pathomechanism, and effect on protein level
Proposed therapeutic approach (ASO + strategy, gene replacement, etc.)
Disease mechanism, phenotype, and expected clinical benefit
Mechanism of action, experimental systems, production and testing teaming strategy
What controls will be used and how will allele-selectivity be studied?
Functional assays
Methods to measure target engagement in vitro and in patients
Biomarkers if applicable
Known regulatory path for bringing a patient-specific therapy into the clinic in your country
Institutional support and resources for N-of-1 trials
Have clinical outcome measures been established?
Have patient and treating clinician been informed of therapeutic development?
Has patient consented to study?
What is the predicted disease course and progression for this patient?
What are the primary treatment goals for this patient?
Is the patient stable enough to still be treatable after the duration required for drug development?
Frequency of the causative variant in the family?
Could other patients benefit from the therapy?
Upload File (optional)
Select a File
Please limit responses to each consideration above to 400 words max.
Additional Criteria
Has your institution executed a Data Sharing Agreement with N1C?
I have read and agree to the N1C Release Policy
I have read and agree to the N1C Review and Management Policy
I have read and agree to the N1C Guidelines for Publicizing Donations
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