As reported in Biospace, Taysha Gene Therapies wants to eradicate devastating genetic epilepsies and central nervous system disorders rooted in a single genetic mutation. That’s going to take some innovative biomedical engineering.
In an effort to advance their gene therapy technology to find cures for debilitating genetic epileptic disorders, Taysha Gene Therapies has announced the beginning of multi-year partnerships with Cleveland Clinic and UT Southwestern Gene Therapy Program (UTSW).
The clinical-stage gene therapy company is creating AAV based gene therapies to find CNS and epileptic treatments, but is still working through some of the nitty-gritty of the incredibly innovative nature of the technology.
Now, the three companies will work together to create a first-of-its-kind mini-gene platform, operating with novel ‘mini-gene payloads’ which will give them access to working with a host of minuscule proteins that are associated with the disorder. This will hopefully allow the scientists to tweak methods to correct for some of the long-standing limitations of AAV gene therapy.
Central Nervous System (CNS) Disorders
Central nervous system (CNS) disorders are a category of neurological disorders affecting the core circuit that runs from the brain through the spinal cord. This includes disorders such as Huntington’s disease, Duchenne muscular dystrophy, and Alzheimer’s disease. Because of the variety of diseases, the symptoms vary enormously. People may experience pain, headaches, tiredness, muscle pain, seizures, rapid decline in cognition, and more.
Taysha is focusing on monogenic CNS disorders—the CNS conditions triggered by a singular genetic mutation. This makes these disorders ideal for gene therapy by creating a clear target.
The Partnership: Working Out the Kinks in Gene Therapy
This partnership wants only the best—Taysha Gene Therapies only dedicates their efforts towards developing cures. By way of the first-class gene therapies, they’ve searched for novel solutions to the devastating and currently untreatable epileptic and monogenic CNS disorders.
Taysha tasked the Cleveland Clinic research with creating the innovative mini-gene payloads, a potential remedy to issues with treatments operating on a more general genetic level. The team at UTSW will then construct the vector constructs and test their efficacy on models in the lab. Dr. Dennis Lal of Cleveland Clinic Genomic Medicine Institute and Neurological Institute said,
“By pushing the boundaries of AAV vector engineering, we may be able to overcome some of the challenges inherent with gene therapy and potentially expand the range of treatable genetic CNS diseases with gene therapies. We appreciate the support from Taysha and UTSW in this work.”
Lal recognized that finding these cures would mean they would need to step up to the plate and tackle the nitty-gritty details of very complex biology. Still, he believes that research could uncover a whole mini genetic world, hopefully knit with answers.
Lal continued, “We believe that our proprietary approach to overcoming current limitations of packaging capacity and our access to data on thousands of protein structures associated with a whole host of monogenic CNS disorders has the potential to enable a deep pipeline of functioning mini-genes.”
Click here to learn more about genetic disorders from the Cleveland Clinic.
Leveraging Resources
This partnership is with two of the world’s preeminent leaders in gene therapy innovation, explained Dr. Suyash Prasad, Taysha’s Chief Medical Officer and Head of Research and Development. They hope pooling so many resources will instigate effective progress towards remedying unmet needs that without redress, have dire consequences.
Prasad said,
“This collaboration is designed to leverage our capabilities and synergies with these institutions to pioneer novel approaches to address vector capacity, which is a common limitation when treating genetic disorders associated with large proteins. We look forward to a productive collaboration with the goal of developing treatments with promising benefits to patients with debilitating genetic epilepsies.”
As they dig deeper into their CNS and epileptic disorders, their advancing technology also becomes increasingly adaptable for other disorder, making their findings are meaningful for the entire CNS and epileptic community. They hope that diving into the smallest building blocks will build a base for a whole host of disorders that are currently untreatable.
