One CaBP4 viral dose restored dim-light vision in adult dogs and thickened a retinal synapse that development never finished. Ribbons grew toward normal length.

Karl Deisseroth, Peter Hegemann, Georg Nagel were awarded the prize for their work on light-gated ion channels and optogenetics.

The whole-brain map—the first of its kind in a vertebrate—identifies five distinct neuron groups.

Re-administration of adeno-associated virus (AAV)-mediated gene therapy remains challenging due to neutralizing antibodies (NAbs) induced by the initial dose. We previously conducted a single-arm trial showing that single-dose administration of AAV-hOTOF gene therapy in individuals with OTOF-related deafness is safe and leads to hearing improvements. Here we initially demonstrate that AAV1-hOTOF re-administration to the contralateral ear in Otof−/− mice with peak serum NAb titers successfully rescued hearing with limited immune activation. After a protocol amendment of our trial, four patients (aged 2.2–3.4 years) with pre-existing NAbs (titers 1:135–1:3,645), who had previously received a single dose of the gene therapy, were enrolled to receive a second dose in the contralateral ear, as part of the ongoing trial, with a follow-up ranging from 26 weeks to 52 weeks. The primary endpoint was the occurrence of dose-limiting toxicities at 6 weeks and secondary endpoints included safety an

Treatment is the first for MPS type IIIA

B.R.P. is supported by the National Institute of Health (grant numbers R01EY024995, R24EY032434, U19NS132296), the Retina Research Foundation, the Daniel M. Albert Chair in McPherson Eye Research Institute, and an unrestricted Grant from Research to Prevent Blindness, Inc. to the UW-Madison Department of Ophthalmology and Visual Sciences. J.A.S. is supported by the National Institute of Health (R01EY033049), the Eye and Ear Foundation of Pittsburgh, and an unrestricted grant from Research to Prevent Blindness, Inc. to the Department of Ophthalmology, University of Pittsburgh. Open Access This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License, which permits any non-commercial use, sharing, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if you modified the licensed material. You do not have permission under this licence to share adapted material derived from this article or parts of it. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and...

The bankrupt gene editing biotech Sangamo is set to sell chunks of its business to PTC Therapeutics and Eli Lilly for up to $264 million following an auction procedure.

With a second delayed report of a child's death in China, criticism is rising of a looser form of clinical trial. One expert: what "bothers me is the lack of transparency.'

A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment more like a routine procedure than a bespoke drug.

Two researchers — one in Massachusetts and one in Shanghai — hoped for the same breakthrough: a gene therapy for deaf children. New Chinese investment in science propelled the one who got there first.

Daniel Cressy, 23, underwent gene therapy at Manning Family Children’s Hospital.

The first-of-its-kind genetic medicine treats a rare and profound otoferlin-related hearing loss, and will be made available by Regeneron for free in the United States.

Li et al. present the preclinical development and phase 1 trial results of LX102, an rAAV2-based gene therapy designed for sustained VEGF-Trap secretion. LX102 achieved long-term efficacy in animal models and shows a favorable safety profile with durable clinical benefits in patients with nAMD in this phase 1 study.

AAV Gene Therapy Stabilizes Retinal Degeneration in Choroideremia Key Highlights Medicine · Biomedicine A systematic review and meta-analysis assessed the efficacy and safety of AAV-mediated gene therapy for choroideremia, a degenerative retinal disease. The therapy showed modest functional benefits and structural preservation signals, stabilizing retinal degeneration rather than restoring vision. For a medical student focused on evidence-based practice, this study highlights the potential of disease-modifying gene therapies and the importance of earlier intervention and optimized delivery strategies for translating genetic treatments into clinical care.

The FDA recently approved the cellular rejuvenation therapy ER-100 for human clinical trials. While vision is the first target, it could have applications for a variety of age-related disease.

The genomic medicine company is weighing a sale or partnership as it advances a BLA-ready Fabry gene therapy and two CNS programmes.

NIH Funding Opportunities and Notices in the NIH Guide for Grants and Contracts: Request for Information: Proposal to Cap the Number of Simultaneous Research Project Grants per Principal Investigator ...

