Remove Drugs Remove FDA Approval Remove Genetic Disease Remove RNA
article thumbnail

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs).

RNA 245
article thumbnail

The evolution of the RNA therapeutics landscape 

Drug Discovery World

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

RNA 59
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

FDA Action Alert: Y-mAbs, Vanda, Alnylam and BioCryst

The Pharma Data

Food and Drug Administration (FDA). The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. The FDA approved the drug on November 24. It is an RNA interference (RNAi) therapeutic. grandbrothers/Shutterstock. Here’s a look.

article thumbnail

Biopharma Money on the Move: December 2 – 8

The Pharma Data

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Remix Therapeutics .

RNA 52
article thumbnail

Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

article thumbnail

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

article thumbnail

Beyond the pandemic: the potential of mRNA technology

pharmaphorum

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. Pfizer already has a head start on competitors in the field, having achieved the first US FDA approval for an mRNA vaccine, alongside BioNTech.

RNA 98