On Monday, Beam Therapeutics Inc. BEAM announced initial safety and efficacy data from its Phase 1/2 trial of BEAM-302, establishing clinical proof-of-concept as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing.
Rhythm Pharmaceuticals' 01/2025 presentation and Q4 2024 preliminary results press release highlight growing financial performance and promising pipeline developments. Investment thesis focuses on Rhythm's robust clinical progress and strategic positioning in the rare genetic obesity market. Rating justification is based on solid revenues, innovative product pipeline, and potential market expansion.
As presented at ARO, 10 of 11 children with at least one post-treatment assessment showed notable improvements in hearing Speech and development progress followed dramatic improvements in hearing in first child treated in the trial TARRYTOWN, N.Y., Feb. 24, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial in 12 children who have profound genetic hearing loss due to variants of the otoferlin (OTOF) gene.
New Patent covers claims which focus on method for identifying genetic markers in patients with alcohol or opioid related disorders
Real-Time PCR* Solution Combines an Assay and a Web Application for Rapid Strain Characterization to Quickly Identify Root Cause Contamination Aiding in More Rapid Decision Making to Minimize Future Recurrence MARCY-L'ÉTOILE, France , Feb. 13, 2025 /PRNewswire/ -- bioMérieux, a world leader in the field of in vitro diagnostics, today announces the launch of GENE-UP® TYPER, a real-time, cutting-edge PCR solution for rapid pathogen detection root cause analysis in the food industry. Each year, an estimated 600 million people fall ill after consuming contaminated food, according to the World Health Organization (WHO)1.
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.