PHILADELPHIA--(BUSINESS WIRE)--#AAV--Latus Bio Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced upcoming oral and poster presentations at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, which is being held from May 13-17th, 2025 in New Orleans, LA. These presentations showcase progress toward advancing Latus’ novel AAV capsid variants - AAV-DB-3 and AAV-Ep+ - to address Huntington’s Disease and CLN2 Batten Disease, respectively.
“Over the last year, our progress on two preclinical gene therapy programs as well as our novel AAV capsid discovery platform has been remarkable,” said P. Peter Ghoroghchian, MD, PhD, CEO of Latus. “We are excited to share novel findings that have the potential to transform the treatment landscape of patients with genetic diseases.”
Latus will present three abstracts in support of its novel capsid variant AAV-Ep+ that is being used to develop a potential gene therapy (LTS-101) to address CLN2 Disease. Data presented include a program overview of LTS-101 as well as preclinical data that demonstrate its safety, biodistribution, significant transgene expression levels in non-human primates, as well as efficacy in a mouse model of CLN2 disease at low vector doses that support its potential clinical utility.
Title: LTS-101: An Intracerebroventricular Delivered AAV Gene Therapy Using a Novel Capsid Variant for the Treatment of CLN2 Batten Disease
Presenter: Dr. Jang-Ho Cha, MD, PhD
Presentation Type: Poster presentation, #1931
Date, Time: May 15, 2025; 5:30 – 7:30 PM
Title: Preclinical Mouse Pharmacology Studies Evaluating the Biodistribution and Pharmacodynamics of LTS-101, a Novel Gene Therapy Candidate for CLN2 Batten Disease
Presenter: Dr. Katie Villa, PhD
Presentation Type: Poster presentation, #1924
Date, Time: May 15, 2025; 5:30-7:30 PM
Title: A Non-GLP Dose Range Finding Study in Cynomolgus Macaques Evaluating the Biodistribution, Expression, and Safety of LTS-101, a Novel Preclinical Gene Therapy Candidate for the Treatment of CLN2 Batten Disease "
Presenter: Dr. David Lieb, PhD
Presentation Type: Poster presentation, #521
Date, Time: May 13, 2025; 6:00 – 7:30 PM
Latus will present three abstracts in support of its novel capsid variant AAV-DB-3 that is being used to develop a potential gene therapy to address somatic expansion in Huntington’s disease (HD). Data presented showcase AAV-DB-3’s capacity to transduce medium spiny neurons and cortical projection neurons in non-human primates and to deliver a microRNA targeting MSH3 to the Q111 mouse model of HD, resulting in the slowing of somatic instability. Latus will also present a novel computational model that allows for potential prediction of the relationship between the level of MSH3 knockdown and its concomitant effects in slowing somatic instability, providing potential insights into the clinical impact of MSH3 knockdown for HD patients.
Title: Direct Comparison of the Engineered Capsid AAV-DB-3 and Wildtype AAV5 in Nonhuman Primate Basal Ganglia and Cerebral Cortex
Presenter: Dr. David Lieb, PhD
Presentation Type: Poster presentation, #1410
Date, Time: May 14, 2025; 5:30-7:00 PM
Title: Targeting MSH3 for Huntington’s Disease: Preclinical Validation of AAV-DB-3-miRNA to Prevent Somatic CAG Repeat Expansion
Presenter: Dr. Bryan Simpson, PhD
Presentation Type: Oral presentation selected from abstracts, #28
Date, Time: May 13, 2025, 2:15 PM - 2:30 PM
Title: A Computational Model Predicts Treatable Huntington’s Disease Patient Population and Potential Effect on Disease Progression for Gene Therapies Targeting Somatic Instability
Presenter: Dr. Paul Ranum, PhD
Presentation Type: Poster presentation, #557
Date, Time: May 13, 2025; 6:00 – 7:30 PM
Finally, Latus will provide an overview of its capsid discovery platform and lead capsids AAV-DB-3 and AAV-Ep+, demonstrating cell- and substructure-specific tropism at potentially industry-leading reductions in dose requirements.
Title: Novel AAV Capsid Variants for Targeted Brain Delivery
Presenter: Dr. Bryan Simpson, PhD
Presentation Type: Invited oral presentation
Date/Time: May 14, 2025; 8:50 -9:15 AM
In total, these studies showcase Latus’ unique capsid discovery platform and ability to identify AAV capsid variants that are optimized for delivery to specific tissues and cell types, seeking to address translational shortcomings to prospectively enable better gene therapies. Latus continues to advance its pipeline of novel AAV capsid variants that target disease-relevant cell types in the central nervous system (e.g., cortex, cerebellum, deep brain, and spinal cord) as well as in peripheral tissues (e.g., ear, eye, heart, kidney and muscle). The Company is developing cutting-edge gene therapies that aim to transform the treatment landscape of genetically defined diseases, including many with high unmet medical needs.
About Latus Bio (Latus)
Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus was founded by technology from Professor Beverly Davidson’s lab at the Children’s Hospital of Philadelphia and is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA.
For more information, visit www.latusbio.com and follow on LinkedIn.
Contacts
Danielle Sliter
info@latusbio.com
