Combination Gene Therapy Could Treat Multiple Age-related Diseases

Technology Networks | November 05, 2019

As people age, they tend to develop diseases such as heart failure, kidney failure, diabetes, and obesity, and the presence of any one disease increases the risk of developing others. Traditional drug treatments, however, each target one condition. That means patients often have to take multiple medications, increasing both the risk of negative side effects and the likelihood of forgetting one. New research from the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School (HMS) suggests that it may be possible someday to tend to multiple ailments with one treatment. In the Wyss study, a single administration of an adeno-associated virus (AAV)-based gene therapy, which delivered combinations of three longevity-associated genes to mice, dramatically improved or completely reversed multiple age-related diseases, suggesting that a systems-level approach to treating such diseases could improve overall health and lifespan. The research is reported in PNAS. “The results we saw were stunning and suggest that holistically addressing aging via gene therapy could be more effective than the piecemeal approach that currently exists,” said first author Noah Davidsohn, a former research scientist at the Wyss Institute and HMS who is now chief technology officer of Rejuvenate Bio. “Everyone wants to stay as healthy as possible for as long as possible, and this study is a first step toward reducing the suffering caused by debilitating diseases.”

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MEDTECH

Versantis to Present Positive Phase 1b Data at AASLD for VS-01 in Patients with Decompensated Cirrhosis

Versantis | November 15, 2021

Versantis, a clinical-stage biotechnology company developing novel therapies for orphan liver and pediatric diseases, announced that positive Phase 1b clinical data from a study of its lead investigational, orphan-designated product, VS-01, in 12 hospitalized patients with decompensated liver cirrhosis will be presented at the American Association for the Study of Liver Diseases (AASLD) annual meeting. The data show that VS-01 was safe and well tolerated in these patients, and demonstrated promising early indications of efficacy in this clinical study. VS-01 is a potentially lifesaving, multi-organ support therapy that aims to timely reverse Acute-on-Chronic Liver Failure (ACLF) by enhancing the clearance of ammonia and other toxins following paracentesis. The Abstract selected for oral presentation is as follows Oral Presentation Title: Safety and Preliminary Efficacy and Pharmacokinetics of Intraperitoneal VS-01 Infustions in Patients with Decompensated Liver Cirrhosis: A First-in-Human, Open-label, Phase 1b Clinical Trial Presenter: Dr. Frank Erhard Uschner, Section for Translational Hepatology, Department of Internal Medicine I, Goethe University Clinic Frankfurt, Session Date/Time: Monday, November 15, 2021, 12:30 p.m. EST The annual Liver Meeting, held by AASLD, brings together attendees from around the world to exchange the latest research, discuss new developments in treatments, and network with others in the field. The primary objective of this single-center first-in-human study was to evaluate the safety and tolerability of i.p.-administered VS-01 on top of standard of care in cirrhotic patients with ascites and mild hepatic encephalopathy following single and multiple intraperitoneal administrations. The secondary objectives were to gather preliminary PK, PD, and clinical efficacy data. In total, all 12 patients completed treatment in the Department of Internal Medicine I, Goethe University Frankfurt and were assigned to receive either a single dose of VS-01 (Part A; n=9) or four consecutive doses (Part B; n=3). Following the drainage of ascites in these patients, VS-01 was then infused into the peritoneal cavity via the existing paracentesis catheter and removed after a dwell time of 2-3 hours. No treatment-related serious adverse events were reported and no patient discontinued treatment due to an adverse event. VS-01 also demonstrated promising clinical efficacy results, including a high and dose-dependent ammonia clearance, promising improvement in hepatic encephalopathy based on psychometric tests, and increased peritoneal clearance of ACLF-related metabolites. “The data show that VS-01 is safe and well tolerated in cirrhotic patients with ascites and covert (mild) hepatic encephalopathy, so very promising. We were able to administer VS-01 using standard hospital equipment via the therapeutic paracentesis catheter, which we believe can easily be incorporated into standard of care for patients There are very few treatments available for these patients and VS-01 is complementary to those, so we are excited to continue advancing it’s development and hopefully generate the data supporting this ground-breaking clinical approach.” Prof. Dr. Trebicka, the principal investigator and head of the Section of Translational Hepatology in Goethe University Frankfurt “VS-01 represents a promising new therapeutic for the potential treatment of patients with ascites and acute complications of cirrhosis. The data from this early study were very promising and importantly show that VS-01 appears to be safe and well tolerated in these patients. The amomonia and ACLF metabolites clearance data is particularly encouraging,” added Vincent Forster, CEO and co-Founder of Versantis. “The successful completion of this study supports future development of VS-01. We are now preparing to initiate a multi-center Phase 2a study in patients with ACLF, a seriously underserved and under-resourced indication. Together with our pipeline of innovative products, we are committed to developing and commercializing new treatment options for patients suffering from acute liver diseases.” About Acute-on-Chronic Liver Failure (ACLF) ACLF is an underserved medical condition, which, despite best possible available care, is associated with high short-term mortality. It is characterized by an abrupt life-threatening worsening of a pre-existing chronic liver disease (e.g., cirrhosis) resulting in liver and extrahepatic organ failure rapidly progressing into coma and death. Every year at least 150’000 patients are hospitalized with ACLF in the US and EU. The incidence is growing due to a higher prevalence of diabetes, obesity, fatty liver diseases, and alcohol consumption. By timely reversing ACLF and the multi-organ complications arising from cirrhosis, VS-01 aims to improve outcomes in these patients and relieve the growing health and economic burden of this advanced liver disease. About Versantis Versantis is a clinical stage biotechnology company focused on addressing the growing, un-met medical need in liver diseases. With a pipeline of drug and diagnostic products covering chronic and acute indications, Versantis believes it can revolutionize current standard of care for patients suffering from acquired and genetic hepatic deficiencies. Versantis’ lead program, VS-01, is in clinical development as a first-line therapy for the timely reversal of acute-on-chronic liver failure (ACLF). It harnesses Versantis’ proprietary scavenging liposomes to extract toxins from the body and, if approved, will be the first drug to take advantage of the intraperitoneal route to potentially support the liver, kidneys and brain, the organs that most often fail in cirrhotic patients. VS-01 has received orphan drug designation (ODD) from the EMA and U.S. FDA, as well as a Rare Pediatric Diseases Designation from the U.S. FDA for Urea Cycle Disorders. Founded by scientists from ETH Zurich with entrepreneurial drive, Versantis has built a team and Board of seasoned industry executives with a proven ability to advance novel therapies from the idea stage into clinical development. The company is headquartered in Zurich, Switzerland.

