March 20 (Reuters) - Staff reviewers of U.S. drug regulator on Monday did not raise any new safety concerns about Biogen Inc's BIIB.O experimental drug for treating a rare type of amyotrophic lateral sclerosis, also known as Lou Gehrig's disease.
Aurisco, a pharmaceutical company and contract development and manufacturing organization (CDMO) in China, is cooperating with global life sciences company Cytiva, to build its first Oligo FlexFactory platform for commercial production.
EUROAPI to Reach 500 kg Peptide and Oligonucleotide Capacity Per Year in Frankfurt by 2025
A phase-III clinical trial of the investigational drug Tofersen in patients with MND caused by the faulty SOD1 gene, has shown it can slow and reduce progression of the disease, according to a statement from the University of Sheffield.
HANOVER, Germany--(BUSINESS WIRE)--Cardior Pharmaceuticals, a clinical-stage biotech company developing non-coding RNA (ncRNA)-based therapeutics for patients with cardiac diseases, today announced the CE marking of its PCR kit which measures circulating microRNA-132 (miR-132) levels in the blood of patients receiving the company’s lead candidate CDR132L, an oligonucleotide-based miR-132 inhibitor. The company is currently conducting its Phase 2 clinical study, HF-REVERT (NCT05350969), to evaluate the safety and efficacy of CDR132L in patients with heart failure and reduced left ventricular ejection fraction (LVEF) after a myocardial infarction. By using the CardiorHealth miR-132 PCR kit, Cardior ensures consistent measurement of miR-132 levels following CDR132L treatment across all clinical trial centers as an indicator of the target engagement and the compound’s mechanism of action.
KriSan Biotech Launched its GMP Oligonucleotides
Oligonucleotides are very promising and versatile therapeutics for rare and genetic diseases. They act on the RNA level through different molecular pathways that we already have described in a precedent article. Because of their mechanism of action, this class of therapeutics has huge possibilities to treat diseases that were incurable before. The use of oligonucleotide therapeutics is well tolerated by the body, selective to their target, and with reduced secondary effects. Despites their huge potential and attractiveness as a treatment, there is a major drawback when dealing with oligonucleotides as drugs: their poor bioavailability and cellular uptake. These aspects are the main limitations for their application as therapeutics. To address these drawbacks, it seems of utmost importance to develop delivery systems that enable oligonucleotides to reach their targets. In a previous article on oligonucleotide conjugates, we have described the use of the trimeric GalNAc (N-acetylgalactosamine) molecule as a carrier of oligonucleotides to specific cells, the hepatocytes. Here, we discuss a delivery system that have been in vogue since almost 40 years, the cell-penetrating peptides (CPP). The first use of CPP, a poly L-lysine, has been described for the delivery of an anticancer drug in vitro and in vivo of mouse model1. It has opened the way for generation of CPP-drug conjugates and has potential as well for oligonucleotides2.
Senior officials from the European Commission (EC - DG SANTE), EMA and the United States Food and Drug Administration (FDA) held their 2020 bilateral regulatory dialogue meeting on 18 and 19 June. During this virtual two-day meeting, the authorities reviewed their ongoing joint initiatives, discussed strategic priorities for the coming years and identified areas where their already close collaboration can be further strengthened.
Thomas Meier, CEO Bachem, im Interview
sk anyone to describe Stanley Crooke, CEO of Ionis Pharmaceuticals, and a single word comes up again and again: intense. Friends and adversaries alike say he operates at an intellectual pace, as both a scientist and a leader, that makes him something of a force of nature. Keep up, or be swept aside.