Filtered By:
Management: Food and Drug Administration (FDA)

This page shows you your search results in order of date. This is page number 2.

Order by Relevance | Date

Total 35 results found since Jan 2013.

Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing
Acc Chem Res. 2021 Oct 20. doi: 10.1021/acs.accounts.1c00500. Online ahead of print.ABSTRACTConspectusSince the U.S. Food and Drug Administration (FDA) granted emergency use authorization for two mRNA vaccines against SARS-CoV-2, mRNA-based technology has attracted broad attention from the scientific community to investors. When delivered intracellularly, mRNA has the ability to produce various therapeutic proteins, enabling the treatment of a variety of illnesses, including but not limited to infectious diseases, cancers, and genetic diseases. Accordingly, mRNA holds significant therapeutic potential and provides a promis...
Source: Cell Research - October 20, 2021 Category: Cytology Authors: Min Qiu Yamin Li Hanan Bloomer Qiaobing Xu Source Type: research

PCV24 A Threshold Analysis of the Cost-Effectiveness of Adjunctive Inclisiran Therapy for Ascvd Patients with LDL ≥70 MG/DL on Maximally Tolerated Statin Therapy
Inclisiran is a first-in-class, small-interfering ribonucleic acid (siRNA) that has been recently submitted to the Food and Drug Administration (FDA) for approval for the treatment of atherosclerotic cardiovascular disease (ASCVD) in patients with low-density lipoprotein (LDL) ≥70 mg/dL despite being on maximally tolerated statin therapy. We created a Markov model to estimate a range of value-based prices for inclisiran compared to currently available therapies: ezetimibe and the proprotein convertase subtilisin/kexin type 9 monoclonal antibodies (PCSK9s).
Source: Value in Health - June 1, 2021 Category: International Medicine & Public Health Authors: K. Cai, B. Devine Source Type: research

Novel treatment options for acute hepatic porphyrias
Purpose of review Acute hepatic porphyrias (AHP) are a group of rare diseases that are characterized by episodic acute neurovisceral pain episodes caused by abnormal accumulation of the neurotoxic porphyrin precursor delta-aminolevulinic acid (ALA). Patient with frequent recurrent acute attacks have been difficult to treat and these patients sometimes require liver transplantation. Recent developments in small interfering RNA (siRNA)-based therapy led to the development of an effective prophylactic treatment for patients with frequent recurrent attacks. This review will describe treatment options for AHP and highlight ...
Source: Current Opinion in Gastroenterology - April 9, 2021 Category: Gastroenterology Tags: LIVER: Edited by Don C. Rockey Source Type: research

Opinion: miRNAs - The new wave of molecular cancer therapeutics
Transl Oncol. 2021 Mar 12;14(6):101064. doi: 10.1016/j.tranon.2021.101064. Online ahead of print.ABSTRACTCutting-edge advances in nanomedicine and the recent approval of two siRNA-based therapeutics by the Food and Drug Administration (FDA) has rekindled the interest in RNA interference (RNAi) as vehicles for the development of novel cancer therapeutics. In this perspective, we will briefly discuss how miRNAs are becoming the next-generation RNAi therapeutic, the advances in delivery vehicles for in vivo miRNA delivery, and where miRNA technology stands in terms of clinical translation.PMID:33721829 | DOI:10.1016/j.tranon.2021.101064
Source: Translational Oncology - March 15, 2021 Category: Cancer & Oncology Authors: Hernando Lopez-Bertoni John Laterra Source Type: research

Givosiran to treat acute porphyria.
Authors: Honor A, Rudnick SR, Bonkovsky HL Abstract Porphyrias are a family of rare diseases chiefly due to inborn errors of heme biosynthesis. The porphyrias are generally characterized either by the main site of overproduction of heme precursors (hepatic or erythropoietic) or the main clinical manifestations (acute or cutaneous). The regulation of 5- (or δ)-aminolevulinic acid synthase 1 (ALAS1) plays a key role in the pathway of normal hepatic heme synthesis, providing insight into the pathophysiologic mechanisms and potential therapeutic targets for the treatment of the porphyrias. Givosiran (Givlaari; Alnylam...
Source: Drugs of Today - February 18, 2021 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Leading RNA Interference Therapeutics Part 2: Silencing Delta-Aminolevulinic Acid Synthase 1, with a Focus on Givosiran
AbstractIn November 2019 givosiran became the second small interfering RNA (siRNA)-based drug to receive US Food and Drug Administration (FDA) approval, it has been developed for the treatment of acute intermittent porphyria (AIP), a disorder characterized by life-threatening acute neurovisceral attacks. The  porphyrias are a group of disorders in which enzymatic deficiencies in heme production lead to toxic accumulation of delta-aminolevulinic acid (ALA) and porphobilinogen (PBG), which are involved in the neurovisceral attacks. Givosiran acts as a conventional siRNA to trigger RNA interference (RNAi) -mediated gene sile...
Source: Molecular Diagnosis and Therapy - December 1, 2019 Category: Molecular Biology Source Type: research

