GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Re: AAT (alpha 1 antitrypsin) program

> Is a lower cost of GTC AAT vs plasma derived AAT part of the rational for the alpha-1 antitripsin program? Are competing products (inhaled) under development also plasma derived?<

Yes, and yes. Additionally, since AAT hereditary deficiency (unlike AT HD) requires chronic treatment, production capacity is a key component of the rationale for GTC’s program.

>Is there any way to characterize the probabilities this product has to succeed and the size, length, expense and difficulties of the trials etc.?<

GTC’s trial designs for injectable AAT will probably be similar to those used in Kamada’s injectable AAT program. I think it’s too soon to get quantitative on the probability of success for GTC’s program, but I haven’t seen anything to make me a doubter.