Cluster-busters Against Cancer and Potential Breakthrough in Leber Congenital Amaurosis
09 Sep 2022
CollaborateGene Therapy
TumorGen and PhenoVista Biosciences partnered to explore metastatic cancer cell clusters to validate technology targeting these clusters and preventing cancer metastasis, which causes 90% of cancer-related deaths.
Using PhenoVista’s analytical imaging technology, the two companies, who partnered last year, reported TumorGen’s micro-fluidic technology platform was able to detect multiple metastatic clusters from several lung cancer patients. The platform showed “tremendous sensitivity and specificity,” which proved to be more effective than previous efforts to collect and characterize the clusters.
The clusters contained cancerous and non-cancerous cells, James Evans, CEO of PhenoVista, noted in the announcement. The companies announced the advancements could lead to improved therapeutic options for these patients.
The study, which the National Cancer InstituteCancer Institute supported, notes that these metastasized clusters can be captured. This will allow for multiple analyses that could identify weaknesses and potential therapeutic approaches, which TumorGen dubbed “cluster busters.” This therapeutic could then be able to target the cells and prevent metastasis.
Jeffrey K. Allen, founder and president of TumorGenTumorGen, noted in the company’s announcement that there is a “tremendous unmet medical need” for anti-metastatic therapies that can prevent tumors from forming across the body. He said the company’s platform is able to identify where these metastatic cancer cell clusters are vulnerable, enabling the development of those needed therapeutics.
TumorGen plans to partner with oncology-focused companies to bring new therapies to market.
Gene Therapy Developed for Rare Blindness Disease
Those two companies were not the only ones to offer new seeds of understanding. Researchers from the National Institutes of Health have developed a potential new gene therapy approach to Leber congenital amaurosis (LCA), a disease that causes blindness in children.
A research team from the National Eye Institute, a division of the NIH, revealed that mutations cause a type of LCA in the NPHP5 gene, which leads to defects in the primary cilium found in cells throughout the body, Technology Networks reported. A deficiency in NPHP5 can cause blindness in children. However, the team explained that deficiency could lead to kidney disease in severe cases.
Defects in 25 different genes can cause LCA. The FDA has approved Spark Therapeutics gene therapy Luxturna for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, a form of LCA. That leaves other forms of this disease without treatment.
Using stem cells collected from two patients with NPHP5 deficiency, the NIH team generated retinal organoids, which possess functional features of the natural retina. A deficiency of NPHP5 was found in the retinal organoids, along with lower levels of the protein CEP-290, which works alongside NPHP5.
Using an adeno-associated viral vector that contained a functional version of NPHP5, the NIH researchers were able to target these extracted stem cells and demonstrate a “significant restoration of opsin protein concentrated in the proper location in outer segments.” The NIH research warrants further study and suggests a functional protein could prevent blindness in these children.
Further research is warranted, particularly in more severe forms of the disease.
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