Bespoke Gene Therapy Consortium award
On May 16, 2023, the Bespoke Gene Therapy Consortium (BGTC) announced our gene therapy project partners Children’s Hospital of Philadelphia (CHOP) as award recipients. The BGTC is a public-private partnership funded by the National Institutes of Health (NIH) and biopharmaceutical and life sciences companies.
This award has funded the manufacturing of the AAV9 gene therapy and Phase I clinical trials and keeps the United MSD Foundation on track with its goal of gene therapy as a potential treatment option.
UPDATE! As of July 6, 2026, the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the first-ever clinical trial of an investigational gene therapy for children with MSD. This landmark decision authorizes the study to move forward into its first-in-human clinical phase, representing years of scientific innovation, collaboration, and unwavering determination.
This achievement reflects the collective efforts of MSD families, researchers, clinicians, generous donors, and partners across the AMP® Bespoke Gene Therapy Consortium (BGTC), the Foundation for the National Institutes of Health (FNIH), the National Institutes of Health (NIH), Children’s Hospital of Philadelphia (CHOP), and many others who believed this day was possible.
Links To Helpful Information
Announcing First-Ever MSD Gene Therapy Clinical Trial Cleared by FDA
A Letter to the MSD Community from our Partners at BGTC
Accelerating Medicines Partnership® (AMP®) program’s Bespoke Gene Therapy Consortium overview
Children’s Hospital of Philadelphia Bespoke selection press release
FNIH Bespoke award press release
FNIH press release re: publication of Regulatory Playbook to Advance AAV Gene Therapies for Rare Diseases (Feb 6, 2024)
FNIH Bespoke Gene Therapy Consortium (BGTC) Regulatory Playbook Version 1.0
Foundation for the National Institutes of Health (FNIH) (BGTC page)
COMMONLY ASKED QUESTIONS
What will the Bespoke award fund?
The Bespoke award will fund the manufacturing of the AAV9 gene therapy and Phase I clinical trials.
Who received the award?
The Children’s Hospital of Philadelphia (CHOP) received the award, with Dr. Rebecca Ahrens-Nicklas and Dr. Laura Adang being the co-Primary Investigators.
What is the United MSD Foundation’s role in the project?
The United MSD Foundation has been a driving force behind the foundational steps to clinical trials, including advocating for research on MSD, establishing a Biobank, and funding a Natural History Study and a toxicology study.
At the end of the award, will gene therapy be available to all MSD children?
We do not have an answer at this time. At the end of the clinical trial, everything will have to go through regulatory approval or the FDA.
When will the clinical trial start?
The U.S. Food and Drug Administration (FDA) cleared the clinical trial to proceed in July 2026. Before participants can be enrolled, several additional steps must be completed, including review and approval by the Institutional Review Board (IRB) at Children’s Hospital of Philadelphia (CHOP) and final clinical site preparation and activation. These important steps help ensure the study is conducted safely and in accordance with all regulatory requirements.
We will continue to share updates as they become available. Please check back regularly for the latest information.
How long will enrollment be open for?
We do not have an answer at this time. Please check back periodically for more information.
If accepted, how long is the estimated trial timeline?
Clinical trials will run for five years from the start of the trial.
Who will be eligible for the clinical trial?
We do not yet know the eligibility criteria for the clinical trial. It will be determined once the protocol is finalized as part of the FDA application.
Are there any exclusion criteria?
We do not yet know the exclusion criteria for the clinical trial. It will be determined once the protocol is finalized as a part of the FDA application.
How do I express interest in enrolling my child in the clinical trial?
When the clinical trial is recruiting, it will be posted on ClinicalTrials.Gov. The United MSD Foundation will also distribute information at that time. If you are not already engaged in the Natural History Study, please consider participating.
Will the clinical trial be open to U.S. and international families?
Yes, the clinical trial will be open to families worldwide.
Will there be travel assistance for participating in the trial?
Yes. As a part of the Bespoke Grant proposal, the United MSD Foundation pledged support toward patient travel for the clinical trial.
Will this reverse the progression of MSD?
There is no scientific evidence that AAV9 gene therapy will reverse the progression of MSD.
With the Bespoke award funding the clinical trial, does that mean the Foundation doesn’t need to fundraise anymore?
No, there are still research and family support funding needs. The Bespoke award solidifies funding for the next steps of the AAV9 gene therapy development.
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