Eli Lilly confirmed it was terminating the development of three candidates. Image credit: Jennie Book / Shutterstock.com

The UK and Japan have announced a sweeping pharmaceutical and technology partnership aimed at accelerating drug discovery.

A letter signed by the groups asks Congress to ensure that scientific expertise remains a priority in the search for a new director of the National Institute of Neurological Disorders and Stroke.

Belief BioMed Congratulates Partner AskBio on IND Acceptance by FDA for Investigational Gene Therapy for Late-Onset Pompe Disease  Yahoo Finance

Genetic conditions like Dravet syndrome, which causes severe childhood epilepsy, are hard to tackle with traditional gene therapy. New approaches in the works include using antisense therapy to boost…

The US Food and Drug Administration (FDA) has announced it will take a flexible approach to reviewing all new cell and gene therapy (CGTs) biologics license applications (BLAs) to expedite the development of these products, rather than applying its flexibilities on a case-by-case basis. @

Belief BioMed proudly announces AskBio's FDA IND acceptance for AB-1009, a gene therapy for late-onset Pompe disease, marking a vital milestone.

Durham, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) --

Everly Green, whose family lives in Fort Collins, has a rare mutation in a gene called FRRS1L, pronounced “frizzle,” which affects how cells in her brain communicate.

Yu et al. demonstrate that diverse frontal eye field signals converge on motor-selective superior colliculus neurons to generate saccades, revealing visuomotor convergence and establishing retrograde ...

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient died.

Focal refractory epilepsy (FRE) affects millions worldwide, with around 2 million patients in the US, UK, and EU alone. Individuals with FRE continue to experience seizures even after trying two appropriate and tolerated medications. These seizures originate from a specific part of the brain, making treatment challenging and leaving patients with limited options. EpilepsyGTx’s Innovative Approach Kent-based biotechnology firm EpilepsyGTx is changing the landscape of epilepsy treatment.

Gene therapy researchers were converging on a holy grail. A few years ago, researchers at labs and companies reported they had engineered viruses that could

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market is projected to reach USD 12.87 billion by 2034  openPR.com

Drug aims to correct genetic defect

Loeb et al. develop an immune-evasive gene therapy vector derived from Muscovy duck, termed AAV.div3A. Rational engineering yields a liver-detargeted variant with enhanced transduction in the heart and diaphragm. AAV.div3A fully evades pre-existing antibodies and vector-induced immunity, enabling effective gene therapy redosing in a Pompe disease model.

UCL Discovery is UCL's open access repository, showcasing and providing access to UCL research outputs from all UCL disciplines.

Viral Vectors-based Gene Therapy for Non-human Primates Market  openPR.com