Voyager Therapeutics to Unveil Groundbreaking Gene Therapy Data at ASGCT 2025 Meeting

April 29, 2025
Voyager Therapeutics to Unveil Groundbreaking Gene Therapy Data at ASGCT 2025 Meeting
  • Voyager Therapeutics, Inc. is set to present eight studies at the upcoming ASGCT 28th Annual Meeting, scheduled for mid-May 2025 in New Orleans, focusing on innovative gene therapies for neurological diseases.

  • Among the highlights will be an oral presentation on VY1706, a tau silencing gene therapy that previously achieved an impressive 73% knockdown of tau mRNA in non-human primates with a single intravenous dose.

  • The conference will showcase the efficacy of VY1706, including data from cross-species studies that demonstrate broad central nervous system (CNS) tau lowering in various animal models.

  • Voyager anticipates submitting two Investigational New Drug applications in 2025 and one in 2026, aiming to validate the performance of their capsids in human trials.

  • Key presentations will include an oral report on VY1706's CNS penetrance and tau lowering, scheduled for May 15, 2025, by Rajeev Sivasankaran, Ph.D.

  • The conference will also feature discussions on the efficacy of cross-species blood-brain barrier-penetrant AAV gene therapy and a vectorized anti-amyloid antibody aimed at enhancing CNS expression.

  • Additional presentations will cover an anti-amyloid gene therapy for Alzheimer's disease and enhancements to Voyager's capsids designed to penetrate the blood-brain barrier.

  • Voyager's TRACER capsid discovery platform has enabled the development of novel AAV capsids that effectively cross the blood-brain barrier and transduce a wide range of CNS regions.

  • Data from their TRACER capsids indicate a transduction rate of 43%-98% in neurons and 87%-99% in astrocytes across various brain regions following a single intravenous dose in non-human primates.

  • Voyager's pipeline targets several neurological diseases, including Alzheimer's, ALS, and Parkinson's, with collaborations involving major pharmaceutical partners like Alexion and Novartis.

  • The company is also enhancing its gene therapy approaches with features aimed at immune evasion to broaden the treatment population.

  • The meeting will provide insights into reducing immunogenicity and improving the manufacturability of AAV capsids, including strategies to evade neutralizing antibodies.

Summary based on 2 sources


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