Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)
-- Trial to assess efficacy on neurodevelopmental status of MPS IIIA patients --
AAVance is a single-arm trial aimed at evaluating the effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. MPS IIIA is caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules. LAF-SAF302 is intended to deliver a functional copy of the SGSH gene and allow the brain to secrete the missing enzyme. The goal of the trial is to show improved or stabilized neurodevelopmental status of MPS IIIA patients. The trial will enroll 20 patients at eight sites in the U.S. and
“The first patient dosed in the AAVance trial is an important step in addressing this relentlessly progressing neurodegenerative disease. Our aim is to stabilize or improve the clinical status of patients with MPS IIIA by providing a permanent source of functional enzyme in the brain,” said
“MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no approved treatment,” said Principal Investigator Dr.
“Every day is an opportunity to make progress in bringing transformative treatments to patients and today’s milestone is an important advancement toward that goal,” said
In accordance with the Worldwide License and Collaboration Agreement signed between
About MPS IIIA
MPS IIIA is a rare inherited neurodegenerative lysosomal storage disorder affecting approximately 1 in 100,000 newborns. Inherited in an autosomal recessive pattern, it is characterized by intractable behavioral problems and developmental regression resulting in early death. It is caused by mutations in the SGSH gene, which encodes an enzyme called Heparan-N-sulfamidase necessary for heparan sulfate (HS) recycling in cells. The disrupted lysosomal degradation and resulting storage of HS and glycolipids such as gangliosides leads to severe neurodegeneration. There are currently no treatment options for patients.
LYS-SAF302 is an AAV-mediated gene therapy, the goal of which is to replace the faulty SGSH gene with a healthy copy of the gene. LYS-SAF302 employs the AAVrh10 virus, chosen for its ability to target the central nervous system. Proof-of-concept was established in MPS IIIA pre-clinical models demonstrating strong expression, broad distribution, and the ability of the compound to correct lysosomal storage defects by producing the missing enzyme. Safety data from an IND-enabling toxicity and a biodistribution GLP study showed that, at any dose level evaluated, LYS-SAF302 was not associated with unexpected mortality, change in clinical signs, body weight, behavior or macroscopic findings in the brain. Sarepta holds exclusive commercial rights to LYS-SAF302 in
About Sarepta Therapeutics
Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 5 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit www.sarepta.com.
Sarepta Forward-Looking Statements
This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the design of the AAVance trial and the goal to show improved or stabilized neurodevelopmental status of MPS IIIA patients; the plan to enroll 20 patients at eight sites in the U.S. and
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Lysogene Forward-Looking Statements
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Source: Sarepta Therapeutics, Inc.
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