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15 Diseases |  |
0 Trials |
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0 Trials |  |
8 News |  |
- | SAF-301 / Lysogene, Alcyone, LYS-SAF302 / Lysogene, Sarepta Therap
Preclinical, Journal: An improved AAV vector for neurological correction of the mouse model of Mucopolysaccharidosis IIIA. (Pubmed Central) - Mar 14, 2020
Biodistribution of SAF302 was further assessed using GFP (SAF302GFP), indicating that vector spread was limited to the area around the injection tract. Further modification of the injection strategy to a single depth with higher injection volume increased vector distribution leading to more widespread GFP distribution and sustained expression, suggesting this approach should be adopted in future trials.
- SAF-301 / Lysogene, Alcyone
Enrollment closed, Trial primary completion date, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Jan 18, 2017
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
Further modification of the injection strategy to a single depth with higher injection volume increased vector distribution leading to more widespread GFP distribution and sustained expression, suggesting this approach should be adopted in future trials. Completed --> Active, not recruiting | Trial primary completion date: May 2016 --> May 2017
- SAF-301 / Lysogene, Alcyone
Trial completion, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Sep 7, 2016
P1/2, N=4, Completed, Sponsor: LYSOGENE
Completed --> Active, not recruiting | Trial primary completion date: May 2016 --> May 2017 Active, not recruiting --> Completed
- SAF-301 / Lysogene, Alcyone
Trial initiation date, Trial primary completion date, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Mar 24, 2016
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
Active, not recruiting --> Completed Initiation date: Oct 2013 --> May 2013 | Trial primary completion date: Jun 2017 --> May 2016
- SAF-301 / Lysogene, Alcyone
Trial primary completion date, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Apr 15, 2015
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
Initiation date: Oct 2013 --> May 2013 | Trial primary completion date: Jun 2017 --> May 2016 Trial primary completion date: Jun 2014 --> Jun 2017
- SAF-301 / Lysogene, Alcyone
New P1/2 trial, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Jan 30, 2014
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
- SAF-301 / Lysogene, Alcyone
Trial completion, Gene therapy, Viral vector: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome (clinicaltrials.gov) - Jul 8, 2013
P1/2, N=4, Completed, Sponsor: LYSOGENE
Trial primary completion date: Jun 2014 --> Jun 2017 Active, not recruiting --> Completed
- SAF-301 / Lysogene, Alcyone
New P1/2 trial, Gene therapy, Viral vector: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome (clinicaltrials.gov) - Nov 16, 2011
P1/2, N=4, Completed, Sponsor: LYSOGENE