Small Interfering RNA Therapeutic Inclisiran: A New Approach to Targeting PCSK9
AbstractHypercholesterolemia is a leading cause of cardiovascular disease and mortality in men and women throughout the USA and abroad. The development of statins (HMG-CoA reductase inhibitors) to lower plasma atherogenic cholesterol levels and improve cardiovascular outcomes represents one of the greatest contributions to clinical science in the twentieth century, although residual risk remains even among statin-treated patients. Our understanding of lipid metabolism took a giant leap forward in 2003 with the discovery of proprotein convertase subtilsin/kexin type  9 (PCSK9), a low abundance circulating protein with an o...
Source: BioDrugs - November 27, 2019 Category: Drugs & Pharmacology Source Type: research

Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran
AbstractIn 2018, patisiran was the first-ever RNA interference (RNAi)-based drug approved by the US Food and Drug Administration. Now pharmacology textbooks may include a new drug class that  results in the effect first described by Fire and Mello 2 decades ago: post-transcriptional gene silencing by a small-interfering RNA (siRNA). Patients with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) present with mutations in the transthyretin (TTR) gene that lead to the formation of amyloid deposits in peripheral nerves and heart. The disease may also affect the eye and central nervous system. The formulatio...
Source: Molecular Diagnosis and Therapy - November 6, 2019 Category: Molecular Biology Source Type: research

Patisiran for the treatment of patients with familial amyloid polyneuropathy.
Authors: Rizk M, Tüzmen S Abstract Onpattro, also commonly known as patisiran, is a small interfering RNA (siRNA) molecule packaged within a lipid nanoparticle and is transported into the cell to target transthyretin gene (TTR) messenger mRNA (mRNA) by attaching to its complementary sequence. The target mRNA is degraded and both mutant and wild-type amyloid transthyretin (ATTR) protein production becomes suppressed. This drug was developed by Alnylam Pharmaceuticals to treat a rare disease called hereditary ATTR (hATTR) amyloidosis. This disease develops as a result of the deposition of toxic aggregates of misfold...
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Gene Therapy Leaves a Vicious Cycle
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - April 23, 2019 Category: Cancer & Oncology Source Type: research

FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
FDA approves new drug for treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR). This is the first FDA-approved treatment for this rare, debilitating and often fatal genetic disease and the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.
Source: Food and Drug Administration - August 10, 2018 Category: American Health Source Type: news

Cancers, Vol. 10, Pages 80: Aptamer Therapeutics in Cancer: Current and Future
mi Tanaka Aptamer-related technologies represent a revolutionary advancement in the capacity to rapidly develop new classes of targeting ligands. Structurally distinct RNA and DNA oligonucleotides, aptamers mimic small, protein-binding molecules and exhibit high binding affinity and selectivity. Although their molecular weight is relatively small—approximately one-tenth that of monoclonal antibodies—their complex tertiary folded structures create sufficient recognition surface area for tight interaction with target molecules. Additionally, unlike antibodies, aptamers can be readily chemically synthesized and modifi...
Source: Cancers - March 19, 2018 Category: Cancer & Oncology Authors: Yoshihiro Morita Macall Leslie Hiroyasu Kameyama David Volk Takemi Tanaka Tags: Review Source Type: research

Identification of KX2-391 as an inhibitor of HBV transcription by a recombinant HBV-based screening assay
This study used recombinant HBV encoding NanoLuc to screen anti-HBV compounds from 1827 US Food and Drug Administration approved compounds and identified several compounds that suppressed HBV infection. Among them, KX2-391, a non-ATP-competitive inhibitor of SRC kinase and tubulin polymerization, was identified as a lead candidate for an anti-HBV drug. Treatment of sodium taurocholate cotransporting polypeptide (NTCP) transduced-HepG2 (HepG2-NTCP) or primary human hepatocytes with KX2-391 suppressed HBV replication in a dose-dependent manner. The anti-HBV activity of KX2-391 appeared not to depend on SRC kinase activity be...
Source: Antiviral Therapy - June 16, 2017 Category: Virology Source Type: research

Identification of KX2-391 as an inhibitor of HBV transcription by a recombinant HBV-based screening assay.
This study used recombinant HBV encoding NanoLuc to screen anti-HBV compounds from 1827 US Food and Drug Administration approved compounds and identified several compounds that suppressed HBV infection. Among them, KX2-391, a non-ATP-competitive inhibitor of SRC kinase and tubulin polymerization, was identified as a lead candidate for an anti-HBV drug. Treatment of sodium taurocholate cotransporting polypeptide (NTCP) transduced-HepG2 (HepG2-NTCP) or primary human hepatocytes with KX2-391 suppressed HBV replication in a dose-dependent manner. The anti-HBV activity of KX2-391 appeared not to depend on SRC kinase activity be...
Source: Antiviral Research - June 14, 2017 Category: Virology Authors: Harada K, Nishitsuji H, Ujino S, Shimotohno K Tags: Antiviral Res Source Type: research