Voyager Therapeutics is focused on developing innovative genetic medicines aimed at treating various neurological diseases, including Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), and Parkinson’s disease. The company leverages significant advancements in human genetics to target the molecular causes of these conditions. A key aspect of their approach is the TRACER™ AAV capsid discovery platform, which has enabled the creation of novel adeno-associated virus (AAV) capsids that demonstrate enhanced delivery capabilities across the blood-brain barrier. This is crucial for effective treatment of central nervous system disorders, as many therapeutic agents struggle to penetrate this barrier.

The company’s pipeline includes several promising candidates, such as the VY7523 anti-tau antibody and various gene therapies targeting specific genetic mutations associated with neurological diseases. For instance, the VY9323 SOD1 silencing gene therapy aims to reduce the expression of the SOD1 gene implicated in ALS, while the VY1706 tau silencing gene therapy targets tau protein in Alzheimer’s disease. Voyager's research has shown that their therapies can significantly lower tau protein levels and SOD1 mRNA expression in preclinical models, indicating their potential effectiveness. Overall, Voyager Therapeutics is at the forefront of developing transformative neurogenetic medicines that could significantly impact the treatment landscape for neurological diseases.

United States

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com.
 
Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc.

Central Nervous System (CNS)

Metabolic Diseases

Nervous System Diseases

Voyager Therapeutics

Cardiovascular disease

Neurological disorder

Voyager’s TRACER system is a broadly applicable, RNA-based functional &nbsp;screening platform that allows for rapid in vivo evolution of AAV &nbsp;capsids with enhanced tropisms and cell- and tissue-specific &nbsp;transduction properties in multiple species, including non-human &nbsp;primates (NHPs). Initial data from the first of many libraries screened &nbsp;in NHPs demonstrated the proprietary capsid variants effectively &nbsp;penetrated the blood-brain barrier and achieved widespread &nbsp;biodistribution and transduction of multiple regions of the brain. &nbsp;Separate results have demonstrated the ability of certain capsids to &nbsp;transduce cardiac muscle and to de-target the dorsal root ganglia. &nbsp;Voyager is proceeding with additional capsid campaigns derived from AAV9 &nbsp;and other capsid serotypes to identify novel AAV vectors optimized for &nbsp;specific therapeutic applications.

AAV Capsids

AAV Capsids (Pfizer)

AAV Capsids (Novartis)

Prion disease

Voyager’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). TRACER generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach. Separate results have demonstrated the enhanced ability of certain capsids to target cardiac muscle and to de-target the dorsal root ganglia. Voyager is expanding its library of AAV capsids optimized to deliver diverse therapeutic payloads to address a broad range of CNS and other diseases. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.

AAV Capsids + ZF-TRs (Sangamo)

Alzheimer's disease

Neurodegenerative disease

VY-TAU01, a monoclonal antibody, is differentiated from other anti-tau antibodies based on the epitope it targets, which is located in the C-terminal rather than the N-terminal, mid-domain, or microtubule binding region (MTBR) region of the tau protein.

VY7523 (formerly VT- TAU01) [anti-tau antibody]

Central nervous system illness

Parkinson's disease

Voyager’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). TRACER™ generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach, while de-targeting the liver and dorsal root ganglia. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER™ capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.

GBA1 gene therapy program 

NBIb-1817 (VY-AADC) is an investigational recombinant adeno-associated viral (AAV) serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 (VY-AADC) is administered into the brain using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

VY-AADC (NBIb-1817)

Huntington's disease

VY-HTT01 is a gene therapy designed to reduce the expression of huntingtin, &nbsp;thereby altering disease progression. VY-HTT01 is comprised of an &nbsp;adeno-associated virus capsid (AAV1) and a proprietary transgene that &nbsp;harnesses the canonical RNA interference pathway to selectively knock &nbsp;down levels of HTT mRNA. Preclinical data in non-human primates &nbsp;demonstrated robust and durable reduction of HTT mRNA and protein and &nbsp;widespread distribution of VY-HTT01 across the striatum and cortex, &nbsp;which are core areas of disease pathology.
<a href="https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-therapeutics-receives-fda-clearance-ind-application-gene">Source</a>

VY-HTT01

Amyotrophic lateral sclerosis

SOD1 gene therapy: novel TRACER-derived capsid with vectorized anti-SOD1 siRNA. Target: superoxide dismutase 1 (SOD1); mutations in this gene can cause toxic gain of function.

Voyager Therapeutics

Company Details