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A gene therapy aimed at correcting fragile X syndrome by restoring the missing FMR1 protein has demonstrated multiple disease-relevant improvements in a mouse model, according to a...
Avenue Biosciences´ machine learning-powered platform engineers and screens signal peptide variants to increase protein secretion from cells. Circio’s circVec technology has demons...
A new preclinical study suggests that gene augmentation therapy may restore sight in a severe form of inherited night blindness. The work, reported in Gene Therapy, targets complet...
In May 2025, a young boy received an injection delivering a healthy copy of a gene called OTOF into the cochlea of his inner ear — one of the first patients with hereditary hearing...
Targeted gene therapy is increasingly viewed as a promising route for patients with drug-resistant epilepsy, a condition that affects roughly one-third of people living with epilep...
Melbourne researchers have made a gene therapy breakthrough that could restore heart function in children with genetic heart disease, sparing them the need for transplants.
The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of el...
Rather than requiring genetic modification of the viral glycoprotein, G-Link uses a modular protein adaptor to blind native VSV-G tropism and redirect delivery after vector product...
How synthetic DNA and RNA are becoming the discovery engines behind a new generation of precision therapies. The post Rewriting Disease: Oligonucleotides Take Aim at the Untreatabl...
A new study reports a strategy for delivering gene-editing technology directly to human haematopoietic stem cells inside the body, using targeted lipid nanoparticles rather than vi...
Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene ther...
Inside the evolving hemophilia treatment landscape, including AAV-based gene therapies, non-factor prophylaxis, and approaches that aim to reduce treatment burden. The post Gene an...
New research coming out of the University of Toronto has shown that transfer RNA may offer a promising therapeutic avenue for treating genetic diseases.
Wall Street analysts view the deal as a risky, high-reward bet on a treatment that’s nearing a possible regulatory approval and could be superior to existing medications.
New funding from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will support lab studies of a one-time gene therapy candidate for Parkinson’s disease. Shape Therapeu...
A preclinical study showed how chemically enhanced suppressor tRNAs combined with a lung-targeted delivery system can restore production of a critical protein in models of cystic f...
Patients with sickle cell disease are often hesitant to undergo transformative gene therapy—the current lengthy process requires multiple hospital visits to collect enough stem cel...
Shannon Knight, a brain and cognitive sciences PhD candidate and McGovern Institute researcher, focuses on developing a novel gene therapy.
The experimental gene therapy was meant to fix a rare genetic condition that caused a delay in cognitive development.
The collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s propri...
Manufacturing costs for therapies based on adeno-associated viruses (AAV) could fall thanks to careful work on plasmid redesign and cell-line selection by a company with long-term...
The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.
A contract manufacturer running multiple adeno-associated virus (AAV) programs says early engagement between key stakeholders, proactive risk assessments and platform knowledge is...
Sensorion has received French regulatory approval to begin a Phase I/II trial of SENS-601, its investigational gene therapy for GJB2-related hearing loss, with first patient dosing...
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