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Spark: , Its First Program Targeting Neurodegenerative Disease

The following excerpt is from the company's SEC filing.

Preclinical Data Published in

Science Translational Medicine

Demonstrate Delay in Onset and Progression of a Form of Batten Disease

PHILADELPHIA, Nov. 11, 2015 - Spark Therapeutics (NASDAQ: ONCE) unveiled today its first gene therapy program targeting a disease of the central nervous system (CNS). Findings published in the current issue of

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 ons et 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

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

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

gene, leading to a deficiency of the...


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