Shilpa Rao’s Post

🧬 Gene Editing: Transforming Biotech Frontiers 🌐 From the monumental Human Genome Project to today's accessible genome sequencing, our journey through genetics has seen incredible strides. The once $95 million endeavor is now a $950 reality. Amidst this genomic understanding, the spotlight turns to gene editing—an exhilarating frontier in biotechnology. Enter CRISPR Therapeutics, a Swiss-based innovator leveraging CRISPR/Cas9 to revolutionize genetic engineering. Their groundbreaking platform holds the promise of treating hereditary diseases, aiming to impact millions of lives. CRISPR Therapeutics envisions a future where gene therapy becomes a beacon of hope. Not only do they pioneer cures, but they also extend their services to in-utero and in-vitro patients, enabling proactive screening for hereditary diseases before birth. #GeneEditing #BiotechInnovation #CRISPRTherapeutics #GenomicRevolution

Thorough analysis of current state and future scenarios in cell and gene therapies. #iqvia #cellandgene

I recently came across an extraordinary article that moved me to tears. It chronicled the relentless efforts of researcher Arkasubhra Ghosh and his team in their quest to develop a gene therapy for Uditi Saraf, a young woman suffering from a rare neurodegenerative disease. Despite the numerous challenges and the tragic outcome, the dedication and passion of these scientists are truly inspiring. Their work represents a significant leap forward in the field of gene therapy. The potential of CRISPR and other gene-editing technologies to treat not only common blood disorders but also rare genetic conditions is nothing short of revolutionary. This cutting-edge approach offers hope to countless patients and their families, who have often been overlooked by traditional pharmaceutical research. The efforts of Ghosh and his collaborators illustrate the messy yet hopeful state of modern drug development. Even though they couldn't save Uditi, the lessons learned from their work will undoubtedly pave the way for future breakthroughs. This story is a poignant reminder that perseverance in scientific research, even in the face of seemingly insurmountable odds, can lead to transformative change. One day, these efforts will pay off. Gene therapy is the new age of treatment, and it will change every scenario. #GeneTherapy #CRISPR #ScientificResearch #HealthcareInnovation #Perseverance #Hope

🌟 The Future of Gene Therapy: Why LNPs Are Gaining Momentum! 🌟 Traditional gene therapy often relies on viral vectors, which can pose safety risks, cause immune reactions, and have limited capacity for genetic material. These challenges have restricted their broader clinical use. Enter Lipid Nanoparticles (LNPs)! 🎉 These non-viral vectors are changing the game with their exceptional safety profile, ability to administer multiple doses, and large genetic capacity. Plus, they’re easier to design and manufacture compared to their viral counterparts. LNPs are quickly becoming the preferred choice in gene therapy, offering a promising alternative that overcomes many of the limitations of traditional methods. 🚀💡 #GeneTherapy #Innovation #LipidNanoparticles #Healthcare #Biotechnology #NonViralVectors https://lnkd.in/gpQynpzH

Genetic disease knows no borders. Neither should #CRISPR cures. Essential reading in https://lnkd.in/g3dKk9Vn ... showcasing the #yeswecan approach to providing gene therapy cures in India, Brazil, Thailand, and beyond. A key goal of the Innovative Genomics Institute Danaher Corporation for CRISPR Cures https://lnkd.in/gUKZRKRq is to have gene editing follow that example and build Platform approaches to make such cures affordable, manufacturable, and deliverable to patients where the unmet medical need is, and not just where the current GMP suites are. #HealthJustice

