Nabil Laoudji’s Post

AI-enabled biotechnology holds the potential to advance the approval of safer, more effective and accessible gene therapies across a wide spectrum of diseases. However, AI applications come with their own set of challenges. Limited and heterogeneous data, extensive compute power requirements and expert knowledge necessary for annotating gene therapy data are just a few bottlenecks to consider. Fortunately, these issues are solvable. Explore the future of gene therapy and discover advanced, customizable AI models that can assist in overcoming these hurdles: https://hubs.li/Q02x0prf0 #GeneTherapy #AI #AAV

🚨Gene Therapy: How We Got Here and Where We're Headed🚨 The United States Food and Drug Administration (FDA) defines human gene therapy as a therapy that ”seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use". But what exactly does this mean 🤔⁉️ What was the first gene therapy⁉️ How many different kinds of gene therapies are there⁉️ And how does gene therapy work⁉️ In this issue, we delve into the science and history of gene therapy! 📖 Let's start with the basics of gene therapy 🧬: There are two main categories of gene therapy - in vivo and ex vivo gene therapy. The main difference between these two categories is that in ex vivo gene therapy, cells are removed from the body 🧫 to then have gene(s) delivered to them externally while in vivo gene therapy delivers genes directly to the cells themselves! 💉 There are three main approached to gene therapy - each falling into one of the two categories. These are: 1. ➕ Gene Addition is for diseases where the patient is lacking a certain protein. In this approach, a viral vector delivers the gene of interest to the patient’s cells 🦠📦🧬 By adding in a specific gene for a desired protein, the modified cell is now able to make more of that protein! One recent FDA-approved gene addition drug is Elevidys which is for individuals with Duchenne Muscular Dystrophy (DMD) 💪! 2. ❌Gene Silencing: is geared toward diseases where too much of a protein is being made. When the gene gets delivered to its target cell, it either fully inhibits or at least slows the progression of the gene making the unwanted protein. Inclisiran was recently approved by the FDA and it is a gene silencing therapy meant to treat people with low-density lipoprotein cholesterol (LDL-C or the ‘bad’ kind of cholesterol) who are at a high risk for developing heart disease ❤️🩹 3. 🎯 Gene Targeting: is a very precise kind of gene therapy. Instead of becoming incorporated into the DNA of the host cell upon delivery by a viral vector, gene targeting therapy uses certain systems to manually change, or edit, the DNA already in that cell ✂️🧬 One great example of this kind of gene therapy is the CRISPR/Cas9 system and the first-ever FDA-approved gene targeting therapy Casgevy for sickle cell disease (SCD) 🌙🩸! 📰 To learn more about these gene therapies, their history, and more recent FDA-approved drugs, read this week's #newsletter at https://conta.cc/3OKroRF and join our mailing list to stay informed https://rb.gy/isydg 🧠 #GeneTherapy #CRISPR #Genetics #Innovation #Science #newsletter #Elevidys #Inclisiran #Casgevy

Cell and gene therapy is at another pivotal moment. While challenges remain, ARM’s 2024 Cell & Gene Meeting on the Mesa outlined ideas on how the sector can mature into a new phase for manufacturing and commercialization. Inside Precision Medicine covers more on how industry leaders at #CGMesa24 are shaping the future for cell and gene therapy. https://lnkd.in/ez9QJzM8 #cellandgenetherapy

🚀 Innovations in Gene Therapy: Exploring Current Trends and Future Directions 🧬 We recently had the opportunity to interview Ibrahim Malki, Cell and Gene Therapy Associate at Minaris Regenerative Medicine, to explore the groundbreaking progress in gene therapy. The industry is evolving rapidly, with innovations like CRISPR technology and enhanced delivery mechanisms driving the field forward. Key takeaways include: 💡 Emerging technologies like CRISPR are setting new standards for precision in treating genetic disorders ⚙️ Innovative delivery methods are addressing challenges in targeted treatment 🧩 Clinical trials still face hurdles in scalability, but new strategies are improving success rates ⚖️ Ethical and regulatory factors are playing a critical role in shaping the future of gene therapies As gene therapy moves from the lab to clinical practice, these developments are reshaping the possibilities for treating complex genetic conditions. Stay tuned for more insights from this insightful discussion. Link to KOL: https://lnkd.in/gZ8wHZvq TechSci Research, Karan Chechi, Alwin Samuel, Shaurya Singh, MANVI SURI

🚀 Exciting news in biotech this week! 🧬 First Gene Therapy Approved for Brain Delivery The FDA has approved the first-ever gene therapy for patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This groundbreaking treatment from PTC Therapeutics is administered directly to the brain, marking a significant advancement in how gene therapies can be applied. AADC deficiency is a rare genetic disorder that affects neurotransmitter production, leading to severe motor and cognitive disabilities. This approval represents hope for affected families and highlights the potential of targeted therapies in rare diseases. 💡 Early-Stage Innovation PTC Therapeutics, a company known for its focus on rare diseases, has taken a bold step in the gene therapy space. Their collaboration with academic institutions has been crucial in developing this treatment. This approval is a testament to the importance of early-stage biotechs in pushing the boundaries of medical science. 🤝 Collaboration is Key In the landscape of biopharma, partnerships are vital. Smaller companies like PTC Therapeutics often collaborate with larger organizations and research institutions to bring innovative solutions to market. These collaborations not only provide the necessary resources but also foster an environment where new ideas can thrive. 📈 Looking Ahead As we celebrate this milestone, it's essential to support and nurture early-stage biotechs that are ready to take on challenges in rare diseases and beyond. The future of biotech relies on the creativity and determination of these innovative companies. Stay tuned for more updates as we continue to champion the incredible work happening in the biotech sector!

