LYSOGENE is a clinical stage pioneering biotechnology company in the field of gene therapy targeting severe genetic pathologies of central nervous system in children.

LYSOGENE Team

LYSOGENE NOW LOOKING FOR ITS CHIEF SCIENTIFIC OFFICER

 

 


 

LYSOGENE is a leading gene therapy biotechnology company developing breakthrough treatments targeting severe genetic pathologies with Central Nervous System involvement and high unmet medical needs.

In 2013, LYSOGENE successfully completed its Phase I/II gene therapy study using its lead product (SAF-301) in patients with Sanfilippo syndrome, More info at: http://clinicaltrials.gov/ct2/show/NCT01474343

Sanfilippo Syndrome is a lethal pediatric neurodegenerative disorder with currently no treatment. It belongs to the group of Lysosomal Storage Disorders and affects 1:100,000 live births.

LYSOGENE aims to expeditiously advance the clinical development and worldwide commercialization of SAF-301.

LYSOGENE is actively expanding its pipeline to additional genetic diseases with high unmet medical needs. Currently, the Company is launching its new gene therapy translational program LYS-XXX. LYS-XXX targets a severe monogenic indication with a strong CNS involvement. The Company aims to translate LYS-XXX to Phase I/II clinical stage in EU and the US no later than 2016.


More information here

 

P1-SAF-301 CLINICAL TRIAL

 

 

 

Four patients have been enrolled in the P1-SAF-301 clinical trial and received the investigational treatment in 2011-2012.

Preliminary data, as of date, provide support that the safety profile of the investigational product SAF-301 is acceptable for use in the treatment of patients with Sanfilippo type A syndrome.

MPS AWARENESS DAY 2013

 

 

 

In order to support the International MPS Awareness Day 2013, LYSOGENE set up different activities targeted to patients and caregivers, medical professionals, researchers, decision-makers and the general public.

We launch a video aimed at communicating about our work and Sanfilippo disease awareness. Additionally, interviews with patient representatives from different countries covering several aspects of their organisations activities were performed.

 
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LAST NEWS

LYSOGENE now looking for its CSO - Chief Scientific Officer

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LYSOGENE AND REGENX BIOSCIENCES ENTER INTO EXCLUSIVE LICENSE AGREEMENT

 

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ESGCT 2013: PRESENTATION OF P1-SAF-301 CLINICAL TRIAL RESULTS

 

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