Latus Bio Advances Gene Therapy Programs for Rare CNS Diseases

PHILADELPHIA & BOSTON, April 28, 2026

Latus Bio, Inc. has announced new preclinical and computational data supporting its investigational gene therapy programs for Huntington’s Disease (HD) and CLN2 disease, ahead of presentations at the 29th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting in Boston. The company will showcase progress for LTS-201, an AAV gene therapy targeting Huntington’s disease, and LTS-101, an investigational therapy for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease), while also celebrating founder Beverly Davidson, Ph.D., recipient of ASGCT’s highest honor, the Outstanding Achievement Award.

LTS-201 Shows Strong Potential for Huntington’s Disease

Latus Bio’s lead Huntington’s disease program, LTS-201, is designed to reduce somatic instability (SI) by lowering MSH3 expression, a key biological driver of disease progression. New computational modeling presented by the company integrates human genetic data with therapeutic parameters to predict clinical outcomes. Simulations suggest that therapies reducing somatic instability may delay motor symptom onset and disease progression by more than a decade, especially when treatment is administered early. Importantly, the level of MSH3 reduction was shown to be a critical factor influencing therapeutic success.

Complementary preclinical studies in nonhuman primates and HD mouse models demonstrated strong biodistribution to disease-relevant brain regions, robust target engagement, and significant reductions in MSH3 expression at both tissue and single-cell levels. These findings support the potential for LTS-201 to become a disease-modifying therapy for Huntington’s disease, a major unmet need in neurodegenerative medicine.

IND Submission Planned for LTS-201 in Q3 2026

According to Chief Scientific and Medical Officer Jang-Ho Cha, M.D., Ph.D., the combined predictive modeling and translational data provide a strong scientific foundation for clinical advancement. Latus Bio is now preparing to submit an Investigational New Drug (IND) application for LTS-201 in Q3 2026, positioning the therapy for entry into human clinical studies later this year.

This milestone represents a major step for the company as it expands its gene therapy platform into broader patient populations beyond ultra-rare disorders, targeting larger neurodegenerative diseases with scalable AAV-based therapeutic solutions.

LTS-101 for CLN2 Disease Advances Toward Human Trials

Latus will also present important new data for LTS-101, its investigational AAV gene therapy for CLN2 disease, a rare and fatal pediatric neurodegenerative disorder. In GLP toxicology and biodistribution studies conducted in nonhuman primates, LTS-101 demonstrated strong tolerability through six months post-administration and produced sustained, potentially therapeutic levels of TPP1 protein in cerebrospinal fluid.

The therapy also established a No-Observed-Adverse-Effect Level (NOAEL) at the highest dose tested, supporting dose selection for future trials. Following IND clearance in December 2025, Latus is preparing to initiate first-in-human studies for LTS-101 in Q3 2026, further strengthening its rare CNS disease pipeline.

In addition, founder Beverly Davidson, Ph.D. received the prestigious ASGCT Outstanding Achievement Award for her pioneering contributions to gene-based therapies for inherited brain diseases, reinforcing Latus Bio’s scientific leadership in the field.

With strong translational data, regulatory progress, and platform scalability, Latus Bio is positioning itself as an emerging leader in next-generation CNS gene therapy, aiming to deliver transformative treatments for patients with devastating neurological disorders.

Source: Latus Bio press release