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CRISPR Base Editing Shows Promise for Cystic Fibrosis Mutation

CRISPR Base Editing Shows Promise for Cystic Fibrosis Mutation

Researchers have developed a CRISPR-based gene therapy to address a challenging mutation in cystic fibrosis (CF) patients. The study, published in Science Translational Medicine, focuses on the 1717-1G>A mutation, which affects a subset of CF patients who do not benefit from existing therapies. The team used an adenine base editing strategy to correct this mutation in cell models, achieving significant editing efficiency with minimal off-target effects. This approach could potentially offer a new treatment avenue for patients with this specific mutation, who currently have limited options.
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Viral Capsid Development Market Projected to Reach USD 840.63 Million by 2035

Viral Capsid Development Market Projected to Reach USD 840.63 Million by 2035

Viral Capsid Development Market Projected to Reach USD 840.63 Million by 2035
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Thryv Therapeutics Begins Patient Dosing in Long QT Syndrome Study with FDA Fast Track Designation

Thryv Therapeutics Begins Patient Dosing in Long QT Syndrome Study with FDA Fast Track Designation

Thryv Therapeutics Begins Patient Dosing in Long QT Syndrome Study with FDA Fast Track Designation
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OpenAI Introduces ChatGPT for Clinicians to Enhance Medical Practice

OpenAI Introduces ChatGPT for Clinicians to Enhance Medical Practice

OpenAI Introduces ChatGPT for Clinicians to Enhance Medical Practice
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Anixa Biosciences to Present Ovarian Cancer CAR-T Therapy at Major Conference

Anixa Biosciences to Present Ovarian Cancer CAR-T Therapy at Major Conference

Anixa Biosciences, a biotechnology company focused on cancer treatment, announced that Cheryl Cox, Operations Director at Moffitt Cancer Center, will present their ovarian cancer CAR-T therapy at the International Society for Cell & Gene Therapy 2026 Annual Meeting in Dublin, Ireland. The presentation will cover the Phase 1 clinical trial of lira-cel, a therapy targeting the follicle-stimulating hormone receptor in recurrent ovarian cancer. The trial is enrolling women who have not responded to at least two prior therapies. The conference, scheduled for May 6-9, 2026, is a significant event in the field of cell and gene therapy, featuring numerous experts and presentations on the latest scientific advancements.
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NIT Rourkela Announces JRF Vacancy for Biotechnology Research

NIT Rourkela Announces JRF Vacancy for Biotechnology Research

The National Institute of Technology (NIT) Rourkela has announced a vacancy for a Junior Research Fellow (JRF) in the field of biotechnology. This position is part of a project focused on developing an in-silico model for targeted drug delivery against viral respiratory disorders. The project, funded by the ANRF in Mechanical Engineering, will be led by Prof. Kishore Singh Patel and is set to last for three years. Candidates with a first-class M.E./M.Tech in Biotechnology or related fields, and who have qualified through national eligibility tests like NET/GATE, are encouraged to apply. The role involves computational fluid dynamics analysis and AI/ML-based modeling, offering a monthly stipend of ₹37,000 for the first two years and ₹42,000 for the third year.
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Allogeneic Cell Therapy Market Projected to Reach USD 5968.77 Million by 2035

Allogeneic Cell Therapy Market Projected to Reach USD 5968.77 Million by 2035

Allogeneic Cell Therapy Market Projected to Reach USD 5968.77 Million by 2035
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Entos Pharmaceuticals and L-CMD Research Foundation Partner to Develop Therapy for Muscular Dystrophy

Entos Pharmaceuticals and L-CMD Research Foundation Partner to Develop Therapy for Muscular Dystrophy

Entos Pharmaceuticals and L-CMD Research Foundation Partner to Develop Therapy for Muscular Dystrophy
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Parents Face Heartbreaking Decision After Unborn Child Diagnosed with Sanfilippo Syndrome

Parents Face Heartbreaking Decision After Unborn Child Diagnosed with Sanfilippo Syndrome

Emily and Angus Forrester, parents from London, faced a devastating decision after discovering their unborn child was diagnosed with Sanfilippo syndrome, a rare genetic disorder also known as childhood dementia. Their two-year-old daughter, Leni, was diagnosed with the condition, which leads to the loss of motor skills and is typically fatal by the mid-teens. After genetic testing revealed both parents carried the recessive gene, they learned their unborn child would also be affected. With no cure available, the couple decided to terminate the pregnancy to focus on Leni's care. The family is now advocating for research funding and newborn screening to detect such conditions earlier.
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Entos Pharmaceuticals and L-CMD Foundation Partner for Muscular Dystrophy Therapy

Entos Pharmaceuticals and L-CMD Foundation Partner for Muscular Dystrophy Therapy

Entos Pharmaceuticals has announced a collaboration with the L-CMD Research Foundation to develop a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD). This rare and severe form of muscular dystrophy affects at least 200 children worldwide, characterized by progressive muscle weakness and heart arrhythmia. The partnership aims to leverage Entos' Fusogenix PLV delivery platform and novel gene editing approaches to create potentially curative therapies. Entos, with its expertise in genetic medicines and manufacturing capabilities, is well-positioned to advance this initiative, which could provide much-needed treatment options for affected children.
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Entos Pharmaceuticals and L-CMD Research Foundation Partner to Develop Therapy for Muscular Dystrophy

Entos Pharmaceuticals and L-CMD Research Foundation Partner to Develop Therapy for Muscular Dystrophy

Entos Pharmaceuticals has announced a collaboration with the L-CMD Research Foundation to develop a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD). This partnership aims to leverage Entos' Fusogenix PLV platform, which enables the delivery of nucleic acids to target cells, to create potentially curative therapies for this severe form of muscular dystrophy. L-CMD is characterized by progressive muscle weakness and heart arrhythmia, affecting at least 200 children worldwide. The collaboration seeks to address the lack of treatments for this condition by combining gene editing approaches with muscle-targeted formulations.
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Courier Health Secures $50M Series B Funding to Enhance AI in Biopharma Patient Experience

Courier Health Secures $50M Series B Funding to Enhance AI in Biopharma Patient Experience

Courier Health Secures $50M Series B Funding to Enhance AI in Biopharma Patient Experience