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

Oct. 01, 2025 -- RetinalGenix Technologies Inc. OTCQB:RTGN (“RetinalGenix” or the “Company”), a pioneering developmental-stage company focused on ophthalmic screening, monitoring, pharmacogenetic mapping, and repurposed drug development for early detection and treatment of eye and systemic diseases, and RGEN Inc., a leader in genetic research and innovation (“RGEN”), today announced a strategic partnership to deliver game-changing advancements in the early detection and risk prediction of Advan

Oct. 01, 2025 -- Aerska, a biotechnology company aiming to redefine the treatment of neurological diseases, today announced it has raised $21 million in seed financing, co-led by Age1, Backed VC and Speedinvest, with participation from Blueyard, Lingotto (Exor), Norrsken VC, Kerna, PsyMed, Saras, and Ada Ventures. The company is developing systemically administered RNA interference (RNAi) medicines designed to silence genes that drive brain diseases. Delivering genetic medicines to the brain

Dotinurad is a next-generation URAT1 inhibitor in two Phase 3 clinical trials with potential for best-in-class safety and efficacy Urica sold dotinurad to Crystalys Therapeutics in 2024 in exchange for equity and a 3% royalty on future net sales of dotinurad Oct. 01, 2025 -- Urica Therapeutics, Inc. (“Urica” or the “Company”), a Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) subsidiary, today announced that Crystalys Therapeutics, Inc. (“Crystalys”), in which Urica maintains an equity pos

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 -- Fifty 1 Labs Inc. (OTC: FITY), a leader in AI-powered healthcare and biotechnology solutions, is excited to announce the signing of a Letter of Intent (LOI) to acquire BioSpark AI Technologies Inc., a Vancouver-based innovator specializing in patented artificial intelligence systems for clinical data extraction, structuring, and real-time analysis. This strategic move builds on a successful history of collaboration between the two companies, reinforcing Fifty 1 Labs' commitme