CureDuchenne is pleased to announce that it has invested in SonoThera, supporting the advancement of its targeted, ultrasound-mediated gene delivery technology focused on the delivery of full-length dystrophin. SonoThera’s approach is designed to address multiple key limitations of conventional AAV-delivered gene therapies such as restricted payload capacity (limited to the delivery of a micro-dystrophin gene), immune responses, high cost and the inability to redose.
CureDuchenne’s funding will help advance SonoThera’s Duchenne muscular dystrophy program into the clinic, bringing this promising program one step closer to individuals and families affected by Duchenne.
This investment reflects CureDuchenne’s continued commitment to accelerating promising therapies through CureDuchenne Ventures, which strategically deploys capital to advance innovative science into clinical development.
Supporting Innovation Through CureDuchenne Ventures
Through CureDuchenne Ventures, CureDuchenne helps bridge the gap between discovery and clinical development by supporting high-potential programs that address significant unmet needs within the Duchenne community. Through funding, CureDuchenne helps de-risk innovative approaches and helps unlock the follow-on investment needed to advance therapies into clinical trials.
“At CureDuchenne, we are committed to supporting innovative approaches that have the potential to transform the lives of individuals living with Duchenne muscular dystrophy,” said Debra Miller, Founder and CEO of CureDuchenne. “SonoThera’s differentiated nonviral platform has the potential to address important limitations in current AAV-delivered gene delivery and bring treatments to families who have been left waiting. We are proud to support the company’s next phase of growth and clinical development.”
A Novel Approach to Duchenne
SonoThera’s technology is designed to enable targeted and repeatable delivery of gene therapy in a safe, well-tolerated and cost-effective manner.
Unlike current gene therapies, which deliver microdystrophin using an AAV vector and cannot be redosed, SonoThera’s approach aims to deliver full-length dystrophin through a nonviral platform.
This fundamentally different approach, which leverages widely available, FDA-cleared diagnostic ultrasound systems, enables a noninvasive outpatient procedure that could:
- Deliver full-length dystrophin protein
- Allow patients to receive multiple doses if needed to continue to benefit from treatment
- Reach all patients, regardless of genetic mutation
- Potentially be used in combination with other treatments
A Shared Commitment to the Duchenne Community
As SonoThera advances, CureDuchenne remains focused on its mission to accelerate treatments, fund innovative science, and ensure that promising research continues moving forward for the Duchenne community.
Through investments like this, CureDuchenne is helping build a stronger pipeline of therapies that can bring more treatment options to more people faster. Together, we are turning promise into progress and advancing toward a future where everyone with Duchenne muscular dystrophy has a transformative treatment.
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