GENE Stock Overview

ASHBURN, Va., Feb. 04, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, has launched the “NETHERTON NOW” campaign to shed light on the profound and poorly understood impacts of Netherton Syndrome, a devastating genetic disease that has been significantly misdiagnosed in the past. It is estimated that up to 20% of babies born with Netherton Syndrome do not survive, highlighting the urgent need for greater awareness and new treatment options.

Provides 23andMe+ Premium members with insight into their likelihood of developing osteoporosis based on thousands of genetic variants Provides 23andMe+ Premium members with insight into their likelihood of developing osteoporosis based on thousands of genetic variants

The Genetic Toxicology Testing market is expected to rise due to rising genetic disease incidence, advancements in genetic toxicology testing technologies, increased R&D activities, strategic collaborations, and a focus on personalized therapy. The Genetic Toxicology Testing market is expected to rise due to rising genetic disease incidence, advancements in genetic toxicology testing technologies, increased R&D activities, strategic collaborations, and a focus on personalized therapy.

More Than 40 Adult Sickle Cell Disease Patients Now Enrolled in BEACON Trial of BEAM-101; Beam Expects to Dose 30 Patients and Present Updated Data by Mid-2025

MIAMI, FL / ACCESSWIRE / January 7, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) ("Telomir"), an emerging leader in age-reversal science focused on treating the root causes of diseases rather than just their symptoms, announced today potentially groundbreaking findings from a preclinical progeria lifespan study conducted in collaboration with Nagi Bioscience SA. Utilizing C.

On Thursday, the FDA approved Ionis Pharmaceuticals, Inc.'s IONS Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (SHTG) that can lead to acute pancreatitis (AP).

Neurocrine Biosciences won Food and Drug Administration approval late Friday for the first new treatment for an adrenal disease in decades. The post Neurocrine Wins FDA Approval For Genetic Disorder Drug appeared first on Investor's Business Daily.

CALGARY, Alberta, Dec. 12, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce a presentation by Dr. Allen Davidoff at the Rare and Genetic Kidney Disease Summit, in Boston, Massachusetts at 10:30 am ET, Thursday December 12, 2024. The presentation entitled “Autosomal Dominant Polycystic Kidney Disease - Genetic and Environmental Factors → Evidence for Aberrant Purine Metabolism as a Second Hit Determining Disease Progression.”

Avidity expands  its leading RNA delivery technology into precision cardiology with two new wholly-owned development candidates targeting rare genetic cardiomyopathies: AOC 1086 to treat PLN Cardiomyopathy and AOC 1072 to treat PRKAG2 Syndrome AOC 1072 and AOC 1086 preclinical data demonstrated robust siRNA delivery to the heart and targeted knockdown with potent reduction of approximately 80% in cardiac PLN mRNA and PRKAG2 mRNA Avidity introduces next-generation technology innovations demonstrating improved siRNA delivery in skeletal muscle and increased durability in preclinical studies AOC 1072 preclinical data will be presented at American Heart Association (AHA) Scientific Sessions 2024 Volume 11 of virtual investor and analyst series today, Tuesday, Nov. 12 at 8:00 a.m. ET SAN DIEGO , Nov. 12, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced it has expanded beyond rare skeletal muscle disorders and is opening up a new therapeutic field, precision cardiology, to address the root cause of genetic diseases of the heart.