#LipidNanoparticles #GeneTherapy #NucleicAcidDelivery | 𝗖𝗮𝗻 𝗹𝗶𝗽𝗶𝗱 𝗻𝗮𝗻𝗼𝗽𝗮𝗿𝘁𝗶𝗰𝗹𝗲𝘀 𝘁𝗿𝗮𝗻𝘀𝗳𝗼𝗿𝗺 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀? 🟣 𝗔𝗶𝗺: Explore the 60-year evolution of lipid nanoparticles (LNPs) in delivering genetic information to target cells. 🟣 𝗞𝗲𝘆 𝗙𝗶𝗻𝗱𝗶𝗻𝗴𝘀: Advances in lipid-based systems have enabled the development of LNPs for nucleic acid delivery. Clinically approved examples include COVID-19 LNP mRNA vaccines and Onpattro (patisiran) for treating hATTR. Research highlights the role of positively charged cationic lipids and ionizable cationic lipids in developing effective LNPs. 🟣 𝗧𝗮𝗸𝗲 𝗛𝗼𝗺𝗲 𝗠𝗲𝘀𝘀𝗮𝗴𝗲: LNPs represent a significant breakthrough in gene therapy, offering potential delivery solutions for various genetic treatments. Check here for more https://lnkd.in/gNXuWQ6h and a big thank you to Pieter Cullis & Phil Felgner for their valuable research in Nature Reviews Drug Discovery. 𝗪𝗲𝗹𝗰𝗼𝗺𝗲 𝘁𝗼 NanoSphere – 𝘆𝗼𝘂𝗿 𝗽𝗿𝗲𝗺𝗶𝗲𝗿 𝗱𝗲𝘀𝘁𝗶𝗻𝗮𝘁𝗶𝗼𝗻 𝗽𝗹𝗮𝘁𝗳𝗼𝗿𝗺 𝗳𝗼𝗿 𝗻𝗮𝗻𝗼𝗺𝗲𝗱𝗶𝗰𝗶𝗻𝗲, 𝘀𝗽𝗲𝗰𝗶𝗮𝗹𝗶𝘇𝗶𝗻𝗴 𝗶𝗻 𝘁𝗵𝗲 𝗹𝗮𝘁𝗲𝘀𝘁 𝘂𝗽𝗱𝗮𝘁𝗲𝘀 𝗶𝗻 𝗻𝗮𝗻𝗼𝘁𝗲𝗰𝗵𝗻𝗼𝗹𝗼𝗴𝘆 𝗮𝗻𝗱 𝗻𝗼𝗻-𝘃𝗶𝗿𝗮𝗹 𝗱𝗲𝗹𝗶𝘃𝗲𝗿𝘆 𝗽𝗹𝗮𝘁𝗳𝗼𝗿𝗺𝘀 (𝗟𝗡𝗣𝘀) 𝗳𝗼𝗿 𝗻𝘂𝗰𝗹𝗲𝗶𝗰 𝗮𝗰𝗶𝗱𝘀.  Follow us for more: https://lnkd.in/ecNd8VDP

Gene therapy medicinal products (GTMPs), which in the European Union (EU) are a subset of advanced therapy medicinal products (ATMPs), may have the potential to transform the treatment landscape for many diseases ranging from inborn errors of metabolism to malignancies, offering the promise of treating diseases that may range from debilitating to fatal. The science of gene therapy (GT) was catalyzed by breakthroughs in DNA technology that led to research strategies aimed at replacing or adding genes. These techniques often employ viral vectors. https://lnkd.in/epv6CuTk

The cell and gene therapy sector is on the rise with record funding, clinical research, and new launches in recent years. Swipe to discover key data points demonstrating this rise, direct from the Institute’s report, Strengthening Pathways for Cell and Gene Therapies: Current State and Future Scenarios. If you found this document useful, be sure to follow IQVIA Institute’s LinkedIn page for more insightful information. Already following us? Share this post to spread the word. #DataSciences #Biotech #LifeSciences #Pharma Diego Correa, MD, MSc, PhD Barbara Isquith Arone, MS Rob Glik Adrian McKemey Carolina Ricarte Dan Winkelman Amritha J. Joshi Venugopal Mark Trusheim American Society of Gene & Cell Therapy Alliance for Regenerative Medicine ISCT, International Society for Cell & Gene Therapy

Too good to be true? Researchers in China say they’ve successfully cured a patient’s type 2 diabetes using a novel regenerative gene therapy. While a one-time therapy that frees hundreds of millions from the burden of daily or weekly injections sounds promising, the devil will be in the details.   Will the new therapy, which works by programming a patient’s immune cells to create pancreatic islet tissue outside the body, succeed in larger studies? And if so, can the manufacturing process be sufficiently streamlined to make it accessible to the more than 500 million people (and growing!) who suffer from diabetes today? Considering the challenges we face making current cell and gene therapies affordable and accessible to comparatively smaller patient populations, that’s going to be a tall order.

AAV is the leader of gene therapy. But what are it's pros and cons? Let's break it down. Pros: - Current leader in vector-based gene therapy - Payload doesn't integrate into patient DNA (normally) - FDA-approved therapies on the market - Can target specific tissues for precise delivery Cons: - High cost of manufacturing - Small payload size - Good quality at scale is a challenge - Single dose administration due to high immunogenicity (for now) - Liver toxicity concerns (for now) I'm excited to see how recent advances in capsid design will fix these drawbacks. Anything else you'd add? Drop it in the comments. For content that breaks down the science of CGT: Check out #cgt_simplified “Created with BioRender.com” #genetherapy #aav #biotechnology