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.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Lineage Cell Therapeutics, Inc. | February 23, 2023
On February 22, 2023, Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology firm focused on the development of allogeneic cell therapies to restore and replace specific cell types of the human body, announced that it has entered into an exclusive option and license agreement with Eterna Therapeutics Inc. (Eterna) for the development of innovative beta 2 microglobulin (B2M)-deficient induced pluripotent stem cell (iPSC) lines that Lineage will assess for the creation of unique, differentiated cell transplant therapies.
These new cell lines to be developed by Eterna are expected to help Lineage generate more product candidates for the treatment of central nervous system (CNS) conditions and other neurology indications. Eterna holds the exclusive license for the crucial intellectual property associated with this partnership, which it obtained from its discovery partner Factor Bioscience.
Following the agreement, Eterna intends to perform specific gene-editing activities and furnish materials to Lineage for assessment. In addition, the agreement grants Lineage the option to obtain an exclusive license to use and sublicense the new gene-edited cell lines for clinical, preclinical, and commercial purposes in the field of CNS diseases. A distinctive feature of the starting cell line is the targeted elimination of the B2M gene, which is aimed at minimizing the immunogenicity of product candidates generated from the cell lines by inhibiting rejection by CD8+ T cells.
Lineage anticipates that this attribute will enhance the overall usefulness of the edited cell lines, including non-immune privileged or non-human leukocyte antigen (HLA) matched indications. Other proposed gene modifications may additionally differentiate the cell line from those currently utilized by competitors. No details of the financial agreement were disclosed.
About Lineage Cell Therapeutics, Inc.
Founded in 1990, Lineage Cell Therapeutics is a clinical-stage biotechnology company focused on developing novel cell therapies to address unmet medical needs. The company's programs are based on its proprietary cell-based therapy platform and related development and manufacturing capabilities. By using this platform, it specializes in the development and manufacture of specialized, terminally-differentiated human cells from its progenitor and pluripotent cell starting materials. These differentiated cells are intended to either support or replace cells that are dysfunctional or absent due to degenerative disease or traumatic injury and can also be administered to help the body mount an effective immune response to cancer.
Premier Research | March 14, 2023
Premier Research has partnered with InSilicoTrials, a leader in using artificial intelligence (AI) and computational modeling and simulation (CM&S) to speed up the development of new therapies and medical devices. Premier Research helps biotech and device companies take their ideas from the idea stage to the commercialization stage. The aim of the partnership is to make it easier, faster, and safer for treatments for rare diseases to get approval from the government.
CM&S and AI have made it possible to use virtual patients in clinical trials in whole or in part, which the FDA approves of in some cases. In silico trials make it possible to create fake control or treatment arms, help figure out how to sign up patients, and predict the safety and effectiveness of new drugs and medical devices more accurately.
The use of synthetic control arms generated using in silico CM&S techniques represents a powerful tool for enabling non-feasible rare disease research, both preclinical and clinical research. In the last five years, Premier Research has been in charge of more than 240 rare disease studies for a wide range of indications. This shows that the company is committed to orphan drug development for a long time. Premier Research is continuing to put money into trials for rare diseases by partnering with InSilicoTrials.
One major advantage of in silico simulation is the ability to investigate 'what if' scenarios, particularly in rare diseases where a larger patient population does not exist. By using biological, molecular, or genetic data to describe rare diseases, in silico models can make synthetic control arms. This, along with Premier Research's expertise in clinical and preclinical development, execution, and regulatory aspects of small population trials, can shorten the time it takes to make effective treatments for rare diseases.
The partnership between Premier Research and InSilicoTrials will focus on smarter in silico study design, leading to a more effective pre-clinical review of trial design parameters and faster submission-ready studies. CEO of InSilicoTrials Luca Emili says that combining modeling and simulation with AI is the best way to improve the R&D process in drug development. This is because it allows sponsors to speed up the development of new treatments, make medical products safer, and lower R&D costs.
About Premier Research
Premier Research is a global company that does clinical research, product development, and consulting. It focuses on using new technologies to design and run smart studies and trials. With more than 2,500 employees in 75 countries, it has a lot of experience in certain therapeutic areas and uses. The company is committed to empowering its customers to meet the unmet needs of patients and offers great benefits and flexible working conditions.