Massachusetts Patent of the Month – February 2024

For years, delivering gene therapy to the brain has been a monumental task, thwarted by the formidable blood-brain barrier. This natural defense mechanism shields the brain from harmful substances, but it also presents a significant obstacle for treatments, like gene therapy, that hold immense promise for neurological disorders. But now, Voyager Therapeutics, Inc. has been granted a patent for their development which could revolutionize the landscape of brain treatment: an AAV capsid variant specifically designed to target brain cells with enhanced efficiency.

Traditionally, AAV (adeno-associated virus) capsids, the viral vectors used in gene therapy, haven’t been adept at navigating the blood-brain barrier. This has limited their effectiveness in delivering therapeutic genes directly to brain cells, hindering the potential of gene therapy for neurological conditions.

However, Voyager’s novel AAV capsid variant changes the game. By incorporating a carefully designed “brain-targeting peptide” into its structure, the capsid gains the ability to act like a key, unlocking the door to brain cells and enabling efficient delivery of the therapeutic payload. This innovation translates into several key advantages:

  • Increased Tropism: The capsid variant exhibits significantly increased tropism, or attraction to, brain cells compared to conventional AAV capsids. This translates to higher concentrations of the therapeutic gene reaching the intended target, potentially enhancing treatment outcomes.
  • Greater Precision: The targeted delivery minimizes exposure to other organs, reducing potential side effects and maximizing therapeutic benefit.
  • Broader Applications: This technology holds promise for treating a wide range of neurological disorders, including neurodegenerative diseases like Alzheimer’s and Parkinson’s, as well as muscular and neuro-oncological conditions.
  • The Future of Gene Therapy: Voyager’s innovation paves the way for a new era in gene therapy for brain disorders. By overcoming the blood-brain barrier and delivering gene therapy with greater precision and efficacy, this technology has the potential to revolutionize the treatment landscape for millions of patients worldwide.

Voyager was founded with the intention of developing treatments and solutions for those afflicted by neurological diseases. At present, they have 4 treatments in their wholly-owned pipeline, targeting Alzheimer’s Disease and ALS. They are further involved in partnerships, advancing treatments for a range of diseases including Parkinson’s and Friedrecih’s Ataxia. The medicines in the pipeline target the genetics underlying these various neurological diseases

Are you developing new technology for an existing application? Did you know your development work could be eligible for the R&D Tax Credit and you can receive up to 14% back on your expenses? Even if your development isn’t successful your work may still qualify for R&D credits (i.e. you don’t need to have a patent to qualify). To find out more, please contact a Swanson Reed R&D Specialist today or check out our free online eligibility test.

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