NecstGen’s Post

Have you ever wondered what impact science can have on our everyday lives? 📈 𝐁𝐎𝐎𝐒𝐓𝐈𝐍𝐆 𝐄𝐂𝐎𝐍𝐎𝐌𝐘 𝐂𝐫𝐞𝐚𝐭𝐢𝐧𝐠 𝐭𝐡𝐞 𝐰𝐨𝐫𝐥𝐝’𝐬 𝐥𝐚𝐫𝐠𝐞𝐬𝐭 𝐫𝐞𝐭𝐢𝐧𝐚𝐥 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 𝐜𝐨𝐦𝐩𝐚𝐧𝐲 pioneering the first clinical trials of gene therapy for the diseases choroideremia and X-linked retinal pigmentosa (spin-out company Nightstar) 𝐅𝐢𝐫𝐬𝐭 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 𝐟𝐨𝐫 𝐡𝐚𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚 development of single-dose gene therapy that restores blood-clotting and can be delivered at 1% of the cost of conventional treatment (spin-out company Freeline Therapeutics) 👩⚕️ 𝐇𝐄𝐀𝐋𝐓𝐇 & 𝐖𝐄𝐋𝐋𝐁𝐄𝐈𝐍𝐆 𝐋𝐢𝐟𝐞𝐬𝐚𝐯𝐢𝐧𝐠 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭𝐬 𝐟𝐨𝐫 𝐧𝐞𝐮𝐫𝐨𝐦𝐮𝐬𝐜𝐮𝐥𝐚𝐫 𝐝𝐢𝐬𝐞𝐚𝐬𝐞𝐬 𝐢𝐧 𝐜𝐡𝐢𝐥𝐝𝐫𝐞𝐧 approval of the first effective treatments for the genetic diseases Duchene Muscular Dystrophy and Spinal Muscular Atrophy (Spinraza) 𝐂𝐞𝐥𝐥 𝐝𝐢𝐬𝐜𝐨𝐯𝐞𝐫𝐲 𝐨𝐟𝐟𝐞𝐫𝐬 𝐩𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥 𝐧𝐞𝐰 𝐚𝐬𝐭𝐡𝐦𝐚 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 discovery of cells that secrete an inflammatory molecule, interleukin-13, which can trigger a reaction in asthma patients (clinical trial in progress) 🦀 𝐂𝐀𝐍𝐂𝐄𝐑 𝐓𝐑𝐄𝐀𝐓𝐌𝐄𝐍𝐓𝐒 𝐀𝐧𝐭𝐢-𝐜𝐚𝐧𝐜𝐞𝐫 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭𝐬 𝐚𝐜𝐫𝐨𝐬𝐬 𝐭𝐡𝐞 𝐰𝐨𝐫𝐥𝐝 development of anti-cancer monoclonal antibodies (e.g.  Ofatumumab and Obinutuzumab for the treatment of leukaemias such as Chronic Lymphocytic Leukemia and Follicular Lymphoma) 𝐓𝐫𝐢𝐚𝐥 𝐢𝐦𝐩𝐫𝐨𝐯𝐞𝐬 𝐩𝐫𝐨𝐬𝐭𝐚𝐭𝐞 𝐜𝐚𝐧𝐜𝐞𝐫 𝐩𝐚𝐭𝐢𝐞𝐧𝐭 𝐡𝐞𝐚𝐥𝐭𝐡 𝐚𝐧𝐝 𝐜𝐚𝐫𝐞 changing the way that men with early-stage prostate cancer are diagnosed and treated 🧓 𝐇𝐄𝐀𝐋𝐓𝐇𝐘 𝐀𝐆𝐈𝐍𝐆 𝐏𝐫𝐞𝐯𝐞𝐧𝐭𝐢𝐧𝐠 𝐟𝐚𝐥𝐥𝐬 𝐢𝐧 𝐨𝐥𝐝𝐞𝐫 𝐩𝐞𝐨𝐩𝐥𝐞 providing the evidence that home adaptation services (e.g. stairlifts, grab-rails, ramps) can help prevent falls 