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Spark Therapeutics
unveiled today its first gene therapy program targeting a
disease of the central nervous system (CNS). Findings published in the
current issue of Science Translational Medicine
(http://stm.sciencemag.org/content/7/313/313ra180) provide preclinical proof
of concept for Spark's SPK-TPP1 program. The study demonstrates the
potential of a one-time administration of Spark's gene therapy to delay
onset and progression of a form of Batten disease, a fatal neurological
disorder that begins in early childhood.
The research team was led by Beverly L. Davidson, PhD, director of the
Raymond G. Perelman Center for Cellular and Molecular Therapeutics at The
Children's Hospital of Philadelphia and a Spark scientific co-founder. Their
study showed in a naturally occurring large preclinical model of TPP1
deficiency that one-time administration of recombinant adeno-associated
viral (AAV) vector encoding for TPP1 produced steady expression of the
enzyme in the cerebrospinal fluid, and resulted in demonstrable clinical
benefits across multiple endpoints.
Administration of Spark's AAV serotype 2 vector delivering the TPP1 gene to
the ependymal cells of the brain ventricular system resulted in delayed
onset of clinical symptoms and disease progression, protection from
cognitive decline and extension of lifespan relative to untreated controls.
Importantly, the novel delivery approach used in the study produced
effective distribution of the enzyme throughout the central nervous system,
as evidenced by immunohistochemistry and enzyme activity assay.
Based on these results, Spark plans to initiate IND-enabling studies this
year.
A form of Batten disease, TPP1 deficiency causes severe early childhood
neurodegenerative disease that results in motor and mental decline, seizures
and visual deficits, and leads to death in a majority of cases during early
childhood. The autosomal recessive disease is caused by mutations in the
TPP1 gene, leading to a deficiency of the soluble lysosomal enzyme
tripeptidyl peptidase 1, or TPP1. TPP1 deficiency, can also be known as CLN2
disease, is the first in a broad portfolio of CNS disease targets that Spark
intends to pursue under rights it obtained from the University of Iowa.
The preclinical model described in this study was also used in an enzyme
replacement therapy program currently in development by another company for
the same form of Batten disease. That program subsequently demonstrated
initial clinical proof of concept in early stage human clinical trials.
"TPP1 deficiency exacts a terrible toll on young children and their
families, and effective therapies that slow or stop the progression of
disease are urgently needed," said Dr. Davidson. "We are highly encouraged
by these results, including the clinical impact seen in this study and the
successful use of a novel route of administration of gene therapy to induce
the production of an enzyme like TPP1 throughout the central nervous
system."
"Dr. Davidson's team has made important strides in demonstrating the
potential of one-time administration of gene therapy to alter the course of
this fatal neurodegenerative disease," said Katherine A. High, MD,
co-founder, president and chief scientific officer of Spark. "As we advance
this program toward the clinic, we are building on the success of our
SPK-RPE65 program, including applying the same vector and manufacturing
technology to potentially overcome the formidable challenge of delivering
enzyme replacement therapies across the blood-brain barrier to the CNS."
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