Biovivium Strategies’ Post

Gene&Cell Therapy >> International society criticizes Utah law for permitting unapproved stem cell therapies: The International Society for Cell and Gene Therapy is pushing back against a Utah law that allows healthcare providers to treat patients with placental stem cell therapies that haven’t been approved by the FDA. The group is concerned that the law will open up a way to work around the FDA. The law, known as the “Placental Tissue Amendments,” went into effect May 1 and allows healthcare providers whose scope includes stem cell therapy to treat patients with non-FDA-approved treatments as long as they notify the patient. But stakeholders are worried that the bill will open the door to patients receiving unproven treatments, posing significant safety risks. Specifically, the healthcare provider must provide patients with the following notice in writing: “THIS NOTICE MUST BE PROVIDED TO YOU UNDER UTAH LAW. This health care practitioner performs one or more stem cell therapies that have not yet been approved by the United States Food and Drug Administration. You are encouraged to consult with your primary care provider before undergoing a stem cell therapy.” The group is urging cell and gene manufacturers to push back against the law and be vigilant about the potential for similar laws to pass in other states in the future. Bruce Levine, chair of ISCT’s Ethics of Cell and Gene Therapy Committee, said that the bill goes against the FDA’s efforts to protect patients and creates ethical and legal concerns. “This opens patients to significant health risks, and negatively impacts their right to informed consent as unethical providers may make false or misleading claims about unproven cell- and gene-based interventions,” he said. #lucidquest #genetherapy #celltherapy

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> International society criticizes Utah law for permitting unapproved stem cell therapies: The International Society for Cell and Gene Therapy is pushing back against a Utah law that allows healthcare providers to treat patients with placental stem cell therapies that haven’t been approved by the FDA. The group is concerned that the law will open up a way to work around the FDA. The law, known as the “Placental Tissue Amendments,” went into effect May 1 and allows healthcare providers whose scope includes stem cell therapy to treat patients with non-FDA-approved treatments as long as they notify the patient. But stakeholders are worried that the bill will open the door to patients receiving unproven treatments, posing significant safety risks. Specifically, the healthcare provider must provide patients with the following notice in writing: “THIS NOTICE MUST BE PROVIDED TO YOU UNDER UTAH LAW. This health care practitioner performs one or more stem cell therapies that have not yet been approved by the United States Food and Drug Administration. You are encouraged to consult with your primary care provider before undergoing a stem cell therapy.” The group is urging cell and gene manufacturers to push back against the law and be vigilant about the potential for similar laws to pass in other states in the future. Bruce Levine, chair of ISCT’s Ethics of Cell and Gene Therapy Committee, said that the bill goes against the FDA’s efforts to protect patients and creates ethical and legal concerns. “This opens patients to significant health risks, and negatively impacts their right to informed consent as unethical providers may make false or misleading claims about unproven cell- and gene-based interventions,” he said. #lucidquest #genetherapy #celltherapy

On Cell and Gene therapy and designated pathways The U.S. FDA's recent expansion of Elevidys (delandistrogene moxeparvovec-rokl) approval exemplifies the benefits of conditional approvals in providing early access to life-saving treatments. Originally approved under the Accelerated Approval pathway for ambulatory individuals aged 4 to 5 with Duchenne muscular dystrophy (DMD), Elevidys has now received traditional approval for all ambulatory individuals aged 4 and older, and accelerated approval for non-ambulatory individuals in the same age group. This decision reflects the FDA's consideration of comprehensive data and the urgent need for effective DMD treatments. The initial conditional approval allowed patients earlier access to Elevidys, while additional data collection verified its benefits, leading to full approval. This process highlights the effectiveness of accelerated pathways in addressing critical health needs swiftly while ensuring safety and efficacy through continued research. Elevidys’ approval marks a significant step forward in the fight against DMD, offering hope to many affected by this debilitating condition. #futureofmedicines #ATMPs #patientaccess https://lnkd.in/d53EUdEz

