Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

Regulus Therapeutics Presents New miRNA Data

By Drug Discovery Trends Editor | April 29, 2011

Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs, announced the presentation of new preclinical data from its cardiovascular and metabolic disease program, performed in collaboration with scientists at New York University (NYU) and Wake Forest University. The data were presented at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2011 Scientific Sessions held April 28-30, 2011 in Chicago, Ill. In an oral presentation titled “microRNA Regulation of Cholesterol Homeostasis,” proprietary chemically modified therapeutic anti-miRs targeting microRNA-33a (miR-33a) and microRNA-33b (miR-33b) were shown to increase levels of high density lipoprotein cholesterol (HDL-C), or ‘good’ cholesterol, and reduce levels of blood triglycerides in non-human primates.

“This important study is the first-ever demonstration of therapeutic benefits resulting from the modulation of a microRNA and its downstream pathways in non-human primates. Equally important, the data support the development of anti-miR-33 as a potential new therapeutic for dyslipidemia, atherosclerosis, and other related metabolic diseases by showing significant increases in levels of HDL-C and lowering of triglycerides,” said Hubert Chen, M.D., vice president of translational medicine of Regulus. “Regulus is advancing several microRNA therapeutic programs to the clinic, including anti-miR-33.”

The new in vivo research was performed using an experimental design of feeding non-human primates a high carbohydrate, moderate cholesterol diet. anti-miR-33 was administered subcutaneously at a dose of 5 mg/kg twice weekly for the first two weeks and then once weekly for the remainder of the 12 week study. Plasma lipids were analyzed weekly, and non-human primates treated with anti-miR-33 compared to mismatch control showed a 50% increase in HDL-C and a 50% decrease in blood triglycerides. Further, anti-miR-33 treatment increased the expression of miR-33a and miR-33b target genes involved in clearance of excess cholesterol from cells to the liver for excretion to the bile and feces, known as reverse cholesterol transport, and also genes involved in fatty acid oxidation that could be responsible for the observed triglyceride lowering.

Kathryn Moore, Ph.D., associate professor in the Department of Medicine at NYU Langone Medical Center and presenting author said, “Much progress has been made in understanding the molecular mechanisms that regulate cholesterol and fatty acid metabolism and the role of microRNAs in this complex genetic network. Our studies with Regulus show that miR-33 specifically targets genes involved in HDL-C biogenesis, cholesterol efflux and fatty acid oxidation, and its antagonism results in direct upregulation of these pathways with clear potential for therapeutic benefit. Modulating dysregulated microRNAs with therapeutic anti-miRs holds tremendous promise as a new innovative class of medicines.”

“Coronary artery disease remains a major killer in the developed world pointing to the need for novel therapeutic approaches,” said Ryan Temel, Ph.D., assistant professor at Wake Forest School of Medicine. “This study in non-human primates demonstrates that anti-miR-33 might be a promising clinical approach in the treatment of cardiovascular disease.”

Regulus is developing microRNA therapeutics targeting both miR-33a and miR-33b. Regulus controls fundamental patent rights related to miR-33, including the miR-33 sequence and complementary sequences covered in the Tuschl III patent series. Additional Regulus patent rights include compositions of matter for various chemically modified anti-miR-33 compounds and methods of use for the treatment of metabolic diseases with anti-miR-33. 

Date: April 28, 2011
Source: Regulus Therapeutics Inc. 


Filed Under: Drug Discovery

 

Related Articles Read More >

Takeda Pharmaceutical in the Drug Discovery & Development Pharma 50
Takeda’s Takhzyro fares well in pediatric hereditary angioedema study
Novartis in the Pharma 50
Novartis to cut up to 8,000 positions
Gilead Sciences in the Drug Discovery & Development Pharma 50
Gilead resubmits application to FDA for twice-yearly HIV drug lenacapavir
George Floyd mural
How the pandemic and George Floyd made clinical trial diversity a priority

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50