UP FRONT
participants in the study to lead
active lifestyles without the need
to take any clotting factor.
This work has made a real
difference to the people who
volunteered to participate in
the trials and the results have
far-reaching implications for
the larger hemophilia patient
population.
Then, later that month, the
biotech company that I co-
founded, Spark Therapeutics,
received regulatory approval for
the first adeno-associated viral
(AAV) gene-therapy product in
the U.S., for a rare form of con-
genital blindness. I’m extremely
proud of that.
“If you’re
really trying
to solve a
problem, ...
remember
that you
own all the
problems –
not just the
[ones] that
you like.”
On the other side, have
you ever had any major
disappointments in your
career – and how did you
handle th