MRI-guided Gene Delivery

Solving one of the toughest challenges of gene therapy for neurological disorders, scientists are now using intraoperative MRI (iMRI) to deliver your adeno associated viral (AAV)-packaged gene therapy directly to specific parts of the brain. Off target central nervous system side effects such as seizures, headaches, fatigue, or tremors are some of the leading causes of drug development failure. A recent major breakthrough has been the real-time visualization of gene and cell therapy delivery into the brain for the treatment of central nervous system (CNS) disorders. Coupling convection enhanced delivery (CED) techniques with iMRI, there is greater potential to enhance drug delivery to the desired brain regions, while minimizing off target side effects.

A computer graphic image of a brain overlaid a piece of DNA strand

Gene Therapy for Neurological Disorders

Precise, Translational Delivery for CNS Gene Therapies.

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Gene therapy for neurological disorders is an exciting and challenging field. We are fully equipped to support your program. Given the recent advancements in both CNS targeted drugs and delivery techniques, there is significant potential over the next decade for major leaps in gene therapy for neurological disorders. Together, we can continue to advance guided delivery techniques so that your novel therapies will have the precise capacity to go straight to the target in a safer and more effective manner.

Going Past the Blood-Brain-Barrier

Using the ClearPoint® System, we’re taking traditional stereotaxic surgical procedures coupled with modern neurosurgical techniques to bring real-time, iMRI-guided test item delivery to the brain. Real-time iMRI-guided targeting allows for precise, predictable, and reproducible delivery into brain structures with submillimetric (< 0.5 mm) accuracy. By coupling the test item with a contrast agent, visualized delivery allows us to adapt to ensure accuracy and on-target coverage, bringing your test item where it needs to be.

Example Target Sites

Infusion Volume (µL)

Infusion Rates (µL/hr)

Putamen

50 – 250*

120 - 300

Globus pallidus

50 - 125

120 - 300

Thalamus

50 - 125

120 - 300

Caudate Nucleus

20 - 100

120 - 300

Dentate Nucleus

50 - 100

120 - 300

Substantia nigra

10

60

4th Ventricle

400

300

*Up to 200 µL for single site infusion and up to 250 µL (2 x 125 µL) for two infusion sites.

 

A computer graphic image of an oblique view of a probe delivering an AAV-packaged gene to the mid-brain.

Oblique view of a probe delivering an
AAV-packaged gene to the mid-brain.

A computer graphic image of a top view of a probe delivering an AAV-packaged gene to the mid-brain.

Top view of a probe delivering an
AAV-packaged gene to the mid-brain.

 

The ClearPoint® System

Real-time visualization of gene and cell therapy application by equipment such as the ClearPoint® System represents the next stage of CNS surgery and therapy. The ClearPoint® System combines specialized MRI-compatible hardware and software to plan trajectories and align and guide the infusion cannula to target. The cannulas and insertion approach are specifically designed to minimize backflow. Dose volume, infusion rates, and targeting can be optimized throughout this minimally invasive neurosurgery to improve tissue coverage and therapeutic efficacy.

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Choosing the Right Viral Vector

Charles River’s Vigene Biosciences acquisition provides AAV vectors for your Parkinson’s Disease research.

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Disease Indications

The ClearPoint® System is already in use in the clinical environment providing the translational functionality necessary to bridge preclinical safety pharmacology and toxicology with your future patients. The disease indications are countless, but already include brain tumors, epilepsy, Parkinson’s, and other movement disorders.

A computer graphic image of automated methods to obtain coverage estimates of gadolinium, and the targeted brain structure using MRI.

Manual and automated methods are used to obtain coverage estimates overlap of gadolinium and the targeted brain structures.

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From Idea To Lead Candidate

Automated ASO design pipeline and off-target analysis can be built by our partners at Fios Genomics. Bioinformatics allows for generation of all possible length ASO's for targeting and filtering sequences based on multiple parameters.

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Frequently Asked Questions (FAQs) About Gene Therapy for Neurological Disorders