AAVB-039 addresses the root cause of the disease, benefitting patients with any ABCA4 mutation AAVB-039 is currently being assessed in the CELESTE interventional clinical trial and we continue recruiting in our STELLA observational study Oct. 02, 2025 --- AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted AAVB-039, the company’s investigation

ANAVEX3-71-002 trial achieved its primary endpoint demonstrating safety and tolerability in both male and female adults Oral ANAVEX®3-71 tablet exhibits superior pharmacokinetics compared to the current immediate-release oral capsule, enabling once-daily dosing NEW YORK, Oct. 02, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkins

CRYPTICS study met the primary efficacy endpoint measure, 24-hour chest tube drainage (Difference in LS Means was 142.0 mL; 95.576% confidence interval of volume difference lower than noninferiority margin) Sensitivity analyses of the primary endpoint were consistent and supportive Comprehensive review of the study efficacy data based on clinically relevant secondary endpoints further supports noninferiority of CLPH-511 to room temperature platelets Adverse events were well balanced between the

Based on previously reported positive Phase 1 results, Kynexis is advancing KYN-5356, a first-in-class KAT-II inhibitor, into a Phase 2 clinical trial in patients with cognitive impairment associated with schizophrenia Phase 2 trial seeks to establish proof-of-concept of KYN-5356; topline results are expected by Q4 2026 Sept. 30, 2025 -- Kynexis, a clinical-stage biotechnology company focused on precision therapeutics for brain diseases, today announced that the first patient has been dosed in

Oct. 01, 2025 -- COUR Pharma, a clinical-stage biotechnology company developing first-in-class, antigen-specific immune tolerance therapies for autoimmune diseases, announced today the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CNP-106, an investigational therapy for the treatment of generalized myasthenia gravis (gMG). CNP-106 is a biodegradable nanoparticle that encapsulates seven acetylcholine receptor (AChR) antigens. It is being evaluated for its p

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased brain myelin volume, resulting in promising functional improvements in children with Canavan Disease Oct. 01, 2025 -- Myrtelle Inc. (“Myrtelle” or the “Company), a pioneering clinical-stage gene therapy company developing transformative treatments for neurodegenerative diseases, today announced the publication of interim results from its Phase 1/2 clinica

Sept. 30, 2025 -- Umoja Biopharma, the clinical-stage leader of in vivo cell therapies that aim to realize the full reach and promise of CAR T cells, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to UB-VV111 for the treatment of relapsed/refractory Large B-cell Lymphoma following two or more lines of prior therapy and relapsed/refractory Chronic Lymphocytic Leukemia following two or more lines of prior therapy. UB-VV111 is an investigation

Sept. 30, 2025 -- PolyPid Ltd. (Nasdaq: PYPD) ("PolyPid" or the "Company"), a late-stage biopharma company aiming to improve surgical outcomes, today announced that Dr. Shmuel Sharoni will present the topline results from the Company's successful Phase 3 SHIELD II trial of D-PLEX₁₀₀ at the upcoming 2025 American College of Surgeons (ACS) Clinical Congress in Chicago, IL. Dr. Shmuel Sharoni will deliver a presentation titled "Efficacy Of A Novel Local Prolonged-release Incisional Doxycycline On

Publication title: “Oral Blarcamesine Phase IIb/III Trial Confirms Identified Precision Medicine Patient Population – Significant Broad Clinical and Quality of Life Improvements for Early Alzheimer’s Disease Patients” Significant improvement in self-assessed Quality of Life (QoL-AD) scores indicating a reversal of negative trajectory for Alzheimer's disease clinical trial participants was observed Using a novel Precision Medicine approach, up to ~70% of Alzheimer's disease participants benefited

Sept. 29, 2025 -- INmune Bio, Inc. (NASDAQ: INMB), a clinical-stage inflammation and immunology company, today announces the submission of a manuscript detailing the results of its Phase 2 MINDFuL trial. The manuscript, titled, “XPro1595, a Selective Soluble TNF Neutralizer, in Early Alzheimer’s Disease with Inflammation (ADi): Results from the Phase 2 MINDFuL Trial,” has been submitted for peer review to npj Dementia, a Nature Portfolio journal. The detailed manuscript provides a comprehens