11 Best Gene Therapy Stocks to Buy According to Analysts
Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.
SA Asks: Which small/mid-cap cardiology stocks are undervalued?
Cardiology drug and device makers will be back in the spotlight this weekend as the annual scientific meeting of the American ...
BioSpace15d
Omeros Corporation Provides Update of Ongoing Zaltenibart Phase 3 PNH Clinical Trial Program
SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today reported that clinical trial site activation for enrollment is underway for the company’s Phase 3 program evaluating zaltenibart in ...
Nasdaq17d
Omeros Advances Zaltenibart Phase 3 Trials For PNH, Data Readout Expected In Late 2026
(RTTNews) - Omeros Corp. (OMER), Friday announced that it has initiated clinical trial site activation for its Phase 3 program evaluating zaltenibart or OMS906 in paroxysmal nocturnal ...
Benzinga.com18d
FDA Approves Expanded Use Of Novartis Rare Disease Drug As Only Treatment For Rare Type Of Kidney Disease
Also Read: Novartis New Data From Single-Dose Gene Therapy Shows Meaningful ... approval in December 2023 for adults with paroxysmal nocturnal hemoglobinuria (PNH). In the fourth quarter of ...
Yahoo Finance18d
FDA Approves Expanded Use Of Novartis Rare Disease Drug As Only Treatment For Rare Type Of Kidney Disease
Also Read: Novartis New Data From Single-Dose Gene Therapy Shows Meaningful Efficacy ... Fabhalta received its first FDA approval in December 2023 for adults with paroxysmal nocturnal hemoglobinuria ...
Yale Environment 36019d
New CRISPR tool enables more seamless gene editing — and improved disease modeling
Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...
NME19d
Gene on reuniting for first live show in over 20 years: “We don’t want any half measures – it needs to be spectacular”
The original Gene line-up have reunited and announced their first live show in over 20 years. Read NME‘s interview with frontman Martin Rossiter below, where he discusses what to expect from the ...
BioSpace20d
Sarepta’s Elevidys Death, FDA’s Vaccine Move, Weldon Whiplash, PDUFAs in Cardio
A patient with Duchenne muscular dystrophy taking Sarepta’s gene therapy Elevidys has died of acute liver ... And second, Milestone Pharmaceutical has a PDUFA coming up for etripamil in paroxysmal ...
rarediseaseadvisor20d
Several Improvements in Gene Therapy Are in the Pipeline for Patients With DMD
“While promising, unfortunately the level of dystrophin expression is good but not optimal,” Nicholas Wein, PhD, from Nationwide Children's Hospital, Ohio, and Ohio State University said of currently ...
Reuters21d
Sarepta says teen died after its gene therapy treatment
Patient was a 16-year-old teen, who underwent treatment in Dec First death reported after Elevidys treatment Liver injury a known risk with gene therapies March 18 (Reuters) - Sarepta Therapeutics ...
Nature22d
Errors in the Huntington’s disease gene accumulate slowly and then all at once
Inherited repeat mutations in the HTT gene expand in neurons over decades before crossing a toxic threshold, revealing a long time window for therapy. CAG repeats in HTT are unstable, meaning that ...