"The increasing cost of manufacturing these treatments makes it extremely difficult, if not impossible, to develop and test gene therapy for many ultrarare diseases where the number of patients worldwide is very small and profitability low.

We were able to deliver these treatments to the children in our ongoing clinical trials thanks only to funding from a generous family whose own child is a participant. This grassroots approach is a common theme in ultrarare disease research – development and testing are often supported by parents, foundations and federal grants."

FYI - if anyone here is interested in helping these kinds of causes, a company called Orchard Therapeutics provides similar treatments and has just signed a historic agreement for reimbursement for treating MLD patients in Europe (you can read more about it here: https://www.globenewswire.com/news-release/2022/02/04/237899...).

Also big discretion: I do have a lot invested in the company and I have incurred significant losses since deciding to invest in it. In the brilliant world of pyramid schemes vested around crypto-coins and NFTs, I don't regret anything and will keep buying more shares to support the dignity of human life.