Research Objective
A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells
Impact
Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS
Major Proposed Activities
This new therapeutic will address a significant unmet medical need in the treatment of amyotrophic lateral sclerosis (ALS) and have important benefits to the patients with ALS and impact on the healthcare and bio industry in California.
Research Objective
AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons.
Impact
Angelman syndrome is a rare (1 in 15,000 births) neurogenetic disorder caused by loss of UBE3A in the brain, causing severe developmental delay, ataxia and epilepsy. There are no treatments or cures.
Major Proposed Activities
In addition to directly benefiting the ~2,500 children and families living with Angelman syndrome in California, this gene therapy with a first-in-kind mechanism of action could bring new treatments and new opportunities to our state. California has long been a hub of innovation, and creates an environment in which new technologies can be born, fostered, and attract others who want to create a better future for our residents. This activity will help train some of them, and inspire many others.
Research Objective
These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition.
Impact
Stem cell-derived extracellular vesicles will address the confounders of stem cells (tumors, immunorejection, immunosuppression) & mitigate debilitating side-effects of cancer therapy on the brain.
Major Proposed Activities
In California, nearly 187,000 patients diagnosed with cancer will be alive in 5 years & more than 1.88 million have a history of cancer. Importantly, adult & childhood cancer survivors suffer from severe & persistent cognitive deficits that adversely affect their quality of life (QOL). A stem cell-based therapeutic could reduce inflammation & restore the cognitive function that may significantly improve patient’s QOL, reduce financial hardship on patients, caregivers & the state of California.
Research Objective
This grant proposes development of a stem cell based therapy that is derived from human induced pluripotent stem cells. These cells are in the form of a brain support cell, an astrocyte.
Impact
The cell candidate will treat vascular dementia, the second leading cause of dementia, and stroke by overcoming a bottleneck in the ability to make large quantities of the cells for clinical use.
Major Proposed Activities
This research will develop a therapy for a disease with no treatment, vascular dementia, that is common and devastating in its consequences. The intellectual property for this therapy is held by a State of California public university (UCLA) and commercialization will directly benefit the State of California.
Research Objective
A cell therapy product comprised of inhibitory neurons that can migrate, integrate and restore neurologic function after traumatic brain injury.
Impact
Traumatic brain injury
Major Proposed Activities
Nearly 6 million Americans - including 700,000 Californians - live with permanent physical or mental health problems resulting from a traumatic brain injury, but there are no treatments. We propose studies to create a cell therapy product that is capable of restoring neurologic function to these patients.
Research Objective
We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS).
Impact
ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients.
Major Proposed Activities
ALS is a fatal, incurable disease and California has one of the highest number of ALS patients of any state. By testing our drug on stem cell-derived nerve cells from Californian ALS patients, we will increase the chances that it will be effective on the types of ALS patients found in California. If successful, our drug will substantially slow or stop ALS disease progression.
