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Abeona Therapeutics, Inc. is developing gene therapy based potential cures for the deadly childhood diseases Sanfilippo (SF) Syndrome Type A and Type B. In SF disease, the predominant symptoms occur due to improper cell function within the central nervous system (CNS), which result in cognitive decline, motor dysfunction, and eventual death. Our two lead products, ABX-A and ABX-B, uniquely deliver the therapeutic product to the CNS with the aim of reversing the effects of the genetic errors that cause the disease. ABX-A and ABX-B induce cells in the CNS and digestive tract to produce the missing enzymes and help repair damage caused to the cells.
Safety studies conducted in large animal models have demonstrated that delivery of ABX-A and ABX-B are well tolerated with minimal side effects. Importantly, efficacy studies in animals with Sanfilippo syndrome have demonstrated unprecedented therapeutic benefit months after treatment. A single dose of ABX-A or ABX-B significantly restored normal cell and organ function and increased the lifespan of animals with SF over 100% a year after treatment compared to untreated control animals. Similarly, animals treated with ABX-A and ABX-B demonstrated significant corrections of cognitive defects that remained months after drug administration. These results are consistent with studies from several laboratories suggesting ABX-A and ABX-B treatment could potentially benefit patients with for Sanfilippo Syndrome Type A and B, respectively.
Abeona, in partnership with Nationwide Children’s Hospital (Columbus, OH), has met with the FDA and is planning to initiate clinical studies of ABX-A and ABX-B in patients with Sanfilippo Syndrome Type A and B set to begin in 2014.
LYSOGENE ANNOUNCES COMPLETE ENROLLMENT IN ITS SAF-301 PHASE I/II CLINICAL TRIAL OF INTRACEREBRAL GENE
THERAPY IN CHILDREN WITH SSANFILIPPO TYPE A SYNDROME.
Paris, France – June 14, 2012 – LYSOGENE announced today that the last planned patient in its phase
I/II clinical trial in Sanfilippo Type A Syndrome (NCT01474343) had been treated with SAF-301, its
investigational intracerebral gene therapy product.
SAF-301 aims at treating this pediatric life threatening disease with a high unmet medical need and
currently no cure.
This open-label, single arm, monocentric, phase I/II SAF-301 clinical study is primarily designed to
evaluate the tolerance and the safety of the intracerebral administration of SAF-301 performed in a
single neurosurgical session. It is also designed to evaluate exploratory efficacy neuropsychological,
radiological and biological endpoints from the perspective of future pivotal studies.
“Completing full enrollment and treatment in this phase I/II study is a crucial milestone in the
development of a safe and efficacious gene therapy as what is intended to become the first line
treatment for Sanfilippo Type A disease. It also sustains the extraordinary potential of gene deliverybased
approaches for numerous other monogenic diseases with central nervous system involvement.
Our hope is to bring significant clinical benefits and quality of live improvements to numerous
patients and their families affected with such diseases worldwide”, Karen Aiach, Founder and CEO of
About Sanfilippo Syndrome and LSDs
Sanfilippo Syndrome or Mucopolysaccharidosis III (MPS-III) is a group of four rare autosomal
recessive lysosomal storage diseases of which Type A accounts for approximately two thirds.
Sanfilippo Syndrome is characterized with a heavy central nervous system involvement and an
extremely severe phenotype, associated with a life expectancy reduced to the midst of the second
Sanfilippo Type IIIA Syndrome is seen in approximately 1 in 100,000 live births and affects a few
thousands patients worldwide.
MPS-III belongs to lysosomal storage diseases (LSDs), a group of over 50 inherited disorders, with a
total combined incidence greater than 1 per 8,000 births, of which 70% have a central nervous
LYSOGENE is a platform biotechnology company specialized in the development of intra-cerebral
gene therapy for the treatment of lysosomal diseases affecting the central nervous system, the main
cause of mortality in childhood neurodegenerative diseases.
LYSOGENE brought its first product SAF-301 from bench to the bedside in less than five years.
LYSOGENE – 52, rue la Boétie – 75008 Paris – France – www.lysogene.com