Sanford Stem Cell Clinical Center

Target Product Profile: Product Name, date

Product Description
Target / Annotations
Describe source material: (autologous/allogeneic); species (human/animal) (ESC, MNC, iPS); cell line designation and source; ex vivo genetic modification / Include summary information regarding acquisition compliant with CIRM, NIH or ESCRO (stem cell source); Freedom to Operate with the source cell line(s); donor eligibility compliant with 21CFR1271 (IRB approved donor consent forms available, donor medical history and blood tests), derivation compliant with Good Tissue Practices
Indications and Usage: Disease or condition for which your product/therapy will be indicated
Target / Annotations
A statement that the product is indicated in the treatment, prevention, or diagnosis of a recognized disease or condition OR of an important manifestation of a disease or condition, OR for the relief of symptoms associated with a disease, OR indicated for a particular indication only in conjunction with a primary mode of therapy / Include summary information regarding completed or planned studies to support the target. Include study identification, and regulatory submission information as appropriate.
Dosage and Administration
Target / Annotations
•Route of administration (site and method, including administration device, if any)
•Expected dose (matrix of volume, active cell number, viability and function)
•Dose range shown to be safe and effective •Dosage intervals if applicable / Include summary information regarding completed or planned studies to support the safety and effectiveness of the proposed dosage and route of administration. Include study identification, and regulatory submission information as appropriate.
Use in Specific Populations (if indicated, otherwise delete rows)
Target / Annotations
When intended use is in pediatric, pregnant, geriatric or other at risk populations. Limitations, need for monitoring, specific hazards, differences in response, or other information pertinent to the population. / Summary information regarding completed or planned studies to support the safety and effectiveness in these patient populations. Include study identification, and regulatory submission information as appropriate.
Nonclinical Pharmacology
Target / Annotations
Summarize mechanisms of action at various levels (e.g., receptor membrane, tissue, organ, whole body). / Summary information supporting MOA. Include study identification, references and regulatory submission information as appropriate.
Nonclinical Safety
Target / Annotations
Summarize safety and tumorigenicity information / Summary information supporting safety. Include study identification, references and regulatory submission information as appropriate.

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