Welcome to the 2nd Annual Gene Therapy for Neurological Disorders
Gene Therapy for Neurological Disorders is dedicated to solving the translational drug development challenges encountered in the development of neurological gene therapies.
Join this definitive, technical conference to delve beyond basic biology to accelerate the development of your gene therapy pipeline and address the significant unmet medical need in rare and common neurological indications.
Learn about innovative, targeted delivery approaches, translatable and clinically relevant animal models and pioneering studies to understand the realities of immunogenicity challenges in the CNS. Leave fully equipped to revolutionize the development and delivery of your neurological gene therapy pipeline.
With insights from AveXis, Sarepta, Voyager, Novartis and more, the agenda will share cutting-edge science on the fields hottest topics including:
- Evaluation of different gene therapy administration routes for more efficient delivery to CNS targets
- Addressing immunogenicity and dosage challenges to identify the optimal therapeutic window
- Introducing novel capsids and advances in capsid engineering for improved cell targeting
- Improving transduction efficiency and exploring advances in technology for monitoring gene expression
- Accounting for limited large mammal model availability and accounting for limitations for improved translation
- Reviewing recent gene therapy clinical successes and failures targeting neurological disorders to overcome translational challenges