Israeli doctors delivered gene therapy into the brain of an eight-month-old baby with a rare genetic epilepsy, a world first. The procedure replaced the missing WWOX gene via a single injection at Schneider Children's Medical Center. The child has been discharged and is in stable condition.

By combining magnetoencephalography and eye tracking, this study sheds light on why people fixate on some parts of natural scenes longer than others. Rather than visual complexity, fixation durations ...

Jeffery Taubenberger, the acting head of the NIAID, the NIH’s infectious disease institute, is reported to have stepped down.

Kitakaze et al. establish proof of concept for treating GM2 gangliosidosis using a single gene therapy encoding a modified lysosomal enzyme. By simplifying gene design and demonstrating efficacy and safety across species, the study provides a foundation for clinical translation in neurodegenerative lysosomal disorders.

Zhang et al. report an AAV6-based intracameral gene delivery strategy that selectively transduces corneal endothelium, achieves long-term transgene expression, and preserves corneal transparency across multiple models of hereditary corneal dystrophy.

Congressional impasse has halted billions in research funding for innovative technologies

Mass General Brigham continues to build its coffers to fund new cancer research as the health care giant braces for its split from Dana-Farber Cancer Institute.

This study tests NeuroD1 AAV-based gene therapy in a non-human primate Alzheimer's disease model. The therapy prevents neuronal damage, inhibits hippocampal atrophy, and reduces neuroinflammation. It also repairs vascular and blood-brain barrier damage, restores cerebrospinal fluid biomarkers, enhances hippocampal glucose metabolism, and improves spatial memory. Transcriptome analysis reveals enhanced neuronal function and reduced neuroinflammation, supporting its therapeutic potential. ABSTRACT There is a pressing demand for neuroregenerative treatment for Alzheimer's disease (AD). Recently, a NeuroD1-mediated neuroregeneration strategy has been proposed, yet its efficacy remains untested in non-human primate (NHP) AD models closely reflecting human pathology. This study evaluates the therapeutic potential of NeuroD1 AAV-based gene therapy in an NHP AD model with hippocampal hTau overexpression, utilizing immunostaining, fluorescence/confocal imaging, MRI and FDG PET scans, Simoa CSF biomarker analysis, behavioral tests, and bulk RNA sequencing. NeuroD1 AAV-based gene therapy prevents neuronal damage and degeneration, inhibits hippocampal atrophy, and reduces neuroinflammation in NHP AD models. It also repairs vascular and BBB damage, restores CSF AD biomarker levels, improves hippocampal glucose metabolism, and enhances spatial working memory. Transcriptome analysis further reveals upregulated neuronal function and synaptic transmission, along with downregulated neuroinflammation and apoptosis. Collectively, our findings demonstrate that NeuroD1 AAV-based gene therapy repairs and restores brain structure and function in NHP AD models, highlighting its therapeutic potential.

Biopharma is a fast-growing world where big ideas come along daily. Our subscribers rely on Fierce Biotech as their must-read source for the latest news, analysis and data in the world of biotech and ...

For all its blockbuster hopes and high-profile status in the gene therapy field, Roctavian's commercial saga has ended.

The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments. The new guidance would enable the agency to approve new treatments for rare diseases based on evidence for a "plausible mechanism" for how the treatment would work. The policy aims to speed the use of state-of-the-art technologies like gene-editing to create treatments tailored to individual patients suffering from diseases that are so rare that it would be difficult if not impossible to conduct a traditional study first. "For decades families heard the same thing: There are not enough patients. The approval will take too long. You just have to wait for the science to catch up with your child, " Health and Human Services Secretary Robert F. Kennedy Jr. said at a briefing announcing the proposed new policy. "That ends today. Individualized medicine is no longer theoretical." "Historically, rare diseases at the FDA have been an afterthought," added FDA Commissioner Marty Makary said at the briefing. "We've come a long way." The new approach, outlined broadly in November, would apply to diseases where there is a plausible expectation that the...