PHILADELPHIA, PA — Latus Bio, Inc. has announced a significant advancement in the field of gene therapy with the publication of its latest research in Nature Communications. The study, led by Latus founder Beverly Davidson, Ph.D., reveals the discovery of an innovative adeno-associated virus (AAV) capsid variant, AAV-DB-3, which has the potential to revolutionize treatments for neurodegenerative diseases such as Huntington’s disease (HD) and Parkinson’s disease (PD).
The novel capsid, AAV-DB-3, was identified through a highly selective screening of a large-diversity AAV library administered to non-human primates (NHPs). Among tens of millions of candidates, AAV-DB-3 emerged for its exceptional capability to target neurons within deep brain structures, a long-standing challenge in the field of gene therapy.
According to the research, AAV-DB-3 offers several critical advantages. It effectively targets Medium Spiny Neurons in the striatum and Projection Neurons in the cortex, regions heavily affected in HD and PD. The capsid achieves this efficiency with doses that are 10 to 100 times lower than currently utilized clinical treatments. Beyond this, it boasts an ability to drive transgene expression at levels more than 180 times higher than wild-type AAV5 in HD-relevant brain areas following direct delivery. Furthermore, in neurons derived from human patients, AAV-DB-3 showed a greater than 60-fold improvement in transgene expression compared to wild-type AAV1, underscoring its potential for clinical application.
“AAV-DB-3 represents a major step forward in solving one of the biggest challenges in both Huntington’s and Parkinson’s disease gene therapy — delivering genetic payloads precisely and effectively to deep brain structures,” said Dr. Jang-Ho Cha, Chief Scientific Officer at Latus.
Dr. Beverly Davidson, Chair of the Scientific Advisory Board at Latus, emphasized the broader implications of the discovery. “These findings suggest that AAV-DB-3 and other capsid variants from our screening platform are highly promising vectors for a broad range of neurodegenerative disorders that affect deep brain and associated cortical brain regions,” she said.
By addressing long-standing barriers in gene therapy and advancing tools for targeted delivery, Latus Bio is poised to reshape treatment possibilities for patients suffering from debilitating neurodegenerative conditions. The study highlights the impact of precision in genetic medicine and positions AAV-DB-3 as a potentially game-changing solution in this critical field of research.
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