Licensing Opportunities: Gene Therapy & Diagnostics

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Licensing Opportunities: Gene Therapy and Diagnostics for Personalized Medicine in Neurodegenerative Diseases

This portfolio of novel technologies developed by leading researchers at MSU offers new hope: these technologies may improve diagnosis, treatment, and quality of life for patients suffering from neurodegenerative diseases and the negative side effects associated with standard treatments. They provide the potential to both aid symptoms and actually modify these diseases, slowing down—and in some cases even halting—their progression.

Specifically targeted at PD treatment, these gene therapies and diagnostics also hold promise for other neurodegenerative disorders, such as Huntington’s disease, Alzheimer’s disease, and Amyotrophic Lateral Sclerosis (ALS).

 

A primary driver in the progression of Parkinson’s disease (PD) is the degeneration of neuromelanin-containing dopamine neurons. So any therapies that can help to slow such degeneration—or regenerate lost neurons—may have a profound impact on the patient’s health and quality of life. Recent research suggests that axonal degeneration of dopamine neurons may be a critical event in early stages of PD, setting off the subsequent degeneration of neurons. A novel gene therapy from researchers at MSU addresses this event, offering promising new treatment options.

MSU’s technology is a neurorestorative gene therapy that promotes regeneration of dopamine neurons in the central nervous system, which may lead to more robust and successful treatment of PD patients. The gene used in the therapy—SPRR1A—is known to facilitate axonal regeneration of sensory neurons in the peripheral nervous system. It has also been shown to reduce the damage to neurons in the first place.

Work on animal models shows that this gene plays an important role in the degeneration of dopamine neurons in the brain. But a gene therapy directed to SPRR1A may actually help to prevent degeneration in dopamine neurons by preserving their axons. The therapy may also stimulate neural regeneration. The treatment approach may be useful for treating PD as well as other neurodegenerative disorders, including Alzheimer’s and amyotrophic lateral sclerosis (ALS).  

Benefits

  • Disease modifying: MSU’s neurorestorative therapy shows promise for both preventing degeneration of dopamine neurons as well as regenerating lost neurons, helping to alter progression of neurodegenerative disease.
  • Versatile: The gene therapy can be used to treat a wide variety of neurological disorders.
  • Cost effective: Gene therapy treatment may help to lower both direct and indirect healthcare costs for patients suffering from PD and other neurodegenerative diseases.

Licensing and Partnering Opportunity
MSU offers this technology for license as well as further development with commercial pharmaceutical partners.

Review Research Poster

Review Listing in Tech Database

 

While gene therapy offers much promise for Parkinson’s disease and other neurodegenerative disorders, its success is dependent on the efficacy of gene delivery to the target cells. And this in turn is dependent on achieving adequate transduction. Transduction levels are of particular importance in therapies injected directly into the brain or central nervous system (CNS); only a limited volume can be injected in these highly invasive procedures—so those small amounts need to reach their targets to pay off. A new viral gene therapy developed by researchers at MSU addresses this challenge by increasing the efficiency of transduction of cells in the CNS—potentially leading to more effective therapy for neurodegenerative disease.

MSU’s new therapy is an engineered recombinant adeno-associated virus (rAAV2) containing mutant capsids. While rAAV is the leading method of gene therapy for the CNS, it has demonstrated low efficacy in many clinical trials due to insufficient viral transduction of neurons. By contrast, the mutants contained in MSU’s engineered therapy have shown a 3-fold improvement in transduction efficacy in the striatum and hippocampus of rats as compared with non-mutated rAAV capsid.

These laboratory test results point to promising use in improving transduction and gene delivery in the human CNS as well. MSU’s viral gene therapy could provide a more potent and effective therapy for not only Parkinson’s, but also other neurodegenerative disorders such as Huntington’s, amyotrophic lateral sclerosis (ALS), and Alzheimer’s disease.

Benefits

  • Efficient: The therapy improves gene transfer to target cells in the CNS, helping to improve treatment efficacy and dosing requirements.
  • Improved safety: MSU’s development reduces the amount of therapeutic material necessary, reducing potential for immune response and adverse reactions.
  • Long lasting: The gene therapy’s transduction efficiency is very high, reducing the need for repeated dosing to achieve effectiveness.
  • Versatile: The treatment may be useful in managing a variety of neurodegenerative disorders.

Licensing and Partnering Opportunity
MSU offers this technology for license, as well as further development with commercial pharmaceutical partners.

Review Research Poster

Review Listing in Tech Database

 

Accumulation of neurotoxins in the brain and central nervous system (CNS) have been linked to many brain and neurodegenerative disorders. In particular, the neurotoxic metabolites from the kynurenine pathway has been linked to Parkinson’s disease (PD), amyotrophic lateral sclerosis (ALS), Alzheimer’s and Huntington’s diseases, as well as brain ischemia, CNS infections, autoimmune disorders, mood disturbances, and schizophrenia. While the extent to which neurotoxin accumulation may contribute to the development or progression of these conditions is unknown, it is clear that decreasing neurotoxin presence may be helpful in aiding treatment for neurological disorders. New technology from researchers at MSU use a variety of gene therapy vectors to alleviate such toxicity.

MSU’s research is based on the theory that if the kynurenine pathway could be manipulated, the degree to which certain metabolites form could be controlled without disrupting the overall function of the kynurenine pathway. This would bypass the formation of the harmful neurotoxins and instead form benign, or potentially neuroprotective, metabolites. The researchers’ therapy achieves this by overexpressing an enzyme in a cell-specific manner, successfully modulating the kynurenine pathway in various cell populations in the brain.

