Experimental drug targets gene driving severe seizures in children
Researchers at the Fralin Biomedical Research Institute are developing a targeted KCNT1 inhibitor that has shown early promise in reducing seizures in preclinical models.
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Researchers at the Fralin Biomedical Research Institute are developing a targeted KCNT1 inhibitor that has shown early promise in reducing seizures in preclinical models.
Chiesi Group and Key2Brain have signed a global license agreement to develop two blood-brain barrier-crossing enzyme replacement therapies for alpha-mannosidosis and Krabbe disease – with the aim to help patients with limited treatment options.
How do new cancer drugs make it to patients? This episode uncovers the challenges, successes, and AI advancements driving oncology treatments from preclinical screening to clinical trials.
Researchers at Southern Medical University have developed a self-propelled ferroptosis nanoinducer that penetrates deeper into tumour tissues - offering a new strategy for safer and more effective cancer treatment.
A new material developed at Cornell University offers a more effective way to deliver mRNA vaccines by replacing polyethylene glycol (PEG) with a water-loving polymer poly(carboxybetaine) (PCB).
SynaptixBio has selected its lead drug candidate, SB H-19642, for clinical trials to treat H-ABC - a rare, fatal neurodegenerative disease with no current cure.
President Trump’s proposed drug pricing reforms are putting pressure on early-stage discovery. To keep pace, teams must rethink how they manage risk, resources and collaboration.
Shift Bioscience has announced new aging research, highlighting the discovery of SB000. This novel single-gene target reverses cellular aging without activating dangerous pluripotency pathways.
What does ethical research look like in drug discovery today? In this interview, Charles River’s Executive Director of Global Animal Welfare shares how global standards, the 3Rs and her own path as a woman in STEM are shaping efforts to reduce animal use in science.
Drug development has long been hindered by fragmented data and complex processes, but a new wave of AI is reshaping the landscape. By integrating genomic, clinical and molecular data, multimodal models are revealing hidden patterns and accelerating more precise advancements in medicine.
While ADCs continue to attract attention in oncology, many developers remain focused on antibodies – overlooking the critical role of payload design. At Sygnature Discovery, Dr Joshua Greally is leading a shift in perspective, advocating for a payload-first approach through the company’s new platform, NewPath ADC. In this interview, he…
Radiopharmaceuticals represent a rapidly advancing field in oncology, using radioactive compounds to both detect and treat cancer at the molecular level. This article explores how targeted radiation is improving patient outcomes while reducing systemic toxicity.
Despite the promise of gene therapies, automated red blood cell exchange (aRBCX) remains an underutilised therapy in the management of sickle cell disease (SCD). In this article, Dr Aaron Haubner and Carly Newton of Terumo Blood and Cell Technologies, highlight the urgent need for partnerships and equitable access to this…
Researchers at the University of Illinois have achieved the first successful metabolic labelling of platelets, a key step toward using them in targeted drug delivery. The technique could enable short-lived, precision therapies for cancer, immune conditions, and clotting disorders.
Preclinical and clinical development are critical to the success of ADCs, where early strategic decisions regarding manufacturability and scalability can make or break a project. Iwan Bertholjotti of Lonza emphasises the importance of proactive planning to avoid costly delays and ensure smooth progress towards clinical success.