ProQR announces rare pediatric disease designation, phase 1/2 results for sepofarsen
Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics. Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene. With the designation, the drug will receive priority review by the FDA, and it could qualify ProQR for a voucher redeemable for priority FDA review of a subsequent marketing application for a different product, the release said.
Four new drugs may help fight the growing threat of fatal fungi
At a medical mycology meeting here earlier this month, scientists presented promising phase II clinical data for rezafungin, a new member of an existing class called the echinocandins developed by Cidara Therapeutics in San Diego.
Biotech Says It Hurdled A Genetic Barrier, But Shares Gave Up A Breakout
Arrowhead Pharmaceuticals stock flirted with a breakout Friday after the biotech company said it’s experimenting with drugs that will “silence” multiple troublesome genes simultaneously. Previously, that was impossible, Chief Executive Christopher Anzalone said late Thursday on CNBC’s Mad Money. Arrowhead Pharmaceuticals (ARWR) is among a slew of biotech companies using technology called RNA interference to silence genes responsible for creating problematic proteins.
Reata CEO on positive trial results for drug to treat Friedreich’s ataxia
Warren Huff, Reata Pharma CEO, joins CNBC’s “Power Lunch” team to discuss his company’s positive clinical trial results for a drug to treat the neuromuscular disease Friedreich’s ataxia.
Lyme Disease Vaccine
If the fear of ticks prevents you from enjoying the great outdoors, there might be some good news on the horizon. Thomas Lingelbach with Valneva has been working on a new vaccine designed to ward off Lyme disease.
Eye Drops To Reverse Vision Loss One Step Closer To Market
Eye drops that can reverse poor vision? It may sound like science fiction, but one Israeli company is aiming to bring this product to market. The drops passed their Phase 2b clinical trial earlier this month, meaning they’ve proven to improve farsightedness, the inability to see or read nearby objects, and are highly tolerable. Although results have yet to be released, Israel-based Orasis Pharmaceuticals, revealed details last week about the latest results of the Phase 2b study for their CSF-1 eye drops.
Arrowhead CEO breaks down drug partnerships with Johnson & Johnson and Amgen
Jim Cramer chats with Arrowhead Pharmaceuticals CEO Christopher Anzalone about projects the company is working on to silence genes that cause rare genetic disorders.
Pharma’s roller-coaster relationship with RNA therapies
Fortunes can shift precipitously in the drug-discovery world. At the start of the twenty-first century, all eyes were focused on a powerful gene-silencing technology called RNA interference (RNAi), and many companies saw the almost limitless potential of harnessing the tool to manipulate genes implicated in diseases.