This process results in the potential for a neurorestorative and/or neuroprotective environment, which may lead to improved efficacy of disease management.

Benefits

  • Neuroprotective: The gene therapy provides a neurotoxin-free environment to help protect existing neurons from damage.
  • Cell-specific targeting: The therapy utilizes a unique library of viral vectors that precisely target specific cells associated with disease, increasing efficacy.
  • Robust: The treatment can be applied to a wide variety of neurodegenerative disease and neurological disorders.
  • Personalized: The technology enables medical providers to treat patients based on their specific disease.

Licensing and Partnering Opportunity
MSU offers this technology for license, as well as further development with commercial pharmaceutical partners.

Review Listing in Tech Database

 

The involuntary, erratic movements in Parkinson’s disease (PD) patients are rarely a result of the disease itself, but rather a side effect of the gold-standard PD treatment—levodopa (L-dopa). Indeed, L-dopa-induced dyskinesia (LID) is seen in more than half of PD patients undergoing prolonged treatment. However, the molecular mechanisms underlying LID have not been well understood by the research community. But new research from MSU offers hope, with evidence that suggests that preventing hyper-expression of particular proteins may inhibit LID development.

In order to better understand the molecular etiology underlying development of LIDs, MSU researchers utilized a full genome array to compare Parkinsonian rats that exhibited LID with rats that had no LID side effects. The research revealed that over-expression of the Nurr1 protein in affected brain areas was linked to LID development. The findings have led to development of a new gene therapy that would be locally applied to the affected striatum with the goal of preventing hyper-expression of Nurr1. This novel therapy has the potential to improve fine-tuned response for motor control in Parkinson’s patients as well as other movement disorders.

Benefits

  • Improved quality of life: The gene therapy may help to lessen LID, thereby improving daily life for PD patients as well as providing prolonged benefits for movement disorders.
  • Personalized treatment: The technology provides insight into how to use Nurr1 as a genetic target in different brain areas, which is useful information for developing a plan for different treatment goals (such as preserving neurons versus preventing LID).
  • Robust research: MSU’s discoveries are useful in the continued development of treatments for neurodegenerative disorders and for ongoing pharmacological research.

Licensing and Partnering Opportunity
MSU offers this technology for license as well as further development with commercial pharmaceutical partners.

Review Research Poster A | Review Research Poster B 

Review Listing in Tech Database

 

Levodopa (commonly called “L-dopa”) has been a gold-standard treatment for Parkinson’s disease (PD) since 1969. While helpful in treating the stiffness, tremors, spasms, and poor muscle control of Parkinson’s patients, L-dopa treatment is unfortunately not without drawbacks. Moderate and long-term L-dopa treatment often results in extended periods in which Parkinson’s symptoms return. In addition, a major negative side effect of the drug is the spontaneous and involuntary movements all too often seen in patients, called L-dopa-induced dyskinesia (LID). A promising new platform technology from researchers at MSU may support therapeutic development to mitigate LID and increase tolerance to L-dopa treatment.

MSU’s technology provides a platform for viral gene therapies that silence the expression of a variety of target genes. Further collaboration with a pharmaceutical partner may also expedite traditional drug discovery efforts to reduce the severity and/or frequency of LID. This is particularly significant when considering that more than half of all PD patients experience LID after 5-10 years of L-dopa treatment. Severe LID may necessitate reduction in L-dopa treatment—but this may worsen the patient’s PD symptoms. MSU’s platform technology offers the promise of new drugs that may help patients experience fewer adverse reactions, enabling them to tolerate higher or more frequent doses of L-dopa. This can significantly improve management of their primary Parkinson’s symptoms, increase their quality of life, and reduce indirect and long-term healthcare costs.

Benefits

  • Research platform: MSU’s technology may provide a useful strategy for future developments in comprehensive PD treatment plans.
  • Research tool: The technology offers a basis for research toward specific drug discoveries that may mitigate LID.
  • Discovery: The high data yield from MSU’s work with this platform technology—especially in collaboration with a pharmaceutical partner—will likely help to improve the data available about target genes linked with LID.

Licensing and Partnering Opportunity
MSU is offering this technology for license as well as further development with commercial pharmaceutical partners.

Review Listing in Tech Database

 

Leading treatments for Parkinson’s disease (PD) include traditional pharmacotherapy (Levodopa) and deep brain stimulation. Both are generally effective in treating the motor symptoms associated with PD, but clinical response to these treatments is highly variable. This is due to differences in the disease’s genotype from one patient to another. A new method developed by leading researchers at MSU is helping shed light on how to pinpoint mutations that lead to more accurate predictions of the most effective therapies for individual patients.

MSU’s technology shows that PD patients who possess a single nucleotide polymorphism respond differently to optimized drug therapy and deep brain stimulation. Given this discovery, the researchers suggest that genotyping to identify this mutation in PD patients may help to inform therapeutic decisions. The information gained may help doctors choose between pharmacological versus surgical management of PD (or some combination of treatments), leading to better patient outcomes, and improved quality of life.

Benefits

  • Improved treatment decisions: MSU’s new method offers medical providers more information with which to make the most considered decisions regarding PD treatment plans.
  • Customized care: This method enables therapeutic plans personalized to the patient’s PD genotype, rather than one-size-fits-all disease management.
  • Improved quality of life: More personalized treatments have the promise to improve overall symptom management, making day-to-day life for PD patients easier and more enjoyable.

Licensing and Partnering Opportunity
MSU offers this technology for license as well as further development with commercial pharmaceutical partners.

Review Listing in Tech Database