𝗙𝗗𝗔 𝗮𝗽𝗽𝗿𝗼𝘃𝗲𝘀 𝗢𝗿𝗰𝗵𝗮𝗿𝗱’𝘀 𝗟𝗲𝗻𝗺𝗲𝗹𝗱𝘆 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆 𝗳𝗼𝗿 𝗘𝗹𝗶𝗴𝗶𝗯𝗹𝗲 𝗖𝗵𝗶𝗹𝗱𝗿𝗲𝗻 𝘄𝗶𝘁𝗵 𝗘𝗮𝗿𝗹𝘆-𝗼𝗻𝘀𝗲𝘁 𝗠𝗲𝘁𝗮𝗰𝗵𝗿𝗼𝗺𝗮𝘁𝗶𝗰 𝗟𝗲𝘂𝗸𝗼𝗱𝘆𝘀𝘁𝗿𝗼𝗽𝗵𝘆 (𝗠𝗟𝗗) MLD is a rare, fatal genetic disorder caused by a mutation in the gene responsible for encoding the enzyme arylsulfatase A 0 leading to neurological damage and developmental regression. Lenmeldy aims to correct the genetic cause of MLD by inserting copies of the human ARSA gene ex vivo  into the genome of a patient’s own hematopoietic stem cells (HSCs) using a lentiviral vector. Then genetically modified cells are re-introduced into the patient, where, they differentiate into multiple cell types, some of which migrate across the blood-brain barrier into the central nervous system and express the functional enzyme. A single treatment has the potential to restore enzymatic function to stop or slow disease progression. #genetherapy #medicine #neurologicaldiseases #neurology #research #fda #innovation #health

John James OBE, CEO Sickle Cell Society, is a key speaker at our forthcoming event exploring the exciting potential of gene therapy and other advanced therapeutic medicinal products (ATMPs) on Thursday 11 July. Gene therapy addresses genetic issues by adding, removing, or altering genes. Cell therapy uses cells, like stem cells, to treat diseases or repair damaged tissues. Tissue-engineered products grow new tissues or organs outside the body for transplantation. They are all ATMPs and are giving hope to patients with a variety of conditions, including genetic diseases, blood disorders and cancer, who previously had limited or no treatment options. Future developments are also likely to transform the treatment of common conditions such as diabetes, cardiovascular disease and dementia. Book now for an essential briefing into these revolutionary treatments shaping the future of healthcare: 👉 https://bit.ly/4cnylBI #genetherapy #atmps #healthcare

🔬 Exciting News in Gene Therapy! 🔬 bluebird bio has reached a pivotal milestone with the successful completion of the first commercial cell collection for LYFGENIA™ (lovotibeglogene autotemcel), their innovative gene therapy for sickle cell disease. This marks a significant step forward following FDA approval in December 2023. #LYFGENIA™, designed to treat patients over 12 with a history of vaso-occlusive events, utilizes a one-time ex-vivo lentiviral vector gene therapy. The therapy aims to reduce disease symptoms by introducing a functional β-globin gene into the patient's own hematopoietic stem cells. This process promises to produce adult hemoglobin with anti-sickling properties, potentially minimizing the frequency of painful sickle cell crises. The first cell collection was conducted at Children's National Hospital in Washington, DC, part of a network of over 60 Qualified Treatment Centers. This network comprises centers recognized for their expertise in transplant, cell and gene therapy, and sickle cell disease. David Jacobsohn, MD, MBA, Chief of the Division of Blood and Marrow Transplantation at Children’s National, expressed his pride in leading the commercial application of this groundbreaking treatment, highlighting its promise as a "groundbreaking treatment alternative" and a "beacon of hope" for patients and families affected by sickle cell disease. As bluebird bio continues their long-term safety and efficacy follow-up study (LTF-307), the healthcare community watches eagerly, hopeful for the positive impact LYFGENIA™ might have on those it aims to help. Please visit the following link for further details: https://lnkd.in/gTkD7S7E #Healthcare #Pharmacy #GeneTherapy #SickleCellDisease #bluebirdbio #Biotech

