Genistein Clinical Trial

Released November 26 2013

Stem Cell & Neurotherapies Group, Centre for Genomic Medicine, University of Manchester
Willink Biochemical Genetics Unit, Centre for Genomic Medicine, St Mary’s Hospital, Central Manchester University Hospitals NHS Foundation Trust

We will soon be recruiting for a phase III, double blinded, randomised, placebo controlled clinical trial of high dose oral genistein aglycone in Sanfilippo diseases (MPSIIIA, B and C). This is funded by the UK society for Mucopolysaccharide Diseases, The National MPS society and the GEM appeal in a grant to Dr Brian Bigger and sponsored by the Central Manchester Universities Hospitals NHS Foundation Trust. We expect to begin recruiting in early 2014, primarily from within the UK…

To read the entire press release click HERE.

Lysogene Pipeline

 In just under five years and on a shoestring budget, Lysogene conducted a Phase I/II clinical trial for its gene therapy product SAF-301. The Sanfllippo community anxiously awaits the announcement of Phase III.

Visit the Lysogene website for complete details.

http://www.lysogene.com

Shire’s Involvement

Shire, a specialty biopharmaceutical company that develops treatment for patients with life-altering conditions, formed a collaboration with primary investigator Andrea Ballabio and his team at the Telethon Institute of Genetics and Medicine (TIGEM). Together, they discovered the capabilities of the gene TFEB and its impact on Sanfilippo and related diseases. Learn about this program on TIGEM’s website.

http://www.tigem.it/en/research/researchers/andrea-ballabio/lysosomal-storage-disorders-from-mechanisms-to-cure

Shire Enters into Novel Research Collaboration with Italian-based Telethon’s Institute of Genetics and Medicine for Rare Diseases.” Click here for full press release:

http://www.shire.com/shireplc/en/investors/investorsnews/irshirenews?id=632

Shire is currently sponsoring a clinical trial for Sanfilippo Syndrome Type A – HGT2310. Phases I and II are nearing completion and preparation is underway for a Phase III global trial. http://www.shire.com/shireplc/en/rd/pipeline

Nationwide & Abeona Therapeutics

Nationwide to conduct Natural History Study for Sanfllippo Types A and B.

Family Foundations Fund Critical Study Necessary For Clinical Trials And Enter Into Agreement With Nationwide Children’s Hospital To Ensure Data Access For Lysosomal Storage Disease MPS III. http://www.nationwidechildrens.org/Search?query=Sanfllippo +Syndrome&x=16&y=14

The Wall Street Journal published a story covering the families’ heroic efforts to bring treatments for Sanfllippo Syndrome to trial.

Families Push for New Ways to Research Rare Disease

http://online.wsj.com/article/SB10001424127887324432004578306364187833702.html

Abeona Therapeutics is a new biotech aimed at commercializing its two lead compounds ABX-A and ABX-B, and utilizing them in a gene therapy program for Sanfilippo subtypes A and B. To find out more about this collaboration between patient groups, Nationwide, and Abeona, visit http://abeonatherapeutics.com.

Synageva Pipeline

Synageva BioPharma Corporation has several therapeutics in development including enzyme replacement therapies (ERT’s) for lysomal storage disorders (LSD’s).  Of key interest is SBC-103 for the treatment of MPS IIIB.  Watch for progress announcements on Synageva’s website.

http://www.synageva.com/programs-pipeline.htm

Biomarin acquires Zacharon

Zacharon Pharmaceuticals lead program is focused on the oral treatment of Sanfilippo Syndrome. January 2013, BioMarin Pharmaceuticals Inc. announced it’s acquisition of Zacharon.  Zacharon’s Substrate Optimization Therapy (SOT) is an exciting program to treat the central nervous system and peripheral manifestations of Sanfilippo Syndrome and potentially other MPS disorders. For the full press release follow the link.

http://investors.bmrn.com/releasedetail.cfm?ReleaseID=731764

Watch for any new developments on their SOT program on BioMarins website.

Esteve & University of Barcelona

Esteve R & D in collaboration with Fatima Bosch, academic researcher at The University of Barcelona are in the process of developing gene therapy for Sanfilippo Syndrome type A.  For the complete overview follow the link to Esteve’s website.

http://www.esteve.es/EsteveFront/PressRoom.do?op=GR&con=3426&div=idi&con=3426&lng=en