According to STAT news “ Novartis and The Gates Foundation aim to create more practical sickle cell gene therapy” Novartis NYSE:NVS and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.
The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.
Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.
Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.Vertex Pharmaceuticals NASDAQ:VRTX has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics NADAQ: BEAM, have also embarked on gene therapy development.
The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.
Avalon GloboCare Corp. (NASDAQ: AVCO) (Avalon or The Company), a clinical-stage global developer of cell-based technologies and therapeutics, today announces the expansion of its co-development research program with the Massachusetts Institute of Technology (MIT) to apply two leading technologies—CRISPR-based genome editing and QTY protein design—to potentially treat and prevent cancer metastasis.
Metastasis—the spreading of cancer cells from the initial tumor site to surrounding tissues and distant organs—is responsible for the vast majority of cancer deaths. In an ongoing co-development research program, Avalon and the laboratory of Dr. Shuguang Zhang at MIT’s Media Lab have developed a novel approach to potentially block the signals that cancer cells require to metastasize in order to prevent cancer spread and death. Cancer cells mobilize and travel to other organs using the chemokine signaling network, made up of chemokine receptors on the surface of healthy cells and soluble ligands that bind these receptors. Dr. Zhang’s MIT laboratory is applying their novel QTY protein code technology to design truncated versions of receptors that can act as decoys, attracting cancer cells and preventing them from spreading to other organs. Avalon and MIT researchers are also working on a second approach—combining the QTY technology with the CRISPR-Cas9 gene editing system to re-program cell types that typically stimulate nearby cancer cells to spread, thereby deterring cancer metastasis.
Avalon and MIT’s collaboration on the QTY code has already successfully generated a prototype device to soak up the excess chemokines and cytokines produced in the body that lead to a potentially fatal ‘cytokine storm,’ which can occur in patients with COVID-19 and in cancer patients being treated with CAR T-cell therapy.
“I am delighted to expand this fruitful and important collaboration with Avalon with the goal of advancing our ability to treat and prevent cancer metastasis,” said Dr. Shuguang Zhang, Head of the Laboratory of Molecular Architecture at MIT’s Media Lab. “We have a talented team at MIT, including the enthusiastic support of MIT and Harvard’s Broad Institute professor, Dr. Feng Zhang, a pioneer and expert of CRISPR-based genomic editing in mammalian cells. Through our collaboration, we have been able to develop cutting edge technology that we believe has the potential to prevent the spread of cancer to other tissues, which ultimately leads to death.”
In the first quarter of 2020, Avalon GloboCare (NASDAQ: AVCO) successfully completed a phase 1 first-in-human clinical study of AVA-001 in China for the treatment of relapsed refractory B cell acute lymphoblastic leukemia (R/R B-ALL). 90% of R/R B-ALL patients achieved complete remission with one dose and within one month of treatment, and then proceeded to a curative-intent allogeneic bone marrow transplant. Also there was minimal and well tolerated side effects with little neurotoxicity or cytokine release. This paradigm of bridging CAR-T cell therapy to bone marrow transplant creates a new horizon with potentially ground breaking commercial application for patients with relapsed/refractory B-ALL and other hematologic cancers.
Avalon Globocare also announced a collaboration with the University of Pittsburgh Medical Center for development of their FLASH-CAR™ RNA-based cellular therapies includes utilization of Avalon’s new Point-of-Care Modular Autonomous Processing System (PMAPsys™). First FLASH-CAR™ candidate, AVA-011, on-track for clinical study in B-cell lymphoblastic leukemia and non-Hodgkin’s lymphoma patients. The FLASH-CAR™ technology modifies patients’ T-cells and natural killer (NK) cells using a ribonucleic acid (RNA)-based platform rather than a viral vector, and is being co-developed with the Company’s strategic partner, Arbele Limited. The adaptable FLASH-CAR™ platform can be used to create personalized cell therapies from a patient’s own cells, as well as “off-the-shelf” cell therapies from a universal donor. By avoiding viral vectors and complicated bio-processing procedures, the FLASH-CAR™ technology significantly reduces manufacturing costs and development times, resulting in more affordable and potentially breakthrough therapies for cancer patients. Avalon’s innovative FLASH-CAR™ technology can be used to generate universal cell therapies that may allow for widespread patient accessibility enabling broader commercial adoption compared to currently available CAR-T cell therapies. Avalon’s first FLASH-CAR™ platform candidate, AVA-011, targets both CD19 and CD22 tumor antigens on cancer cells and is in development for patients with relapsed/refractory B-cell lymphoblastic leukemia and non-Hodgkin’s lymphoma.
“This is an important milestone in the clinical development and production of AVA-011 and other RNA-based FLASH-CAR™ candidates ahead of the start of our first-in-human clinical trials,” said David Jin, M.D., Ph.D., President and Chief Executive Officer of Avalon GloboCare. “We are excited about our partnership on the PMAPsys™ with UPMC, which we believe will help accelerate the path to bringing our own and third-party cell therapies to market. We are working diligently to fast-track development and commercialization of our cellular immune-oncology therapeutic products, while providing the highest quality and safety standards for our patients.”
Avalaon GloboCare (NASDAQ:AVCO) is teaming up with some of the brightest minds in CAR-T and CRISP based technologies including Dr. Yen-Michael Hsu, M.D., Ph.D., Director of the Immunologic Monitoring and Cellular Products Laboratory at the UPMC Hillman Cancer Center and Dr. Shuguang Zhang at MIT’s Media La to rapidly develop potentially lifesaving technological advancements with major commercial applications. Recently some of the smartest money on Wall Street has been extremely bullish on this specific niche including Catherine Wood at ARK invest, in a recent interview she stated that her ARKG (ARK Genomic) ETF has the most potential upside over the next five years as the fund has been deploying billions to companies that produce or enable CRISPR, another Targeted Therapeutics. Over the past few weeks microcap stocks have been under more than usual pressure resulting in Avalon GloboCare NASDAQ (AVCO) to be trading at their 52 week low at just around S1.00 a share. I strongly believe Avalon’s pipe line along with a market cap of less than 100 million creates a highly asymmetric risk reward opportunity.