Lysogene Begins Multi-National Observational Study as it Prepares to Initiate Pivotal Clinical Trial for the Treatment of Sanfilippo Type A

PARIS–(BUSINESS WIRE)–#GeneTherapy–Lysogene, a leading, gene therapy clinical-stage biotechnology company,
today announces the enrolment of the first patient in its Sanfilippo A
multi-national observational study (SAMOS); Sanfilippo A is also known
as Mucopolysaccharidosis Type IIIA (MPS IIIA). This is a prospective
natural history study, so no experimental drug will be given. Patients
will receive study-related care. Recruitment is planned before the start
of Lysogene’s gene therapy (LYS-SAF302) trial. The information gained
from this study will enable optimal trial design, greater understanding
of disease progression and better predictions of future therapeutic

The protocol for this observational study is the result of an
international multi-stakeholder collaboration to reach agreement on the
cognitive and related assessments of Sanfilippo Type A. “This study is
critical for comparability of clinical endpoints with the future gene
therapy trial and will complement existing published data,” stated
Soraya Bekkali, Lysogene’s Chief Medical Officer.

“The neuropsychology-focused group organized by Lysogene brought
together renowned experts in the field and afforded a much needed
occasion to consolidate experience,” said Jan Pieter Marchal, Research
Psychologist at AMC Medical Research BV at the University of Amsterdam.
“It was a milestone for participants who rarely meet in such early
stages of protocol design.”

“Lysogene is proud to collaborate with MPS
(Patient Access to Clinical Trials) for travel arrangements for
families to clinical study sites,” said Samantha Parker, Lysogene’s
Chief Health Policy and Patient Access Officer. “They will provide a
safe and independent logistical resource to families of children
recruited to the study.”

Lysogene conducted a phase I/II clinical trial, in 2011-2012, with its
first generation gene therapy formulation LYS-SAF301. Currently,
Lysogene is planning a clinical trial with its next generation
formulation LYS-SAF302. LYS-SAF-302 has the potential to replace the
defective gene in the cells, of Sanfilippo Type A patients, which will
allow for the production of the enzyme and prevent the progressive
nature of the neurological damage. Individuals included in the
observational study will be provided the opportunity to enroll in
subsequent trials if they satisfy the specified enrolment criteria.
However, participating in the observational study does not guarantee a
place in the gene therapy clinical trial.

About Mucopolysaccharidosis Type A (also known as Sanfilippo A):

MPS IIIA is a lysosomal disease caused by an autosomal recessive defect
of the SGSH gene and affecting approximately 1:100,000 live births. MPS
IIIA presents in early childhood, causing progressive neurodegeneration
associated with intractable behavioral problems and developmental
regression. Life span is shortened, and there is currently no treatment.

About Lysogene

Lysogene is a clinical stage biotechnology company pioneering in the
basic research and clinical development of AAV gene therapy for CNS
disorders with a high unmet medical need. Since 2009, Lysogene has
established a unique platform and network, with lead products in
Sanfilippo A and GM1 Gangliosidosis, to become a global leader in orphan
CNS diseases.

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Marion Janic, +1 212-223-4017