AbbVie and Voyager Therapeutics will develop and commercialize vectorized antibodies directed at pathological species of alpha-synuclein.

 Antibody-based drugs can be difficult to develop for neurodegenerative diseases that require frequent systemic injections, because it is difficult to achieve delivery of sufficient quantities of antibodies across the blood-brain barrier (BBB), and very large doses are required.

Voyager Therapeutics is tackling this problem with a vectorized antibody platform and approach designed to deliver –– using BBB-penetrant adeno-associated virus (AAV) capsids –– the genes that encode for the production of applicable therapeutic antibodies. The intended result is to generate higher levels of the therapeutic antibodies in the brain than possible to deliver via systemic administration.

The approach has attracted the attention of AbbVie, which recently announced an exclusive, global strategic collaboration and option agreement with Voyager Therapeutics. The companies will develop and commercialize vectorized antibodies directed at pathological species of alpha-synuclein for the potential treatment of Parkinson's disease and other diseases (synucleinopathies) characterized by the abnormal accumulation of misfolded alpha-synuclein protein.

A key feature of Parkinson’s disease, the second most common neurodegenerative disorder globally, is the accumulation of misfolded alpha-synuclein, which can lead to the formation of protein deposits and progressive neurodegeneration. If the generation of misfolded alpha-synuclein can be inhibited, the progression of Parkinson’s disease could potentially be delayed. The approach may also be useful for other synucleinopathies including Lewy body dementia and multiple system atrophy.

Voyager’s technology allows for the development of AAV gene therapies designed to knock down disease-causing gene expression, increase the expression of missing proteins or enable the expression of therapeutic antibodies through vectorization, according to President and CEO Andre Turenne.

AbbVie will pay Voyager an up-front cash payment of $65 million, up to $245 million in preclinical and phase I option payments, up to an additional $728 million in milestone payments for each alpha-synuclein vectorized antibody compound, tiered royalties on the global commercial net sales of each alpha-synuclein vectorized antibody and up to $500 million in commercial milestones.

Voyager will be responsible for research and preclinical development work to vectorize antibodies directed against alpha-synuclein that are designated by AbbVie. For those candidates selected by AbbVie, Voyager will be responsible for the research, IND-enabling and phase I clinical activities and costs. AbbVie can license the vectorized alpha-synuclein antibody program for further clinical development and global commercialization for indications including Parkinson's disease and other synucleinopathies following the completion of phase I clinical development.