23rd Annual Meeting of the American Society of Gene and Cell Therapy
2020 American Society of Gene & Cell Therapy Annual (ASGCT) virtual Meeting Lysogene will present preclinical results for its gene therapy candidate LYS-GM101 for GM1-Gangliosidosis. Abstract: Investigational AAV Gene Therapy LYS-GM101 for GM1-Gangliosidosis Presenter: Ralph Laufer, Lysogene CSO
European Forum for Good Clinical Practice - Digital Tools for Paediatric Clinical Trials
Connecting families coping with all forms of Tay-Sachs, Sandhoff, Canavan and GM-1.
Optimising clinical development strategies for gene therapies and orphan drugs as a whole
Assess the current manufacturing landscape in the context of the COVID-19 pandemic. What are the short and long term implications for advanced therapeutics development?
The 4th Annual Gene Therapy for Rare Disorders Europe is dedicated to overcoming manufacturing, clinical, and commercialization challenges drug developers face when delivering gene therapies to market. Considerations for Selection of NovelEndpoints and Appropriate Controls • Discussing some of the creative manners gene therapy developers have used to optimise clinical trial design • Collecting the […]
Strategy, advocacy and partnering for the orphan drug industry World Orphan Drug Congress 2020 Panel: Optimising clinical development strategies for gene therapy. Full panel to be confirmed. Samantha Parker, Senior Vice President & Chief Patient Access Officer, Lysogene Mathew Pletcher, Head of Rare Diseases, Roche Christeen Moburg, Senior Director, Patient Advocacy, Sangamo Therapeutics
World Sanfilippo Awareness Day is about spreading awareness and sparking conversations globally about Sanfilippo Syndrome, a disease few have heard of. This special day of Awareness is in honor of the children around the world living with Sanfilippo Syndrome today, and those who have passed away. It also honors the families of these precious children. […]