Text of Orchard's Announcement Terminating its Bubble-Baby Treatment License

Here is the text of Orchard Therapeutics’ abandonment letter concerning its ADA-SCID treatment and the flap over how the company was dealing with compassionate care denials.

May 28, 2021 

To Advocates and Partners in Advancing Treatments for ADA-SCID: 

I am writing today to update you on decisions we have made with respect to OTL-101, an investigational gene therapy for ADA-SCID. 

As many of you know, I have dedicated my career to advancing novel gene therapies to address ADA-SCID and other rare primary immune diseases in children. After decades  pioneering this work in the academic setting, I helped establish Orchard Therapeutics in  2016 with the singular goal of bringing potentially transformative hematopoietic stem cell gene therapies forward to help as many patients as possible. 

Orchard has made significant financial investments and collaborated closely with University  of California at Los Angeles (UCLA), University College London (UCL) and the California  Institute for Regenerative Medicine (CIRM) to develop and advance OTL-101 for ADA-SCID.  We believe the clinical data for this program are very promising and we consider the recent publication in the New England Journal of Medicine to be a testament to that. 

Although the clinical data for investigational OTL-101 are very encouraging, we have encountered technical issues specific to the commercial-grade manufacturing processes for this particular therapy that must be addressed before we, or any other entity, could progress the program toward a regulatory submission. Without the ability to reliably manufacture OTL 101 at a commercial standard there is no way to receive FDA approval for the gene therapy.  We have actively worked with CIRM, UCLA, UCL and our external manufacturing partners to determine potential solutions to address these manufacturing requirements. In addition, we have sought partners willing to invest to help us take this work forward, but we have not identified a viable path forward on either front. 

We understand the importance of OTL-101 to the ADA-SCID community, and we are proud  of the progress we have made in advancing OTL-101 to date. However, we also recognize  and wish to respect the community’s strong preference that we return the program to our  academic partners at this time. For these reasons, we have made the difficult decision to  inform our collaborators at UCLA and UCL of our intent to terminate our license agreement  and facilitate a smooth transfer of OTL-101 back to them. 

Despite our decision to return the license, we will continue to stand by the commitment we made to our academic partners to support them with financial and material resources to seek to treat ADA-SCID patients under a separate compassionate use program that would be led and administered by their institutions.  

This would include commitments from Orchard to:  

• Make our data and Investigational New Drug (IND) application available so it can be  referenced when our partners seek to treat ADA-SCID patients under compassionate  use; 

• Donate clinical-grade vector for compassionate use; and, 

• Provide financial support to enable our partners to manufacture OTL-101 for their  compassionate use program.

I join you in hoping that the contributions we have collectively made to OTL-101 will ultimately result in a therapy that can receive regulatory approval and be widely available to the children and families who need it. 

With sincere gratitude for all you do, 

Bobby Gaspar, M.D., Ph.D. 

CEO, Orchard Therapeutics

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