New data validates tau silencing gene therapy for Alzheimer’s

Tau proteins aggregate to neurofibrillary tangles in a neuron axon

Voyager Therapeutics has announced new data from its two preclinical programmes targeting pathological tau for the treatment of Alzheimer’s disease.

Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy programme will be presented at the upcoming 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD 2024), taking place March 5-9, 2024, in Lisbon, Portugal.

Voyager’s tau silencing gene therapy programme combines vectorised tau-targeted siRNA with a proprietary, blood-brain barrier (BBB)-penetrant capsid derived from the company’s TRACER discovery platform.

The data demonstrate that a single intravenous (IV) administration of one of Voyager’s tau silencing gene therapy candidates in mice expressing human tau resulted in broad AAV distribution across multiple brain regions.

There were dose-dependent reductions in tau messenger RNA (mRNA) levels of up to 90%, which were associated with robust reductions in human tau protein levels across the brain.

Based on these data, Voyager has advanced this programme into late research and expects to file an investigational new drug (IND) application in 2026.

VY-TAU01 anti-tau antibody

Voyager will also report new preclinical data demonstrating that VY-TAU01, its lead antibody targeting pathological tau, and Ab-01, its murine surrogate, were well-tolerated and showed favourable pharmacokinetic profiles following IV administration in NHPs and P301S transgenic mice expressing pathological human tau.

Voyager continues to anticipate an IND filing for VY-TAU01 in the first half of 2024.

“The Voyager team is increasingly excited about the potential for treatments targeting pathological tau to play an important role in improving clinical outcomes for patients suffering with Alzheimer’s disease,” said Todd Carter, Chief Scientific Officer of Voyager Therapeutics.

“Recent third-party data with tau-targeted investigational therapies have demonstrated reduced spread of pathological tau, as measured by tau PET imaging, and favourable trends in cognition. We look forward to continuing to advance our two programmes targeting tau, and observing anticipated data read-outs from others in this space that will play an important role in further validating this target.”

By DIANA SPENCER, Senior Digital Content Editor, DDW

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