Category: Diseases!
- Opus reports FDA approval of phentolamine eye drops for presbyopia ()
Published October 16, 2026. Opus Genetics announced on October 16 that the U.S. FDA approved phentolamine ophthalmic solution 0.75% for adults with presbyopia, the age-related difficulty focusing on nearby objects. The decision expands the indications for RYZUMVI, previously approved for reversing drug-induced pupil dilation. The company says the approval was supported by the Phase 3 […]
- Opus reports early vision improvements in BEST1 and LCA5 gene therapy studies ()
Published October 16, 2026. Opus Genetics reported early clinical findings from two investigational retinal gene therapies on October 15 at Retina in Rio. The programs target inherited vision disorders associated with BEST1 and LCA5 mutations. For OPGx-BEST1, the company reported improved retinal sensitivity in three of four evaluable participants in the initial low-dose BIRD-1 cohort. […]
- Updated CAR T-cell consensus emphasizes trials and long-term monitoring in autoimmune disease ()
Published October 16, 2026. An October 15 announcement from the International Society for Cell & Gene Therapy highlights updated international guidance on CAR T-cell therapy for severe autoimmune diseases that resist standard treatment. EBMT described the recommendations on October 6; they appear in The Lancet Rheumatology. The consensus, developed by 31 experts with EBMT, ISCT […]
- EB103: first repeat-dose patient reported in STARLIGHT-1 lymphoma trial ()
Published October 16, 2026. Estrella Immunopharma reported on October 15 that the first patient to receive two EB103 infusions in the STARLIGHT-1 expansion phase had no treatment-related serious adverse events reported during the first month. Each infusion delivered 5 million T cells per kilogram, with three weeks between doses. EB103 is an investigational ARTEMIS T-cell […]
- SER-428 liquid microbiome therapy shows preclinical pathogen reductions ()
Published October 16, 2026. Seres Therapeutics announced on October 15 that its experimental microbiome product SER-428 reduced intestinal pathogens in preclinical models. The company plans to present the findings at IDWeek in Washington, D.C., on October 22. SER-428 is a liquid preparation of live bacteria designed for patients unable to swallow capsules, including people receiving […]
- Estuary Launches with $103 Million for Kidney-Targeted Gene Therapies ()
Published October 16, 2026. Estuary Biotherapeutics launched today with US$103 million in committed financing to develop gene therapies for kidney disease. The total combines a US$93 million Series A led by RA Capital Management with US$10 million in earlier seed financing. Its first programs target autosomal dominant polycystic kidney disease (ADPKD). EST-201 is designed to […]
- Stroke Alters Astrocyte Signaling and Accelerates Glioma Growth in Experimental Models ()
Published October 16, 2026. An October 15 research update from Baylor College of Medicine highlights how stroke can reshape the environment around gliomas in experimental models. The underlying study appeared in Nature Cancer on September 25, 2026. Researchers found that stroke increased tumor infiltration into injured brain regions and shortened survival in the models studied. […]
- Vanda pauses VQW-765 after phase 3 social anxiety trial misses primary endpoint ()
Published October 15, 2026. Vanda Pharmaceuticals announced on October 14 that its phase 3 trial of VQW-765 for on-demand treatment of acute anxiety in adults with social anxiety disorder missed its primary endpoint. The company has put the program on hold while it analyzes the full dataset. The randomized, double-blind trial enrolled 546 adults. Participants […]
- Senescent-like B cells linked to progression toward rheumatoid arthritis ()
Published October 15, 2026. A study published in Nature Aging on October 14 identifies an immune-cell population associated with progression toward rheumatoid arthritis, offering a potential direction for prevention research. Researchers found that activated naive B cells carrying CXCR5 and CD69 markers expanded before clinical disease appeared in people positive for anti-citrullinated protein antibodies who […]
- ENTR-601-45: early Duchenne trial reports a small dystrophin increase without observed functional benefit ()
Published October 15, 2026. Entrada Therapeutics reported initial results on October 14 from ELEVATE-45-201, a phase 1/2 trial of ENTR-601-45 for Duchenne muscular dystrophy amenable to exon 45 skipping. This experimental oligonucleotide aims to enable production of a shortened dystrophin protein. The first cohort included eight participants randomized to treatment or placebo. After three doses, […]