Latus Bio, Inc. (Latus), a company advancing scalable genetic medicines for broad patient populations, today announced it will present new data at the 29th annual American Society of Gene and Cell Therapy (ASGCT) meeting, taking place May 11-15, in Boston, MA. The Company also announced that its founder, Beverly Davidson, Ph.D., was awarded ASCGT’s highest honor, the Outstanding Achievement Award.
The Company will present one oral presentation and two posters at this meeting. Presentations will focus on Huntington’s disease (HD), including new modeling and preclinical data supporting LTS-201, an investigational AAV gene therapy designed to reduce somatic instability (SI) by lowering MSH3 expression, as well as preclinical safety and biodistribution data for LTS-101, being developed for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
In the oral presentation, the Company will introduce a computational framework that integrates human genetic data and therapeutic parameters to model HD progression and predict clinical benefit. Simulations suggest that SI-lowering therapies may delay motor symptom onset and HD progression by more than a decade when administered early. Further, the magnitude of MSH3 reduction is a key driver of efficacy. Complementary poster data for LTS-201 combine this modeling approach with in vivo studies in nonhuman primates and HD mouse models. These studies demonstrated highly specific biodistribution to disease-relevant brain regions, robust target engagement, and substantial reductions in MSH3 expression at both tissue and single-cell levels. Data support the potential for LTS-201 to provide meaningful clinical benefit in HD.
“These data are highly supportive of the applications of our gene therapy platform to larger disease populations, beginning with Huntington’s disease,” said Jang-Ho Cha, M.D., Ph.D., Chief Scientific and Medical Officer at Latus Bio. “By combining predictive modeling with translational data, we are building a strong foundation for LTS-201 as a potential HD-modifying therapy and are on track to submit an IND in the third quarter of 2026.”
Latus will also present a poster on the results from a GLP toxicology and biodistribution study of LTS-101, an investigational AAV gene therapy for CLN2 disease. In nonhuman primates, LTS-101 was well-tolerated through six months post-administration. The therapy produced sustained, potentially therapeutic levels of TPP1 protein in cerebrospinal fluid and established a no-observed-adverse-effect level (NOAEL) at the highest dose tested, supporting clinical dose selection. These data provide continued validation for Latus’ capsid platform as the Company prepares to initiate first-in-human studies of LTS-101 in 3Q 2026, following its December 2025 IND clearance.
In addition, Latus founder Beverly Davidson, Ph.D., has been awarded the ASGCT Outstanding Achievement Award, the Society’s highest honor, for her major discoveries in the molecular mechanisms underlying fatal inherited brain diseases and her pioneering contributions to gene-based therapeutic strategies. She will deliver a keynote presentation during the George Stamatoyannopoulos Memorial Symposium on Thursday, May 14 at 1:30 p.m. ET. The Company will host a celebration event in her honor on Monday, May 11, prior to her keynote address.
Oral Presentation Details
Title: A computational framework for understanding the therapeutic potential of somatic instability-lowering therapies targeting Huntington’s disease
Abstract Number: 389
Presenter: Paul Ranum, Ph.D., Senior Scientist at Latus Bio
Date: Thursday, May 14
Time: 4:15 p.m. EDT
Poster Presentation Details
Title: Computational modeling and preclinical evidence supporting LTS-201, an MSH3-lowering therapy for Huntington’s disease
Abstract Number: 3455
Presenter: Bryan Simpson, Ph.D., Senior Scientist at Latus Bio
Session: General Poster Session
Date: Thursday, May 14
Time: 5:00-6:30 p.m. EDT
Title: A 3 and 6 month GLP toxicology study in cynomolgus monkeys of a single intracerebroventricular dose of LTS-101 to support development as an AAV gene therapy candidate for treatment of CLN2 Batten disease
Abstract Number: 3482
Presenter: David Leib, Ph.D., Associate Director at Latus Bio
Session: General Poster Session
Date: Thursday, May 14
Time: 5:00-6:30 p.m. EDT
About Latus Bio
Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via innovative and scalable gene therapies. The Company is advancing a broad therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus 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.
About Huntington’s Disease
Huntington’s disease is an inherited neurodegenerative disorder affecting greater than 100,000 patients in major gene therapy markets. It results from a decades-long expansion of DNA repeats in the Huntingtin gene (HTT). Affected patients develop normally but subsequently experience progressive cognitive, neuropsychiatric, and motor changes that result from the accumulation of mutant HTT protein, which leads to loss of medium spiny neurons in the deep brain (caudate and putamen) as well as projection neurons in the cerebral cortex. Currently, there are no approved disease modifying therapies.
About LTS-201
LTS-201 (AAV.DB3.miMSH3) is an investigational AAV gene therapy candidate that encodes an engineered microRNA to reduce the expression of the DNA repair enzyme MSH3. Its design is based on AAV-DB3 – a novel and proprietary AAV capsid variant that targets medium spiny neurons and cortical projection neurons in the central nervous system after administration into the deep brain. A single low-dose injection of LTS-201 is intended to reduce MSH3 expression in these cells, to dramatically slow somatic repeat expansion in mutant HTT, and to slow or halt disease progression. LTS-201 is intended to provide durable benefit for patients affected by Huntington’s disease and has the potential for facile administration at any neurosurgical center.
About Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
CLN2 disease, a form of Batten disease, is an ultra-rare, fatal neurodegenerative disorder affecting approximately 1 in 200,000 children worldwide. It results from a lack or loss of function in the tripeptidyl peptidase 1 (TPP1) enzyme, which helps to break down and remove waste materials from neurons. Beginning at about two years of age, affected children experience developmental delays, loss of motor function, seizures, and blindness, with reduced life span. Currently, the only approved treatment is an enzyme replacement therapy that partially delays disease progression, requires frequent infusions via a surgically-implanted ventricular access device, and is only available at select centers.
About LTS-101
LTS-101 (AAV.Ep⁺.TPP1) is an investigational AAV gene therapy candidate that encodes a functional copy of the human TPP1 gene. Its design is based on AAV-Ep+, a novel and proprietary AAV capsid variant that targets ependymal cells and neurons in the central nervous system after intracerebroventricular (ICV) administration. A single low-dose injection of LTS-101 is intended to restore TPP1 enzyme activity to steady-state levels in the brain and spinal cord. LTS-101 is intended to provide durable benefit for children affected by CLN2 disease with one administration at any neurosurgical center.
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