SonoThera Demonstrates Novel Ultrasound Gene Delivery for X-Linked Alport Syndrome Treatment
核心洞察
SonoThera (搜索) presented new data at ASN Kidney Week 2025 showing successful delivery of full-length Col4a5 (搜索) gene expression using ultrasound-mediated delivery technology in murine and non-human primate models.
The company's proprietary RIPPLE (搜索) ultrasound-mediated delivery platform enables targeted biodistribution of genetic payloads without size restrictions in a non-invasive, redosable manner.
The research addresses X-Linked Alport Syndrome (搜索), a hereditary kidney disorder that currently has no approved disease-modifying treatments available.
SonoThera (搜索) announced promising preclinical results for treating X-Linked Alport Syndrome (搜索) (XLAS (搜索)) using its proprietary ultrasound-mediated gene delivery technology at the American Society of Nephrology (搜索)'s annual meeting in Houston, Texas. The biotechnology company presented data demonstrating successful delivery of full-length Col4a5 (搜索) gene expression in both murine and non-human primate models.
Novel Ultrasound-Mediated Delivery Platform
The company's RIPPLE (搜索) ultrasound-mediated delivery (UMD) technology represents a nonviral, non-invasive approach to genetic medicine delivery. The platform enables broad, highly targeted biodistribution of diverse payload formats without size restriction, allowing for genetic medicines that can be re-dosed and are designed to be safe, well-tolerated, and cost-effective.
The poster presentation, titled "Ultrasound-Mediated Delivery of Nonviral Col4a5 (搜索) Gene Therapy Vector in X-Linked Alport Syndrome (搜索) (XLAS (搜索)) Disease Model Mice and Nonhuman Primates," was presented by Ivan Krivega, PhD, VP of Gene Therapy at SonoThera (搜索), during the Monogenic Kidney Diseases session.
Addressing Unmet Medical Need
X-Linked Alport Syndrome (搜索) represents a significant unmet medical need, with no approved disease-modifying treatments currently available. The hereditary disorder affects kidney function, making the development of targeted genetic therapies particularly important for patients and their families.
"This new data further demonstrates the tremendous potential which ultrasound-mediated delivery of genetic medicines has in the treatment hereditary disorders such as XLAS (搜索), where presently, no approved disease modifying treatments exist," said Kenneth Greenberg, PhD, CEO of SonoThera (搜索). "In particular, UMD's ability to deliver full-length expressions of genes such as Col4a5 (搜索) is quite promising."
Technology Advantages
SonoThera (搜索)'s nonviral technology is designed to overcome prevailing limitations of genetic medicine. The ultrasound-mediated delivery approach enables the delivery of diverse genetic payloads without size restriction, addressing a key challenge in gene therapy development. The redosable nature of the treatment and its non-invasive delivery method distinguish it from traditional viral vector approaches.
The company, founded by Drs. Kenneth Greenberg, Michael Davidson, and Steve Feinstein, is headquartered in South San Francisco and focuses on developing genetic medicines that treat the root cause of human diseases. Their technology platform leverages novel capabilities to enable a pipeline of products addressing various genetic disorders.