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.