Latus Bio AAV Gene Therapy LTS-101 Cleared by FDA to Treat Fatal CLN2 Disease

December 02, 2025- Latus Bio, Inc. has announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for LTS-101, an AAV (adeno-associated virus) gene therapy candidate targeting the central nervous system (CNS) manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.

LTS-101 is designed as a one-time AAV gene therapy intended to provide durable expression of the missing TPP1 enzyme in the brain and spinal cord of affected children.

FDA Grants All Possible Designations to LTS-101 AAV Therapy

The FDA clearance is coupled with the granting of Fast Track, Orphan Drug, and Rare Pediatric Disease designations, collectively underscoring the potential of LTS-101 to address a high unmet medical need.

“FDA clearance of our first IND represents a major milestone for Latus,” said P. Peter Ghoroghchian, M.D., Ph.D., CEO of Latus Bio. “The receipt of all possible FDA designations at this stage in development supports the promise of LTS-101 to address the urgent and unmet needs of children affected by CLN2 disease.”

The Fast Track designation provides the benefit of more frequent FDA interaction and potential eligibility for accelerated approval, while Orphan Drug designation offers market exclusivity and financial incentives. The Rare Pediatric Disease designation makes the therapy eligible for a transferable Priority Review Voucher (PRV) upon approval (if the program is renewed).

Novel AAV Capsid Drives Potential for Lower Dosing

LTS-101 utilizes a novel and proprietary AAV capsid variant, designated AAV-Ep+. This specific AAV capsid is designed to target ependymal cells and neurons within the CNS following a single, low-dose intracerebroventricular (ICV) administration.

• Mechanism: LTS-101 (AAV.Ep+.TPP1) delivers a functional copy of the human TPP1 gene. The single low-dose injection is intended to restore TPP1 enzyme activity to steady-state levels, providing durable benefit.

• Convenience: The therapy has the potential for “facile administration at any neurosurgical center,” contrasting with the current treatment options.

CLN2 Disease: A Fatal Neurodegenerative Disorder

CLN2 disease, a form of Batten disease, is an ultra-rare, fatal neurodegenerative disorder affecting approximately 1 in 200,000 children globally.

The disease is caused by the lack of function in the tripeptidyl peptidase 1 (TPP1) enzyme, which is necessary for clearing waste materials from neurons. Children typically begin showing developmental delays, loss of motor function, seizures, and blindness around two years of age, and tragically, often die between the ages of 8 and 12.

The current standard of care is an enzyme replacement therapy that only partially delays progression, requires frequent infusions via a surgically-implanted device, and is available only at select centers. LTS-101 represents a potentially transformative shift toward a one-time, durable AAV treatment option.