LTS-101 is a one-time AAV gene therapy candidate intended to provide durable expression of the TPP1 enzyme in the central nervous system (CNS) of children with CLN2 disease
FDA designations underscore the potential of LTS-101 to address a high unmet medical need
Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in adeno-associated virus (AAV) gene therapy, today announces clearance by the U.S. Food and Drug Administration (FDA) of the Company’s Investigational New Drug (IND) application for LTS-101 - a gene therapy candidate that’s intended to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The FDA has also granted Orphan Drug, Rare Pediatric Disease, and Fast Track designations for LTS-101.
“FDA clearance of our first IND represents a major milestone for Latus,” said P. Peter Ghoroghchian, M.D., Ph.D., Chief Executive Officer 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. It further underscores the potential of Latus’ novel AAV capsid discovery platform to yield transformative therapies that are enabled via one-time administration and at dramatically lower doses than are typically employed with conventional gene therapies.”
The FDA’s Fast Track designation is granted to new products that demonstrate the potential to treat a serious condition and to address an unmet medical need. It provides a pathway for more frequent interactions with the FDA, eligibility for priority review, and, if relevant criteria are met, the potential for accelerated approval. The Orphan Drug designation offers potential financial incentives, including tax credits and up to seven years of market exclusivity in the U.S. after approval. The Rare Pediatric Disease designation provides Latus eligibility to receive a transferable Priority Review Voucher (PRV) if the rare pediatric disease PRV program is renewed and LTS-101 is approved.
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; they typically die between the ages of 8 and 12. Currently, the only approved treatment is an enzyme replacement therapy that partially delays disease progression, that requires frequent infusions via a surgically-implanted ventricular access device, and that 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.1 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 and has the potential for facile administration at any neurosurgical center.
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 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.
1. SCIENCE TRANSLATIONAL MEDICINE, 14 May 2025, Vol 17, Issue 798 DOI: 10.1126/scitranslmed.adr2531
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