Groundbreaking CoCas9 Enzyme Unveiled: A Compact and Efficient CRISPR Tool for Genetic Therapy
Introduction: A revolutionary stride has been made in the field of genetic engineering, as researchers from the University of Trento introduce a novel CRISPR enzyme, CoCas9, which promises to enhance the delivery and efficacy of in vivo gene therapy using...
Pfizer’s New Gene Therapy BEQVEZ™ Wins FDA Approval for Hemophilia B
In a notable advancement for hemophilia treatment, Pfizer Inc. (NYSE: PFE) has received U.S. Food and Drug Administration approval for BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy aimed at adults suffering from moderate to severe hemophilia B. This...
Astellas Teams Up with Poseida Therapeutics for Innovative CAR-T Cell Therapy Development
Astellas Pharma, through its subsidiary Xyphos Biosciences, has entered into a second collaborative agreement with Poseida Therapeutics to pioneer the development of off-the-shelf cell therapies for solid tumors. This follows their initial partnership in 2023, which...
Cytiva Introduces New Cell Manufacturing Lines for Viral Vectors
Viral vector-based gene therapies hold immense promise for treating a multitude of diseases. However, their widespread adoption has been hindered by inefficient manufacturing methods and the absence of established standardized processes, resulting in high costs and...
Top 10 Best-Selling Cell and Gene Therapies
When it comes to cell and gene therapy revenues, it’s feast or famine. The feast side is well covered in this A-List, which ranks the top 10 therapies according to sales figures from 2023. The famine side begins almost exactly where this A-List ends. Future A-Lists...
Cytiva Introduces ELEVECTA Cell Lines
Global life sciences company, Cytiva, is creating solutions to the challenges of viral vector-based gene therapies with its new ELEVECTA cell lines. According to Emmanuel Abate, President Genomic Medicine, Cytiva: “To make gene therapy a realistic option for more...
An electrifying discovery may help doctors deliver more effective gene therapies
Experimental treatment groups involving hepatocytes exposed to various doses of AAV, AAV* (AAV pre-treated with EP), or plasmids, with or without electric field pulse (EP) exposure (illustrations not drawn to scale). Credit: PLOS ONE (2024). DOI:...
Lilly scraps trials of Verzenio, Jardiance and a gene therapy from Prevail
As part of its quarterly cleaning, Eli Lilly removed a few trials from its pipeline, including late-stage studies of approved cancer drug Verzenio and its Boehringer Ingelheim-partnered drug Jardiance. The Indianapolis-based pharma giant also removed a Phase 2 trial...
Researchers explore new cell target for cystic fibrosis treatment
Schematic model of working hypothesis of how (A) pulmonary ionocytes and (B) club cells function in pHBE cell culture stimulated with forskolin plus IBMX. Credit: American Journal of Respiratory and Critical Care Medicine (2024). DOI: 10.1164/rccm.202309-1565OCA team...
Groundbreaking Intravenous Gene Therapy Offers New Hope for Metachromatic Leukodystrophy Patients
A recent study conducted by researchers from the TIDU GENOV at the Institut du Cerveau in Paris, France, presents promising results for the treatment of Metachromatic Leukodystrophy (MLD), a severe neurodegenerative disease. Led by Emilie Audouard with key...
Regeneron & Mammoth Partner on CRISPR Therapies
Regeneron Pharmaceuticals Inc. and Mammoth Biosciences Inc., have joined forces to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types. Regeneron is developing adeno-associated viral vectors (AAVs) using...
Abzena Launches EpiScreen® 2.0, a Next Generation Immunogenicity Tool for De-Risking the Development of Complex Biologics and Bioconjugates
Abzena, the leading end-to-end integrated CDMO for complex biologics and bioconjugates, has announced the launch of its enhanced bioassay platform EpiScreen® 2.0, a comprehensive suite of assays that predict and evaluate potential risks of preclinical immunogenicity...
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