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 --

CAMBRIDGE, MA, USA and PARIS, France I February 14, 2019 I Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases, and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that the first patient has been dosed in AAVance, a global Phase 2-3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA).

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 Europe. More information can be found on

“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 Karen Aiach, Founder and Chief Executive Officer of Lysogene.

“MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no approved treatment,” said Principal Investigator Dr. Ronald Crystal, Chairman of the Department of Genetic Medicine and the Bruce Webster Professor of Internal Medicine at Weill Cornell Medicine, and a physician at NewYork-Presbyterian/Weill Cornell Medical Center, who is a paid consultant for Lysogene. “It is a great motivation to know that the work we are doing here has the potential to make a life-changing difference to so many unfortunate children, and I am very much looking forward to further advancing this innovative therapy.”

“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 Doug Ingram, President and Chief Executive Officer, Sarepta. “Sarepta is committed to working with Lysogene to advance this program with the greatest urgency on behalf of patients.”

In accordance with the Worldwide License and Collaboration Agreement signed between Lysogene and Sarepta, the dosing of the first patient with LYS-SAF302 in a Phase 2-3 clinical trial triggers milestone payments of USD18 million from Sarepta to Lysogene.

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.

About LYS-SAF302
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 the United States and markets outside Europe; and Lysogene maintains commercial exclusivity of LYS-SAF302 in Europe.

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

About Lysogene
Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A pivotal clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1-2 clinical trial in GM1 Gangliosidosis is in preparation. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.

SOURCE: Lysogene

La Merie Biologics

FREE Weekly News Bulletin

Sign Up

2018 Sales ofAntibodies & Proteins

New Product Alert

For La Merie Publishing

Sign Up