Latus Bio Achieves Major Regulatory Milestone with IND Clearance and Triple FDA Designations for LTS-101 in CLN2 Disease
Latus Bio, Inc., a clinical-stage biotechnology company at the forefront of adeno-associated virus (AAV) gene therapy innovation, has announced a significant advancement in its mission to treat devastating neurodegenerative disorders. The U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for LTS-101, a novel one-time gene therapy candidate designed to address the central nervous system (CNS) manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease—a rare, rapidly progressive, and fatal lysosomal storage disorder that primarily affects young children.
In addition to IND clearance, the FDA has granted three key regulatory designations for LTS-101: Fast Track, Orphan Drug, and Rare Pediatric Disease. These designations collectively highlight the high unmet medical need in CLN2 disease and affirm the therapeutic potential of LTS-101 to meaningfully alter the course of this devastating condition.
CLN2 disease is caused by mutations in the TPP1 gene, which leads to deficient activity of the tripeptidyl peptidase 1 (TPP1) enzyme. Without functional TPP1, toxic materials accumulate in neurons, resulting in progressive neurodegeneration. Symptoms typically emerge between ages 2 and 4 and include seizures, loss of motor and language skills, cognitive decline, and, ultimately, premature death—often by mid-childhood. While enzyme replacement therapy exists, it requires frequent intracerebroventricular infusions and does not halt disease progression for all patients. A one-time, potentially curative therapy like LTS-101 could transform the treatment landscape.
LTS-101 is engineered using Latus Bio’s proprietary next-generation AAV capsid platform, which enables efficient CNS delivery at significantly lower vector doses compared to conventional AAV gene therapies. The therapy is designed to deliver a functional copy of the TPP1 gene directly to neurons, supporting sustained, endogenous production of the TPP1 enzyme throughout the brain and spinal cord. This approach aims to halt or significantly slow neurodegeneration with a single administration—offering hope for a durable, life-changing intervention.
“FDA clearance of our first IND marks a pivotal moment for Latus Bio and the families we aim to serve,” said Dr. P. Peter Ghoroghchian, Chief Executive Officer of Latus Bio. “Receiving all three FDA designations—Orphan Drug, Rare Pediatric Disease, and Fast Track—at this early stage demonstrates strong regulatory recognition of both the urgency of this disease and the innovation behind LTS-101. Our novel capsid technology not only enhances delivery precision but also reduces the total vector load, which we believe may improve the safety and tolerability profile of gene therapy in pediatric patients.”
The Fast Track designation facilitates more frequent communication with the FDA and opens the door to accelerated approval and priority review, should clinical data support it. The Orphan Drug designation provides Latus with potential financial benefits, including tax credits for clinical trial expenses and seven years of U.S. market exclusivity upon approval. Meanwhile, the Rare Pediatric Disease designation makes the company eligible for a Priority Review Voucher (PRV)—a valuable asset that can be used to expedite review of a future product or sold to another pharmaceutical company, contingent on Congressional reauthorization of the PRV program.
With IND clearance now secured, Latus Bio is preparing to initiate a Phase 1/2 clinical trial in children with CLN2 disease. This first-in-human study will evaluate the safety, tolerability, and preliminary efficacy of LTS-101, with biomarker and clinical endpoints designed to assess neurological stabilization or improvement.
For families facing the heartbreak of CLN2 disease, this milestone represents more than scientific progress—it offers tangible hope. Latus Bio’s advancement of LTS-101 exemplifies how cutting-edge gene therapy platforms, combined with regulatory support, can accelerate the development of transformative treatments for the world’s rarest and most vulnerable patient populations.
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