Replies to post #245803 on Biotech Values

[rfj1862]

Have you gotten any feedback from people in the biotech industry on the ideas for gene-therapy programs that you posted? TIA

I'm sorry Dew I completely missed this.

I haven't gotten feedback from people in industry, but it is certainly a point that I have discussed with my medical geneticist colleagues. The average medical geneticist can treat dozens of different diseases in a single practice, and they express frustration that for the vast majority of patients with readily addressable monogenic genetic disorders will never have a viable gene therapy because of the approval process and the fact that it currently isn't commercially viable to do the full approval process for diseases that affect only a few hundred patients. By "readily addressable" I mean that the disease is caused by a defect in a single gene that is well understood.

Given the seriousness of these conditions and the lack of viable therapeutic options the entire process should be streamlined. Certainly for AAV, which is easy to administer. Lentiviral gene therapies, which generally require a BMT-like procedure, may be less attractive for a platform approach.

I guess if I *were* to invest in a gene therapy company (and I'm not) I would focus on AAV rather than lentiviral for the reason above.