About Event

Welcome to the 4th Annual Gene Therapy for Neurological Disorders

With systemic toxicity challenges plaguing the AAV field, direct administration for CNS indications creates an ideal target for gene therapy. This is an exciting year for the neurological space with clinical data to show and new biotechs coming out of the woodwork to release the next wave of gene therapies with more advanced CNS targeting for a wider selection of neurological targets.

The 4th Gene Therapy for Neurological Disorders meeting is returning to Boston with a completely new agenda to solve the preclinical, translational and clinical challenges of targeting a wide range of neurodevelopmental and neurodegenerative disorders with both ‘first’ and ‘next’ generation gene therapies. Across 2 dedicated tracks of content, this physical meeting will host the fields leading industry scientists and neurosurgeons to cover:

Comparing different case studies of administration route from intrathecal, intra cisterna magna and direct injection into the CNS

Evaluating the dosage and distribution challenges of translating from rodents into NHPs and humans

Discovering novel AAV, lentiviral and non-viral vectors for CNS specificity

Improving the neurological biomarkers being used in preclinical and clinical studies to determine gene therapy transduction and efficacy

Overcoming the clinical challenges of CNS administration and specific regulatory hurdles for gene therapy

Neurosurgical implementation and scale up for surgeons, devices and centres

Discussing the commercial opportunity of targeting different neurological indications – time of intervention, patient population size and age, monogenic vs complex disorders

Join the expanding audience of gene therapy experts seeking to overcome the translational hurdles of developing gene therapies for CNS targets, from discovery, preclinical, translational, clinical, and commercial departments at the likes of Capsigen, AviadoBio, AskBio, Novartis, Prevail Therapeutics, Passage Bio, Voyager Therapeutics, UniQure, Eli Lilly and more.