B. AP PD_f.pdf

Version 11-30-2016-1 FAR Part 11—Describing Agency Needs

PURCHASE DESCRIPTION

1. Statement of Need and Purpose: The Therapeutic Development Branch (TDB( requires FCDI iCell motor neurons to generate electrically active cultures that will be used in microelectrode array assays for toxicity screening for the N=1 Ionis ASO collaborative project.

2. Background Information and Objective:

3. The National Institutes of Health (NIH) is the nation’s leading medical research agency and the primary Federal agency whose mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge. The mission of the NIH also includes enhancing health, lengthening life, reducing illness and disability, conducting, supporting and making medical discoveries that improves and saves people’s health and lives.

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5. The National Center for Advancing Translational Sciences (NCATS) is a part of the NIH, with a mission to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

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7. The Therapeutic Development Branch (TDB) within the National Center for Advancing

Translational Sciences (NCATS) Division of Preclinical Innovation (DPI) collaborative project with Ionis is aimed at developing assays for predictive toxicity of antisense oligonucleotides (ASOs). To predict neurotoxicity in 2D culture, pilot studies using human induced pluripotent stem cell (IPSC)-derived (from non-fetal tissue source) spinal motor neurons suggest neuronal activity in microelectrode array (MEA) assays is impacted by treatment with neurotoxic ASOs.

These recently completed pilot studies using iPSC neurons from FCDI (POTS# 22- 001605, 22-003110) established the optimal culture conditions for measuring activity, such as cell density and timing of ASO exposure. The optimal number of kits (includes nutrients required for cell culture) to generate a 48-well plate was also determined. For the next phase of this study, enough neurons to generate 25 48-well plates will be required for MEA assays for ASO toxicity screens.

8. Generic Name of Product: Cryopreserved human iPSC derived motor neurons

4. Purchase Description:

Trade name: iPSC Motor Neuron Kit

Version 11-30-2016-1 FAR Part 11—Describing Agency Needs

Brand Name: iCell Motor Neurons Kit Manufacturer: FCDI Model: Kit, Donor 01279 Catalog: R1049

5. Salient characteristics: The Contractor’s kits includes specifically uniquenutrients and supplements required for the start of the culture.

iPSCs that are derived from the same patient as cells used in other assays reduces the impact of genetic diversity on assay variability.

6. Quantity: 50

7. Delivery Date: Within 30 working days of date of award

8. Period of Performance: N/A

9. Option(s): N/A