Text of Maria Millan's Statement on the Orchard Bubble Baby Decision
 |
Here is the text of the statement by Maria T. Millan, president and CEO of the California stem agency, on Orchard’s decision to give up its license on the ADA-SCID treatment.
“The news that Orchard Therapeutics is returning OTL-101 to UCLA is wonderful news for families who have children with ADA-SCID. This means that UCLA will now be able to begin the process of applying to the Food and Drug Administration for permission to start an expanded use protocol to treat patients. This may take several months but CIRM will be helping and supporting UCLA at every stage of the process to make this happen as quickly as possible.
“We know this has been a difficult time for the families of children with ADA-SCID and we are grateful to them for their patience and support as we worked through this. Throughout this process our sole purpose was to ensure that children in need were once again able to access this life-saving therapy. CIRM has supported the work of Dr. Don Kohn and his team for many years, and we are gratified to be able to help them once again.
“We want to thank Dr. Bobby Gaspar and his team at Orchard and our colleagues at UCLA for working hard to make this happen. We have been in discussions for many months trying to find a way forward for this therapy, and now we have done that.
“This is why CIRM was created. We are far more than just a funding agency. We are committed to doing everything we can to bring therapies to patients, and that means overcoming challenges and obstacles whatever they are. This is why we come to work every day, because patients are at the heart of everything we do.
“This is a good day for CIRM, Orchard and UCLA. But it’s a great day for our patients and their families.”
(CIRM later posted the statement on its website. That version can be found here.)
| 1 |
As a patient advocate of many years, I would like to express my appreciation to Orchard Therapeutics for giving up its license on OTL-101, the ADA-SCID treatment. That cannot have been an easy decision. Thanks are due to Dr. Bobby Gaspar and his team, and also the California Institute for Regenerative Medicine (CIRM) which has funded the research of Dr. Don Kohn, which saved the lives of 50 children. Now the way is clear for progress; a wonderful therapy can move forward.
It will take time and continuing effort, I am sure. But that is what CIRM does; and why California voted to continue it.
Thank you, one and all.