April 2019, Barcelona

April 27 2019, Barcelona (Spain)

The second meeting took place in Barcelona in April 2019, this time as part of a meeting of the EHDN Advanced Therapies Working group (http://www.ehdn.org/advanced-therapies-wg/).  The aims of the Advanced Therapies Working group overlap extensively with SC4HD, but are broader, extending to delivery of substances in a fluid , such as for gene therapies.  The outcome of the broader group can be found on the EHDN website (http://www.ehdn.org/es/advanced-therapies-wg/).  The second meeting of SC4HD focused on the strategy for forming the network and included discussions of delineating a charter and an overarching framework and pipeline to actively develop guidance.  There is a significant need to define minimum criteria and pre-clinical rationale for disease-specific cell-based therapeutics.  For SC4HD, the key focus is to define the challenges and opportunities specific to HD to provide desired endpoints and criteria to advance stem cell based therapeutics for HD. There are also opportunities for alignment with organizations such as ISSCR and other disease focused networks (e.g. G-force for Parkinson’s disease) for regulatory engagement, general preclinical guidelines, surgical considerations and defined attributes for cell manufacturing.

A charter group of members was formed and a discussion paper planned to build on the knowledge gained since the first HD fetal cell transplant paper 25 years ago.  At this meeting, the aims of SC4HD were consolidated, several goals and focus areas were identified, as listed below, and work has begun on the production of reports and to set up relevant task forces and working groups.

Attendees were:

Irina Antonijevic (Triplet Therapeutics) 
Romina Aron Badin (CEA France)
Neil Aronin (University of Massachusetts Medical school) 
Krystof Bankiewicz (University of California San Francisco) 
Gerard Bauer (UC Davis (remote))
Monica Busse (Cardiff University) 
Josep M. Canals (University of Barcelona) 
Philipp Capetian (University Hospital Würzburg)
Nathalie Cartier (Brainvectis) 
Emanuele Cozzi (Università degli studi di Padova) 
Cheney Drew (Cardiff University) 
Stephen Dunnett (Cardiff University) 
Zdenka Ellederova (Institute of Animal Physiology and Genetics)
Pei Ge (Triplet Therapeutics) 
Steve Goldman (University of Copenhagen)
Liam Gray (Cardiff University) 
Philippe Hantraye (CEA France)
Bernhard Landwehrmeyer (Ulm University)
Mariah Lelos (Cardiff University) 
Christopher Man (Asphalion) 
Laura Mills (Cardiff University)
Unai Perpiña (University of Barcelona) 
Anselme Perrier (INSERM) 
Ralf Reilmann (George Huntington Institut) 
Anne Rosser (Cardiff University) 
Scott Schobel (Roche)
Lorenz Studer (Memorial Sloan Kettering Institute)
Leslie Thompson (University of California) 
Marcos Timon (AEMPS) 
Thomas Vogt (CHDI)