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Opus Genetics Aligns with FDA on Phase 3 Trial Design for OPGx-LCA5 in Inherited Retinal Disease
July 6, 2026 — Opus Genetics announced that it has reached alignment with the U.S. Food and Drug Administration on the design of its registrational Phase 3 clinical trial evaluating OPGx-LCA5 for LCA5-associated inherited retinal disease, an ultra-rare, early-onset inherited retinal dystrophy that can cause severe childhood blindness. The alignment followed
FDA Clears IND for AAV9/SUMF1 Gene Therapy in Multiple Sulfatase Deficiency
July 6, 2026 — The U.S. Food and Drug Administration has cleared an Investigational New Drug application for an investigational AAV9/SUMF1 gene therapy for multiple sulfatase deficiency, or MSD, enabling a planned first-in-human clinical study in children with the ultra-rare lysosomal storage disorder. The IND is sponsored by the National Center for Advancin
MeiraGTx Secures Investment to Support Late-Stage AAV Gene Therapy Programs
July 7, 2026 — MeiraGTx announced that it has entered into an agreement with Oberland Capital Management for an investment of up to $400 million, including up to $375 million in non-dilutive royalty funding and up to $25 million in equity investment. The financing is designed to support MeiraGTx as it advances multiple late-stage genetic […]
FDA Clears IND for CoRegen’s CRISPR-Engineered Treg Cell Therapy in Advanced Solid Tumors
July 07, 2026 — The U.S. Food and Drug Administration has cleared CoRegen’s Investigational New Drug application for CRG-150, allowing the company to begin a first-in-human Phase 1/2a clinical trial of its autologous CRISPR-engineered regulatory T cell therapy in patients with advanced solid tumors. The study will evaluate safety and preliminary efficacy in
Epicrispr Completes Dose Escalation in First-in-Human AAV Gene Therapy Trial for FSHD
July 7, 2026 — Epicrispr Biotechnologies announced that it has completed enrollment and dosing in the dose-escalation portion of its first-in-human clinical trial evaluating EPI-321 for facioscapulohumeral muscular dystrophy, or FSHD. All 12 patients have been enrolled and dosed across two dose cohorts in the ongoing open-label study. The trial is evaluating
Engineered AAV5 Vectors Use Glymphatic Delivery to Target Human Glial Cells Across the Brain
July 08, 2026 — A new study published in Nature Biotechnology describes a gene therapy strategy that combines engineered AAV5 vectors with delivery through the brain’s glymphatic transport system to distribute therapeutic genes broadly across the brain while preferentially targeting human glial cells. The approach addresses two major barriers in neurological
Genespire Reports Preclinical Data Supporting Immune-Shielded Lentiviral Gene Therapy for MMA
July 08, 2026 — Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy, announced the publication of preclinical data supporting its liver-directed immune-shielded lentiviral vector gene therapy approach for methylmalonic acidemia, or MMA. The findings, published in the Journal of Hepatology, showed that a single
Allotera Therapeutics Raises Additional $35M to Advance Off-the-Shelf CAR-T Therapy for T-Cell Cancers
July 08, 2026 — Allotera Therapeutics, formerly Wugen, announced the closing of a $35 million financing round comprising equity and venture debt, bringing the company’s total Series C financing to $150 million. The company also introduced its new name, Allotera Therapeutics, reflecting its focus on developing allogeneic, off-the-shelf CAR-T cell therapies fo
Atsena Selects Lead Candidate for Dual-Vector AAV Gene Therapy Program in Stargardt Disease
July 9, 2026 — Atsena Therapeutics announced the selection of a lead clinical candidate for ATSN-401, its investigational gene therapy program for Stargardt disease, the most common inherited macular dystrophy. The company is now advancing ATSN-401 through IND-enabling studies. Stargardt disease is most commonly caused by mutations in the ABCA4 gene, which e
Apertura Enters CRADA with NIH Institutes to Evaluate BBB-Crossing AAV Capsid for NPC1 Gene Therapy
July 09, 2026 — Apertura Gene Therapy announced that it has entered into a Cooperative Research and Development Agreement, or CRADA, with the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Human Genome Research Institute to evaluate systemic delivery of an investigational gene therapy for Niemann-Pick Disease