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CELL AND GENE THERAPY

TaiGen Partners with GPCR to develop Burixafor & Taigexyn(R)

Burixafor | November 09, 2020

TaiGen Biotechnology Company, Limited ("TaiGen") announced today that they have signed an exclusive agreement with GPCR Therapeutics, Inc. ("GPCR"), a leading Korean biotechnology company, for the continued development of Burixafor worldwide and the commercialization of Taigexyn® (nemonoxacin) in South Korea. Burixafor is a highly potent CXCR4 inhibitor currently under clinical development. It can be used as a stem cell mobilizer for hematopoietic stem cell transplantation and a chemosensitizer in hematological and solid tumors. It can also be used for stem cell collection in healthy individuals for personalized regenerative medicine. Taigexyn® is a novel safe and effective antibiotic for the treatment of bacterial infections including those caused by drug-resistant bacteria. Under the terms of the agreement, GPCR Therapeutics will be wholly responsible for the development, registration, and commercialization of Taigexyn® in S. Korea and Burixafor worldwide. Apart from upfront fees, TaiGen will receive shares of GPCR Therapeutics as well as future milestone and royalty payments.

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RESEARCH

Kineta and Samsung Biologics Announce Anti-VISTA Antibody Immunotherapy Development and Manufacturing Agreement

Samsung Biologics | July 21, 2021

Kineta, Inc., a clinical-stage biotech firm developing novel immunotherapies in oncology, has signed a strategic partnership agreement with Samsung Biologics, one of the world's top contract development and manufacturing companies (CDMO). To support the IND filing for KVA12.1, Kineta's new anti-VISTA antibody under development to treat solid tumors, Samsung Biologics will offer end-through-end CDMO services ranging from cell line development to cell line development clinical drug substance and drug product manufacturing. VISTA, which is overexpressed on myeloid-derived suppressor cells (MDSC) and regulatory T cells, is a major driver of the immunosuppressive tumor microenvironment (TME) (Tregs). It is an important immune checkpoint in myeloid cells, and VISTA blockade can reprogram suppressive myeloid cells and restore anti-tumor immune function. Preclinical studies show that KVA12.1 has anti-tumor activity as a single agent and that targeting VISTA in combination with PD-1, PD-L1, or CTLA-4 can substantially enhance the anti-tumor efficacy of those checkpoint inhibitors. Thus, Kineta's KVA12.1 is intended to reprogram the TME in hard solid tumors. The manufacturing cell line will be created with assistance from Samsung Biologics' R&D Center in San Francisco. The clinical trial materials will be manufactured in Samsung Biologics' headquarters in Incheon, South Korea. Samsung Biologics provides one-stop CDMO research and development services from its headquarters in Incheon, South Korea, and its newly built R&D Center in San Francisco. Providing quality-driven development services at a faster pace, the company delivers cell line development to drug substance manufacturing in six months and drug product manufacturing in seven months, the industry's fastest pace. Samsung Biologics is currently building its fourth and largest facility in Incheon, Korea, which will have a biomanufacturing capacity of 620,000 liters upon completion in 2023. About Kineta, Inc. Kineta, Inc. is a clinical-stage biotechnology company dedicated to developing game-changing life science technologies that address unmet patient needs. We have used our immunology expertise to advance a focused pipeline of oncology investigational drugs. To develop our innovative products, we actively work with a diverse range of private, government, and industry partners. About Samsung Biologics Co., Ltd. Samsung Biologics is a fully integrated CDMO that provides state-of-the-art contract development, manufacturing, and laboratory testing. Samsung Biologics is an award-winning partner of choice, with proven regulatory approvals, the largest capacity, and the fastest throughput. As a result, it is uniquely able to support the development and manufacturing of biologics products at every stage of the process while meeting the evolving needs of biopharmaceutical companies worldwide.

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