Voyager Therapeutics Presents New Data Supporting Tau Antibody Program for Alzheimer’s Disease and GBA1 Gene Therapy Program for Parkinson’s Disease at the AD/PD™ Conference
“Voyager continues to advance multiple programs towards the clinic. For our anti-tau antibody program for Alzheimer’s disease, these new preclinical data support our recent selection of VY-TAU01 as the lead development candidate, with an IND filing expected in the first half of 2024,” said
Selection of an Anti-tau Antibody Candidate Targeting Pathological Tau for the Treatment of Alzheimer's Disease (
- Tau pathology, and specifically the neuron-to-neuron transmission of pathologic tau, is hypothesized to contribute to cognitive decline and neurodegeneration in AD. Voyager is advancing an anti-tau antibody that blocks pathologic tau spreading for the treatment of AD.
- Through multiple immunization campaigns that resulted in a pool of 728 candidates, Voyager identified four novel antibodies that met criteria regarding selectivity, affinity, functional inhibition of pathologic tau in vitro and in vivo, and developability. Three of these antibodies target the same epitope on the C-terminal domain of tau, while one antibody targets the mid-domain; all four are differentiated from N-terminal targeted approaches that have previously failed in clinical trials. All four Voyager antibodies demonstrated robust preclinical efficacy in blocking the spread of pathological tau in a P301S seeding-propagation tauopathy mouse model. Ab01 demonstrated superior efficacy (>70% reduction in tau pathology, as presented at AAIC 2022), while N-terminal antibodies typically lack efficacy in this model.
- Ab01 was humanized. After analysis of multiple humanized Ab01 variants, Voyager selected VY-TAU01 as the development candidate based on a number of developability characteristics, including favorable affinity, activity, specificity, stability, and immunogenicity.
Development of AAV-GBA1 gene replacement therapy via single-IV-delivery with a blood brain barrier penetrant AAV capsid (Charlotte Chung, April 1, 2023, 11:55 CET, #1696)
- The GBA1 gene encodes the lysosomal enzyme β-glucocerebrosidase (GCase). Mutations in GBA1 decrease GCase activity and cause increased levels of substrate; these mutations are one of the greatest genetic risk factors for PD. Voyager and Neurocrine Biosciences are advancing a GBA1 gene therapy leveraging a novel, intravenously administered, blood-brain-barrier-penetrant TRACER™ capsid for the treatment of PD.
- Voyager has previously demonstrated that this GBA1 gene therapy approach delivered therapeutically-relevant levels of GCase to the brain in mouse efficacy models, following a single intravenous dose using a blood-brain-barrier penetrant capsid.
- These new data from additional mouse efficacy studies showed that all three candidates demonstrated significant improvement in several efficacy biomarkers.
About Voyager Therapeutics
Voyager Therapeutics (Nasdaq: VYGR) is a biotechnology company dedicated to breaking through barriers in gene therapy and neurology. The potential of both disciplines has been constrained by delivery challenges; Voyager is leveraging cutting-edge expertise in capsid discovery and deep neuropharmacology capabilities to address these constraints. Voyager’s TRACER AAV capsid discovery platform has generated novel capsids with high target delivery and blood-brain barrier penetration at low doses, potentially addressing the narrow therapeutic window associated with conventional gene therapy delivery vectors. This platform is fueling alliances with Pfizer Inc., Novartis and Neurocrine Biosciences as well as multiple programs in Voyager’s own pipeline. Voyager’s pipeline includes wholly-owned and collaborative preclinical programs in Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and Friedreich’s Ataxia, each with validated targets and biomarkers to enable a path to rapid potential proof-of-biology. For more information, visit www.voyagertherapeutics.com.
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “target,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements.
For example, all statements Voyager makes regarding preclinical data for its antibody programs and AAV-based gene therapy programs, including the presentation of preclinical data at the 2023
All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; the ability to initiate and conduct preclinical studies in pre-clinical animal models; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER capsid discovery platform; Voyager’s ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform and Voyager’s pipeline developmental programs; the initiation, timing, conduct and outcomes of Voyager’s preclinical studies; the ability to attract and retain talented contractors and employees; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives.
These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the
Source: Voyager Therapeutics, Inc.