We have identified four promising research areas:
We are always in a review process, vetting new projects to understand research capabilities, budgets, timelines, current funding, and paths to commercialization.
What is...?
Gene Therapy
Genes are the body's instruction manuals. They instruct cells to produce protein to carry out bodily functions. A genetic disorder is characterized by a mutated gene that is unable to instruct the protein to express properly. Gene Therapy includes an array of therapies, all aimed at adjusting the protein expression to normal levels.
AAV9 Gene Therapy
The Adeno-Associated Virus acts as a delivery system to transport healthy genetic material into living tissue, such as the brain. The virus itself does not cause disease and cannot replicate, but it can penetrate human cells and it can cross the blood-brain barrier. Its DNA is replaced with new DNA, making it a vector that can carry healthy genes.
Stem Cell Therapy
Stem cells are taken from bone marrow, or sometimes from umbilical cord blood, of the patient. The cells are genetically modified and then transplanted back into the body, usually intravenously. The modified cells target the affected neurons in order to increase missing protein levels, restoring function.
Repurposed Drug Therapy
Simply put, approved drugs are studied to identify new purposes. Using an approved drug shortens the drug development timeline for the new use, and the FDA approval timeline, and it also decreases any risk associated with testing new compounds. Existing drugs can potentially, as a side effect of their originally intended purpose, alter protein expression of certain genes.
Antisense Oligonucleotide (ASO) Therapy
An ASO-based drug is usually delivered via injection into the cerebrospinal fluid, which results in wide tissue distribution. These short, synthetic chains of DNA/RNA molecules can then target any gene. Once targeted, an ASO can act within a cell to modify gene expression and therefore adjust protein production.
Details: The goal of this project is to identify FDA-approved drugs that will alleviate symptoms associated with FOXG1. This is done using a rapid turnaround, multi-species phenotypic screening approach that starts with the development and phenotypic characterization of genetically personalized yeast strains and culminates in a drug repurposing screen. Success will result in a list of potential drug candidates.
Details: Human hematopoietic stem cells (stem cells that can develop into blood cells) are genetically modified to produce functional FOXG1 protein. These stem cells are then injected intravenously into FOXG1 protein-deficient mouse models, and are therefore introduced throughout the body, including the brain, thereby delivering FOXG1 protein to affected neurons. Successful results will act as proof of concept in order to move to human clinical trials.
Details: The goal of this project is to use an Adeno-Associated Viral 9 vector as a delivery system for gene replacement. "9" refers to the serotype, or variation, of the AAV vector. In using the AAV9 vector, healthy FOXG1 genes are engineered into the vector, which is then injected directly into the brain. Once through the blood-brain barrier, the healthy FOXG1 genes can produce the FOXG1 missing protein due to the mutated FOXG1 gene. Currently, the therapy is in development. From there, there is comprehensive testing with dosing, toxicity and optimization studies. Then, the filing with the FDA. From there, the drug follows a path to commercialization, usually by partnering with a biopharmaceutical company.
Details: The goal of this project is to develop an ASO drug that can target the FOXG1 gene and regulate the FOXG1 protein expression. The project is currently underway with funding at $500k. All research and development is expected to complete by early 2024. At this stage, the drug will submit for FDA approval. The FDA approval process could take up to one year. From there, the drug follows a path to commercialization, usually by partnering with a biopharmaceutical company.