👂🌟 Regeneron's DB-OTO Gene Therapy Shows Promise in Treating Genetic Deafness! 🔍 In a ground-breaking development, Regeneron Pharmaceuticals' investigational gene therapy, DB-OTO, has demonstrated significant efficacy in treating profound genetic deafness in children, as revealed in the Phase 1/2 CHORD trial. DB-OTO, a cell-selective adeno-associated virus (AAV) gene therapy, targets variants of the otoferlin gene responsible for congenital hearing loss. Two young participants showed promising results: one child achieved normal hearing levels within 24 weeks, while the other displayed initial improvements after six weeks. Audiometry tests indicated an average 84dB improvement in key speech frequencies for the first participant and a 19dB average improvement for the second. 💡 These findings suggest DB-OTO's potential as a transformative therapy for otoferlin-related deafness, offering hope for enhanced speech development and quality of life. Regeneron's commitment to innovative medical solutions continues to drive progress in addressing unmet medical needs. Clinical trial investigator and Columbia University’s Department of Otolaryngology – Head & Neck Surgery chair Lawrence Lustig said: “These impressive results showcase the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related deafness, and we are excited to see how this translates into an individual’s development, especially since early intervention is associated with better outcomes for speech development.” To read similar ground-breaking news, subscribe to the World Pharmaceutical Frontiers page!! #Regeneron #GeneTherapy #CHORDTrial #HearingLoss #MedicalAdvancements #worldpharmaceuticalfrontiers

Innovation in Gene Therapy for Genetic Deafness Regeneron Pharmaceuticals, Inc. has shared encouraging results from the Phase I/II CHORD trial of DB-OTO, their investigational gene therapy targeting genetic deafness due to otoferlin gene variants. The study, presented at the ASGCT annual meeting, shows substantial hearing improvements in children, with one achieving normal hearing levels within 24 weeks. This development is particularly significant as it addresses a rare genetic condition, offering hope for transformative treatment options. DB-OTO has also received critical designations such as Orphan Drug and Fast Track from regulatory bodies, emphasizing its potential impact. #GeneTherapy #HearingLoss #Regeneron #DBOTO #ASGCT24

Gene therapy is revolutionizing modern medicine, but finding a safe and effective delivery vehicle remains a critical challenge. While liposomes offer significant promise over viral-based delivery methods, they struggle with limited delivery efficiency across various cell types. A key obstacle is our incomplete understanding of the cellular structures necessary for liposome uptake, particularly in migratory versus epithelial cells. In our latest research, we've discovered the cellular structures required for efficient liposome-based gene delivery using human pluripotent stem cells. This advancement could pave the way for more effective and safer gene therapy applications in the future. #GeneTherapy #BiomedicalResearch #StemCells #Liposomes #MedicalInnovation #NIGMS #NIH #NEI Basic and Translational Research, Dept of Ophthalmology, IUSM https://lnkd.in/gvCfnAwM

#molecularbiology #primerdesign The first prime editor will soon enter the clinical phase #Chronicgranulomatousdisease (CGD: Chronic Granulomatous Disease) is a rare hereditary disorder that weakens the body's #immune system. A genetic defect in the NADPH Oxidase enzyme gene causes xenobiotic cells to not function properly in dealing with various infections, and this condition causes patients to be exposed to serious infections. 👉Pioneering PM359 gene therapy provided by Prime Medicine Company is promising news in this field. This method is the first gene editing in humans to target this debilitating disease. This innovative treatment uses a new method called "Prime editing" to precisely correct the genetic mutation of the gene responsible for CGD in the patient's own hematopoietic stem cells (HSCs). These stem cells, which are responsible for the production of blood cells, are modified outside the body (extracorporeal) and then injected into the patient. In this way, this method has the ability to provide a permanent solution and cure for this disease. 👉Receiving #Food and #Drug Organization (FDA) approval for a phase I/II clinical trial for PM359 is an important step in evaluating its safety and effectiveness in patients with CGD. The design of the trial was carried out with high accuracy and included the registration of participants in three separate groups. Initially, adults with CGD with stable disease status will enter the trial to assess the safety of this treatment. If PM359 shows positive results in this initial group, the trial will be expanded in the next phase to groups including adolescents, children and even patients with active infections or severe inflammation. This step-by-step approach, while ensuring patient safety, helps to gradually evaluate the effectiveness of PM359 in a wider population of patients with CGD. 👉The development of PM359 shows a revolutionary progress in the field of gene editing and is promising for the treatment of patients with CGD. This treatment offers a potential therapeutic approach, unlike current treatment methods that mainly focus on controlling infections. Beyond CGD, Prime Medicine is actively developing gene editing treatments for various other diseases. The success of PM359 could pave the way for a new era of gene-based therapies and provide revolutionary treatment options for many untreatable genetic diseases. Key phrase: Prime editing: A new type of gene editing method that, unlike the CRISPER/Cas9 method, enables the rewriting of short sections of DNA without the need to create double-strand breaks. More information👇 https://lnkd.in/eC23n58f