CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Ligand Pharmaceuticals | February 21, 2023
In a recent announcement by biopharmaceutical firm, Ligand Pharmaceuticals, the U.S. Food and Drug Administration (FDA) has given accelerated approval for FILSPARITM (sparsentan). This will be helpful in treating adults with primary IgAN condition who are at risk of rapid disease progression, typically defined as a urine protein-to-creatinine ratio (UPCR) of less than 1.5 g/g.
FILSPARI, a once-daily oral medicine, is the first and only non-immunosuppressive therapy authorized for the treatment of IgAN. Two critical pathways are selectively targeted of IgAN (endothelin-1 and angiotensin II) to check disease progression. Up to 150,000 Americans may have the rare kidney illness IgAN, which is the main cause of kidney failure brought on by glomerular disease. Among these patients, it is predicted that 30,000 to 50,000 can be treated using the indication that has received accelerated approval. Travere has stated that it anticipates FILSPARI to be available from February 27, 2023, and it will offer a thorough patient support program all the way through the patient's therapeutic journey.
Eric Dube, Ph.D., President and CEO of Travere Therapeutics, said, “The accelerated approval of FILSPARI is a significant milestone on our path to advancing a transformative treatment for the IgA nephropathy community.” He further added, “As a first-of-its-kind, non-immunosuppressive therapy, we believe FILSPARI has the potential to ultimately become the new standard of care for IgA nephropathy and offer hope to those living with this condition who until now have had few treatment options. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have worked alongside us to develop this innovative first-in-class therapy.”
(Source – Business Wire)
About Ligand Pharmaceuticals
Ligand Pharmaceuticals creates or acquires technologies that aid in the discovery and development of drugs by pharmaceutical firms. The company’s business model generates value for stockholders by offering a diverse portfolio of biotech and pharmaceutical product income streams, that are underpinned by an effective and low corporate cost structure.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Sana Biotechnology | January 30, 2023
Sana Biotechnology, Inc., a firm focused on producing and delivering engineered cells as medicines, recently announced that the FDA has approved the company's Investigational New Drug (IND) application to begin a first-in-human trial of SC291 in patients with different types of B-cell malignancies.
SC291 is an allogeneic CAR T cell therapy that targets CD19. It is developed by Sana using its hypoimmune platform. The hypoimmune platform's objective is to overcome the allogeneic cells' immunologic rejection, which, if valid for SC291, could lead to prolonged CAR T cell persistence and increased rates of durable complete responses in patients with B-cell lymphomas or leukemias.
The hypoimmune platform includes disruption of the major histocompatibility (MHC) class I and class II expression to conceal cells from the adaptive immune system, which consists of antibody and T cell responses and overexpression of CD47 to inhibit activation of the innate immune cell system, especially macrophages and natural killer (NK) cells. The company has provided evidence from numerous preclinical models demonstrating the potential of this platform to cloak cells from immune detection and the therapeutic potential of SC291 for patients with B-cell malignancies.
About Sana Biotechnology
Founded in 2018, Sana Biotechnology is a firm focused on utilizing engineered cells as medicines for patients. It is constructing differentiated capabilities across the spectrum of cell and gene therapy. The company is motivated by three goals as it seeks solutions for people with poor outcomes or diseases that are currently incurable. The first is the ability to repair and control the genes in any cell in the body. It is developing innovative delivery technologies in order to deliver any payload to any cell in a precise, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next comes the ability to develop pluripotent stem cells ex vivo into immune-cloaked functional cells, to replace any missing or damaged cells in the body. Finally, it believes that it can also enable broader access to its therapies by focusing on scalable manufacturing solutions, the manufacturing costs, and aligning with key stakeholders.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Caris Life Sciences | March 09, 2023
Caris Life Sciences®, a leading molecular science and technology firm and Incyte Corporation have recently announced a strategic research partnership to advance precision medicine approaches for Incyte's oncology pipeline.
Caris' innovative technology integrates data from whole exome sequencing, protein analysis, whole transcriptome sequencing and proprietary AI models and signatures to enhance patient outcomes through personalized medicine development. The partnership will initially apply Caris' data and analytics tools, comprehensive molecular liquid and tissue profiling services, and clinical trial enrollment program capabilities spans two therapeutic programs, with the option to expand to four total programs.
Incyte will use Caris' data insights and analytics capabilities to uncover novel biomarkers and enhance clinical positioning strategies for its oncology initiatives. Patients enrolling in Incyte-led clinical trials will be subjected to longitudinal testing with Caris' comprehensive tissue and liquid molecular profiling tests for drug candidates developed as part of the agreement. Incyte will also use Caris' biomarker-driven patient selection for clinical trials, with the possibility of collaborating on creating companion diagnostics for the partnership's programs.
Caris Life Sciences President and Chief Scientific Officer David Spetzler, MS, Ph.D., MBA, commented, "This partnership with Incyte will leverage Caris' leading molecular science and technology solutions to support Incyte's oncology research and development efforts." He further emphasized, "The aggregate strength of our platform, which provides patient level DNA and RNA data both in tissue and blood, may help to better identify and predict patient response to therapy, which in turn may accelerate clinical trial enrollment, optimize clinical positioning and potentially enhance technical and regulatory success."
(Source – PR Newswire)
About Caris Life Sciences
Caris Life Sciences® is a leading molecular science firm that utilizes advanced technologies to revolutionize healthcare. Its suite of molecular profiling offerings, which assess RNA, DNA and proteins, enables patients, researchers and physicians to improve outcomes and save lives by revealing a molecular blueprint. Additionally, it is advancing precision medicine through the use of advanced AI and machine learning algorithms by utilizing its proprietary analytics engine, DEAN™, to develop a better understanding of the molecular mechanisms of cancer. This information helps physicians to better identify and predict patient response to therapy and supports researchers and biopharmaceutical companies in research development, clinical trials, and target identification. With headquarters in Irving, Texas, the company has offices in several locations worldwide, providing services throughout the US, Europe, Asia, and other international markets.
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