Discuss What are the considerations for preclinical studies?Specifically, what animals should be used?What are the three R’s?

Human Gene Therapy for Retinal Disorders

I.Introduction Is this document legally binding?

II.Considerations for Chemistry, Manufacturing, and Controls What’s the core guidance document that serves as a reference for this section?What CMC considerations are special for GT products that treat retinal disorders?

III.Considerations for Pre clinical Studies What are the considerations for preclinical studies?Specifically, what animals should be used?What are the three R’s?

IV.Considerations for Clinical Studies

a.Natural History Studies This talks about “natural history.” What does an understanding of the natural history of a disease mean in this context?

b.Study Design In the Study Design section, they talk about the use control groups. What are the considerations here?

c.Study Population And characteristics what do they recommend for the study population?
d.Study Use e.Safety Considerationsf.Study End points What are some of the endpoints that the FDA discusses? g.Follow-Up Duration

h.Patient Experience What is the value of this?

V.Expedited Programs We talked about the different programs that the FDA has for expediting approval of a drug.What are several of the programs?What programs does the FDA suggest in this document that a GT to treat hemophilia might be eligible?

VI.Communication with FDA What is the FDA’s recommendation in this section?Again, they mention OTAT within CBER and INTERACT.

VII.References Just notice that the guidance papers that we are reading make up quite a few of the reference

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