Sanford Stem Cell Clinical Center TARGET PRODUCT PROFILE: Product Name, date Product Description Target Describe source material: (autologous/allogeneic); species (human/animal) (ESC, MNC, iPS); cell line designation and source; ex vivo genetic modification Annotations 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 Include summary information regarding treatment, prevention, or diagnosis of a completed or planned studies to support the recognized disease or condition OR of an target. Include study identification, and important manifestation of a disease or condition, regulatory submission information as OR for the relief of symptoms associated with a appropriate. disease, OR indicated for a particular indication only in conjunction with a primary mode of therapy Dosage and Administration Target Annotations •Route of administration (site and method, Include summary information regarding including administration device, if any) completed or planned studies to support the safety •Expected dose (matrix of volume, active cell and effectiveness of the proposed dosage and number, viability and function) route of administration. Include study •Dose range shown to be safe and effective identification, and regulatory submission •Dosage intervals if applicable information as appropriate. Use in Specific Populations (if indicated, otherwise delete rows) Target Annotations When intended use is in pediatric, pregnant, Summary information regarding completed or geriatric or other at risk populations. Limitations, planned studies to support the safety and need for monitoring, specific hazards, differences effectiveness in these patient populations. Include in response, or other information pertinent to the study identification, and regulatory submission population. information as appropriate. Nonclinical Pharmacology Target Annotations Summarize mechanisms of action at various levels Summary information supporting MOA. Include (e.g., receptor membrane, tissue, organ, whole study identification, references and regulatory body). submission information as appropriate. Nonclinical Safety Target Summarize safety and tumorigenicity information Annotations Summary information supporting safety. Include study identification, references and regulatory submission information as appropriate.