Successfully reported this slideshow.
We use your LinkedIn profile and activity data to personalize ads and to show you more relevant ads. You can change your ad preferences anytime.

2016 BDSRA Mole CLN1, CLN2, CLN3, CLN5, CLN6, CLN7,

493 views

Published on

Developing new therapies for Batten disease

Published in: Health & Medicine
  • Be the first to comment

  • Be the first to like this

2016 BDSRA Mole CLN1, CLN2, CLN3, CLN5, CLN6, CLN7,

  1. 1. 8  experimental  WPs:   NEW  MODELS   Development,  validation and optimisation of essential  new models &  tools PATHWAY  LEADS   Identifying therapeutic target pathways using genetics &  biology METABOLOME   Identifying therapeutic target pathways and developing new diagnostic &   monitoring techniques using metabonomics NATURAL  HISTORY   Natural  history of the brain and beyond COMPOUND  LEADS   Identifying new therapeutic compound leads ZEBRAFISH  TRIAGE  Determination  of therapeutic potential  using zebrafish GENE  THERAPY   Gene  therapy for the brain,  eye and other organs DRUG  THERAPY   Delivering new small molecule therapy to the mouse 3  facilitating  or  supporting  WPs: Patients’  Organisation  involvement Management Ethics Developing  new  therapies  for  Batten  disease PARTNERS BATCure Consortium Coordinator  -­ Sara  E  Mole MRC  Laboratory  for  Molecular  Cell  Biology,  UCL  Institute  of  Child  Health  and  Department  of  Genetics,   Evolution  &  Environment,  University  College  London,  London  WC1E  6BT,  UK.  s.mole@ucl.ac.uk CONCEPT ORGANISATIONAL  STRUCTUREPROJECT  DESIGN 4-­5  Tasks/WP 90  Deliverables 50  Milestones Disease&Models& Iden-fica-on&of& Surrogate& Markers& Lead& Iden-fica-on&&& Op-miza-on& Drug&&&Gene& Therapeu-c& Strategies& Prepare&for& Clinical&Trials& WP01&+&WP04& WP02&+&WP03& WP05&+&WP06& WP07&+&WP08& WP09& PreNdiscovery& Discovery& PreNclinical& Disease&Models& Iden-fica-on&of& Surrogate& Markers& Lead& Iden-fica-on&&& Op-miza-on& Drug&&&Gene& Therapeu-c& Strategies& Prepare&for& Clinical&Trials& SMALL MOLECULE UCL, AcureO, USAL, Orphazyme Leads that rescue yeast & zebrafish models Leads from disease biology GENE$THERAPY$ UCL Toxicity CLN3, CLN6 Vectors CLN3, CLN6 Special Responsibility Leads: Communications Dissemination Public Engagement Ethics Exploitation & Innovation Executive Board General Assembly (Decision Making) Participating academic, SMEs & Patient Organisations Work Package Leaders Researchers External Expert Advisory Board Coordinator (UCL) Scientific Coordinator (Mole) Project Manager (Ellis) Project Support (ERIO) Coordina(on) &)Resources) Advice) Repor(ng) Repor(ng) Liaison) Leadership) Advice)&)Support) Repor(ng) Repor(ng) &)Admin) Advice)&) Support) Advice)&) Support) BATCure) BATCure Novel&Models&&&Tools Effective&Treatments&&&Diagnosis Batten disease Understanding&&Awareness WP01&New&Models WP07&Gene&Therapy WP05&Compound&Leads WP10&Management WP11&Ethics WP02&Pathway&Leads WP03& Metabolome WP04&Natural&History WP09&PO&Involvement WP06&Zebrafish Triage WP06&Drug&Therapy BATCure transmembrane:   CLN3  CLN6  CLN7   CLN8   soluble  lysosomal:   CLN1  CLN2  CLN5   BATCure will investigate the natural history of Batten disease, elucidate the function of key proteins and determine disease mechanisms, as well as develop new therapies for three forms of the disease. Batten disease is one of approximately 50 lysosomal storage disorders, in which genetic mutations disrupt the cells ability to recycle waste. Children and young adults with Batten disease suffer progressive neurological impairment, which includes: seizures, visual impairment or blindness, personality and behaviour changes, dementia, loss of motor skills and loss of the ability to walk, talk and communicate. There is currently no treatment. This consortium brings together ten leading scientific research groups, three companies and one patient organisation from across Europe, with half applying their expertise and skills to Batten disease for the first time. This  project  has  received  funding  from  the  European  Union’s  Horizon  2020  research  and  innovation  programme under  grant  agreement  No  666918 H2020-­PHC-­14-­2015:  New  therapies  for  rare  diseases Project:  666918 Duration:  36  months Start  date:  1/1/2016 Award:  €5  995  769 Web  site:   www.batcure.eu Twitter:  @BAT_Cure Facebook:  BATCure

×