Biotech Values

[DewDiligence]

ADVM—Dose escalation is always planned…

Some phase-1 trials start high and use dose de-escalation, while other phase-1 trials use a combination of escalation and de-escalation (e.g. “3x3” design). But the ADVM trial in question is clearly one with escalation only.

…if the efficacy is good (especially in Gene Therapy) they don't go up [to a higher dose].

This strikes me as painting with too broad a brush. Seeing early efficacy in the ADVM trial is going to be almost impossible for the reason I mentioned in the prior post in this thread.

Do you feel the same about Dry AMD?

Dry AMD is thought to be somewhat less complex than wet AMD, so my argument against gene therapy is weaker than in wet AMD; however, the admonition against GT still pertains to a lesser degree, IMO.

…AAVL (in your referenced post) is ADVM today [following a reverse merger with Annapurna]…

I suppose that qualifies as irony :- )