CRISPR

Rice University researchers in Houston used the foundation of CRISPR/Cas9 technology to develop “drive-and-process” arrays, or DAP for short. The name may be simple, but the technology is complex and could be as groundbreaking as its predecessor. DAP is a mechanism that enables the editing of multiple DNA sites – rather than one at a time – to comprehensively reverse polygenic diseases resulting from multiple genetic predispositions.

Sander Flaum

Looking past COVID, the medical world is in turmoil. It’s hard enough for us in manufacturing and marketing to keep up with innovative technologies. Patients and caregivers have it especially tough since they are on the brunt end of disruptive innovations. Every day, patients face a ceaseless onslaught of sort of truths, half-truths, and flat-out lies about their healthcare, and HCPs no sooner graduate from med school than they discover much of their training is obsolete. Flaum Navigators Principal Sander A. Flaum explores how we can engage patients and physicians in the “Next Big Thing.”

As Novartis explores the possible sell-off of its Sandoz business, the company is undergoing a significant transformation that it says will save about $1 billion in operations costs by 2024.

The U.S. Food and Drug Administration approved ViiV Healthcare’s Cabenuva (cabotegravir, rilpivirine) for the treatment of HIV-1 in virologically suppressed adolescents (HIV-1 RNA less than 50 copies per milliliter [c/mL]) who are 12 years of age or older and weigh at least 35kg on a stable antiretroviral regimen, with no history of treatment failure, and with no known or suspected resistance to either cabotegravir or rilpivirine.

Lilly is investing $700 million to build a state-of-the-art facility in the Boston Seaport, as part of plans to advance the company’s RNA-based research and development activities. 

RNA-focused Remix Therapeutics agreed on a strategic small-molecule discovery and development collaboration with Janssen Pharmaceutica N.V., a division of Johnson & Johnson.

Aside from vaccines, the advent of several effective COVID-19 antiviral therapies, including Pfizer’s Paxlovid, provides hope for ending the pandemic. Details about the U.S. government’s contract with Pfizer for 10 million doses of Paxlovid are emerging, with some unusual elements.

Temps are falling and so is investor cash as BioSpace reviewed which biotech companies are scooping up the dollars.

Led by new CEO Dr. Yvonne Greenstreet, Alnylam Pharmaceuticals has a stated ambition to transition the RNA interference therapeutics trailblazer to a top 5 biotech – measured by market capitalization – within the next five years.

Belgium’s UCB is acquiring California-based Zogenix, a company focused on rare diseases, in a deal valued up to €1.7 billion ($1.9 billion).