FDA clears late infantile Batten disease gene therapy for testing
LTS-101 a one-time treatment administered directly into brain fluid
LTS-101, a one-time gene therapy designed to treat children with late infantile Batten disease, is poised to enter clinical testing, according to an update from its developer, Latus Bio.
Latus announced that the U.S. Food and Drug Administration (FDA) has granted clearance to its investigational new drug (IND) application for LTS-101. An IND is essentially a formal request seeking permission to begin a trial testing a new therapy in humans. Latus didn’t give any specifics regarding the expected timeline or design of the newly cleared trial.
“FDA clearance of our first IND represents a major milestone for Latus,” P. Peter Ghoroghchian, MD, PhD, Latus’ CEO, said in a company press release.
Batten disease therapy designed to deliver healthy copy of key gene
Late infantile Batten disease, also known as CLN2 disease, is caused by mutations in the gene that provides instructions for making tripeptidyl peptidase 1 (TPP1), an enzyme needed to break down specific fatty molecules. In people with CLN2 disease, the enzyme is absent or dysfunctional, so these fatty molecules build up to toxic levels in nerve cells, driving symptoms such as vision loss and seizures that usually begin in early childhood.
LTS-101 is designed to deliver a healthy copy of the gene encoding TPP1 to nerves and other cells in the brain and spinal cord, thereby restoring TPP1 enzyme activity and facilitating the clearance of toxic fatty molecules, with the ultimate goal of halting disease progression.
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 therapy is administered directly into the fluid in the brain, and it delivers its genetic payload using a viral vector, which is essentially a virus that has been engineered to deliver a therapeutic gene instead of causing infection. The specific viral vector, which is based on the adeno-associated virus (AAV), has been engineered by Latus to deliver the healthy gene to key brain tissues.
The FDA has previously granted LTS-101 orphan drug and rare pediatric disease designations, both of which are designed to offer incentives to drug developers who invest in treatments for rare diseases that affect children. The FDA has also granted LTS-101 fast-track designation, which is designed to accelerate the development of experimental treatments that have the potential to address serious unmet medical needs.
“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,” Ghoroghchian said. “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.”
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