Gene Therapy Challenges: A Setback for Bluebird Bio The gene therapy landscape has encountered a significant hurdle. Bluebird Bio's gene therapy for a rare neurological condition has been linked to 7 cases of blood cancer in children. Background Bluebird Bio's gene therapy, designed to treat transfusion-dependent beta-thalassemia and sickle cell disease, uses a disabled virus to deliver healthy copies of a gene into patients' stem cells. Concerns Raised The reported cases of blood cancer (myelodysplastic syndrome and acute myeloid leukemia) in children treated with Bluebird Bio's gene therapy have sparked concerns: 1.⁠ ⁠Vector safety: The use of viral vectors to deliver genes raises questions about their potential to cause cancer. 2.⁠ ⁠Insertional oncogenesis: The risk of genetic material inserting into unintended locations, leading to cancer. 3.⁠ ⁠Long-term efficacy and safety: Gene therapies' durability and potential long-term consequences. Implications These developments underscore the challenges facing gene therapy: 1.⁠ ⁠Regulatory scrutiny: Increased oversight and rigorous testing will be crucial. 2.⁠ ⁠Patient safety: Balancing therapeutic benefits with potential risks. 3.⁠ ⁠Innovation: Encouraging responsible innovation while addressing safety concerns. The Future of Gene Therapy Despite setbacks, gene therapy holds promise. Researchers must: 1.⁠ ⁠Invest in safety research 2.⁠ ⁠Improve vector design 3.⁠ ⁠Enhance regulatory frameworks Let's continue the conversation: What are your thoughts on gene therapy's challenges and future?plz share #GeneTherapy #BluebirdBio #CancerRisk #RegulatoryScrutiny #PatientSafety #Innovation #Biotech #Pharmaceuticals #Healthcare

Thrilled to share this interview I did with TechSci about the current state and future potential of cell and gene therapy! #TechSci #CellandGeneTherapy #CGT #Biopharma #Biopharmaceuticals #Biotech #Biotechnology #SubjectMatterExpert #SME #RegenerativeMedicine #Innovation #PersonalizedMedicine #GeneEditing #Innovation #InnovationInHealthcare #HealthcareTransformation #ClinicalTrials

Top Story of 2024: Switching Gene Therapy On and Off with a Pill Gene therapy has a reputation as a one-time intervention that has a lifelong effect. It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a daily basis, with a simple pill? The possibility has inspired MeiraGTx to develop a riboswitch technology that is designed to allow for the precise, dose-responsive control of gene expression by oral small molecules. https://hubs.li/Q02_RC3f0

🌟 Exciting Advances in Gene Therapy! 🌟 In recent years, the field of gene therapy has seen tremendous progress, offering new hope for patients with previously unmet medical needs. A shining example of this breakthrough is Hemgenix, a revolutionary treatment for hemophilia B. Hemophilia B is a genetic disorder characterized by insufficient blood clotting due to a deficiency in factor IX, a crucial clotting protein. Patients have relied on frequent and costly infusions of clotting factors to manage their condition for years. However, Hemgenix is changing the game. Hemgenix is a gene therapy that delivers a functional copy of the gene responsible for producing factor IX directly into the patient’s liver cells. This innovative approach allows the patient’s body to produce the necessary clotting protein on its own, potentially eliminating the need for regular infusions. The results have been remarkable. Clinical trials have shown significant increases in factor IX levels and a substantial reduction in bleeding episodes. This not only improves the quality of life for patients but also represents a major advancement in the treatment of hemophilia B. South Korea is at the forefront of gene therapy research, with numerous clinical trials underway. The success of treatments like Hemgenix underscores the critical importance of gene therapy in addressing unmet medical needs and providing long-term solutions for genetic disorders. As we continue to explore and expand the potential of gene therapy, we are paving the way for a future where genetic disorders can be effectively treated, improving the lives of countless individuals worldwide. 🌍💉🔬 Please refer to this RSS feed. You are able to know how many clinical trials in Gene therapy are being conducted in South Korea. https://lnkd.in/g6tmvdqz #GeneTherapy #HemophiliaB #Hemgenix #MedicalInnovation #HealthcareRevolution #ClinicalTrials #SouthKorea #Biotech #KoreaClinicalTrials #CRO #RareDisease #CRNResearch #씨엔알리서치 #유전자치료제 #임상시험 #글로벌임상시험 #희귀질환

1) Calling for better delivery vectors (than AAV). 2) Treatment window: before immune system reaches maturity? 3) Scope: What about diseases (conditions) not caused by single mutation.