Clinical Trial

CRISPR Therapeutics Presents New Preclinical Data for CTX460™ Demonstrating In Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™ Editing Platform

-Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile- -The AATD...

AskBio Presents Interim Safety Results from Phase 1/Phase 2 LION-CS101 Clinical Trial of AB-1003 in Participants with Limb-Girdle Muscular Dystrophy 2I/R9

Research Triangle Park, N.C., Oct. 10, 2025 (GLOBE NEWSWIRE) -- Data from first cohort of five participants suggest an acceptable...

Anavex Life Sciences Announces Presentation at the 35th Alzheimer Europe Conference ‘Connecting Science and Communities: The Future of Dementia Care’

Presentation title: “Advancing Alzheimer’s Disease Care: Convenience for Both Patients and Families with Oral Blarcamesine” NEW YORK, Oct. 10, 2025...

Biogen and Stoke Therapeutics Present New Data at the 54th Child Neurology Society (CNS) Annual Meeting that Support the Potential of Zorevunersen as a Disease-Modifying Medicine for Dravet Syndrome

Cabaletta Bio Presents First Rese-cel Data with No Preconditioning Demonstrating Biologic Activity and Early Clinical Responses at the 2025 ESGCT Annual Congress

– Complete B cell depletion, rapid reduction in autoantibodies and near-complete resolution of clinical symptoms in two of three refractory...

Alkeus Pharmaceuticals Announces Presentation of Gildeuretinol Data at the American Academy of Ophthalmology (AAO) 2025 Annual Meeting, October 18-21 in Orlando

Results from the study of oral gildeuretinol in moderate Stargardt disease to be presented as late-breaker during Retina Subspecialty DayCAMBRIDGE,...

Alamar Biosciences Launches NULISAqpcr™ BD-pTau217 Assay: A Breakthrough in Non-Invasive, Brain-Specific Biomarker Detection for Alzheimer’s Disease Research

New Blood-Based Assay Delivers Unparalleled Sensitivity and Specificity to enable the next generation of assay development.  FREMONT, Calif., Oct. 9,...

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