SMA

The Gaynors’ daughter Sophia had been diagnosed with SMA five years earlier.

But their relationship had started to fray when — with success in sight — Kaspar became part owner of AveXis, a biotech startup that had snapped up the rights to his SMA drug.

At the time, there were no treatments to slow or stop SMA.

Because his drug was a gene therapy, public grant money and private philanthropy played an especially central role, with the National Institutes of Health alone putting over $450 million into science related to SMA.

After an associate’s twins were born with SMA, he said he became convinced that Kaspar’s gene therapy was the answer.