Voyager Therapeutics Special Pilot Project Program

For UMass Chan Faculty:

The University of Massachusetts Center for Clinical and Translational Science (UMCCTS), in collaboration with Voyager Therapeutics, is pleased to announce a special round of Pilot Project Programs focused on understanding and optimizing recombinant adeno-associated viral (rAAV) vectors for therapeutic use.

Voyager Therapeutics is developing gene therapies for fatal and debilitating diseases of the central nervous system. Voyager’s current lead programs include Parkinson’s Disease, Amyotrophic Lateral Sclerosis (ALS), Friedreich's ataxia, Huntington's disease, and additional indications. Voyager is committed to advancing the field of AAV gene therapy by innovating and investing in areas such as vector optimization and engineering, and dosing techniques, as well as process development and production.

Voyager Therapeutics looks for topics that are: 

Targeted Systemic Dosing of rAAVs

Methods of designing rAAV vectors which efficiently transduce target organs or cell types via systemic dosing (e.g., i.v.) while significantly limiting off-target transduction. Target tissues and cells may be in the CNS (preferred), heart, liver or lung. Methods may include (but are not limited to) modifications to the capsid, the transgene cassette, formulation, dosing route, device or other methods.

Preexisting Immunity to rAAV Vectors

Methods of understanding, measuring and avoiding preexisting immunity to rAAV vectors. Topics could include (but are not limited to) a) understanding the effect of preexisting immunity (cellular and humoral) on rAAV vector activity delivered via multiple routes; b) assays for characterizing and measuring rAAV-associated pre-existing and acquired immunity; and c) vectors and methods of designing vectors which can circumvent preexisting immunity; and d) methods to mitigate pre-existing immunity while preserving transduction of target tissues,in particular CNS.

Effect of Anti-capsid T Cell Responses on rAAV Expression

It has been suggested that anti-capsid T cell responses may play a primary role in the limitation on the magnitude and duration of rAAV vector expression after systemic delivery. Studies to determine the effect of such responses on CNS directed delivery and approaches to mitigate those effects would be of interest.

Regulated Expression of the Transgene Product

Studies that use modern/novel molecular biology tools to regulate the expression of the rAAV transgene product are of interest.

Thank you for your interest in the Voyager Therapeutic Innovation Pilot Project Program. We look forward to your ideas.