𝐈𝐝𝐞𝐧𝐭𝐢𝐟𝐲𝐢𝐧𝐠 𝐠𝐞𝐧𝐞𝐭𝐢𝐜 𝐫𝐢𝐬𝐤 𝐨𝐟 𝐀𝐥𝐳𝐡𝐞𝐢𝐦𝐞𝐫’𝐬 𝐝𝐢𝐬𝐞𝐚𝐬𝐞 identifying 75 genes associated with an increased risk of developing Alzheimer’s disease 👩💼 𝐈𝐍𝐅𝐎𝐑𝐌𝐈𝐍𝐆 𝐇𝐄𝐀𝐋𝐓𝐇 𝐏𝐎𝐋𝐈𝐂𝐘 𝐔𝐧𝐝𝐞𝐫𝐬𝐭𝐚𝐧𝐝𝐢𝐧𝐠 𝐭𝐡𝐞 𝐭𝐫𝐚𝐧𝐬𝐦𝐢𝐬𝐬𝐢𝐨𝐧 𝐚𝐧𝐝 𝐜𝐨𝐧𝐭𝐫𝐨𝐥 𝐨𝐟 𝐂𝐎𝐕𝐈𝐃-19 providing key data supporting recommendations and policies during the COVID-19 pandemic (school closure policy, social gatherings) 𝐆𝐞𝐧𝐨𝐦𝐞 𝐬𝐞𝐪𝐮𝐞𝐧𝐜𝐢𝐧𝐠 𝐭𝐨 𝐢𝐧𝐟𝐨𝐫𝐦 𝐨𝐮𝐭𝐛𝐫𝐞𝐚𝐤 𝐫𝐞𝐬𝐩𝐨𝐧𝐬𝐞 development of rapid whole genomic sequencing methods (spin-out company Oxford Nanopore Technologies) 🌏 𝐆𝐋𝐎𝐁𝐀𝐋 𝐇𝐄𝐀𝐋𝐓𝐇 𝐏𝐫𝐨𝐭𝐞𝐜𝐭𝐢𝐧𝐠 𝐡𝐮𝐦𝐚𝐧 𝐡𝐞𝐚𝐥𝐭𝐡 𝐟𝐫𝐨𝐦 𝐢𝐧𝐟𝐞𝐜𝐭𝐢𝐨𝐮𝐬 𝐝𝐢𝐬𝐞𝐚𝐬𝐞 𝐢𝐧 𝐥𝐨𝐰-𝐫𝐞𝐬𝐨𝐮𝐫𝐜𝐞 𝐬𝐞𝐭𝐭𝐢𝐧𝐠𝐬 reducing human health risk from cholera, ebola, typhoid, and childhood diarrhoea 𝐈𝐦𝐩𝐫𝐨𝐯𝐢𝐧𝐠 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 𝐨𝐟 𝐦𝐞𝐧𝐢𝐧𝐠𝐢𝐭𝐢𝐬 𝐢𝐧 𝐝𝐞𝐯𝐞𝐥𝐨𝐩𝐢𝐧𝐠 𝐜𝐨𝐮𝐧𝐭𝐫𝐢𝐞𝐬 demonstrating the efficacy of low-cost antifungal drug flucytosine Original article: https://lnkd.in/eeRYYijA Author:Buddhini Samarasinghe 304/365

🌟 Incredible News! 🌟 Texas Children's Hospital and Baylor College of Medicine have made history with NGN-401, a groundbreaking gene therapy for #Rettsyndrome . Administered via a one-time intracerebroventricular route, NGN-401 delivers the therapeutic MECP2 gene to the brain, offering new hope for Rett syndrome patients. The first recipients have shown no serious side effects, marking a promising milestone in the fight against this rare neurodevelopmental disorder. 💙 Humanized mouse models offer advantages in mimicking human physiology and pathology, making them valuable for disease studies and drug evaluations. However, existing models have limitations, prompting the need for full-length genomic DNA humanized mice that faithfully replicate human gene expression and regulation. Cyagen's HUGO-GT™ program addresses this need using TurboKnockout-Pro technology, achieving in-situ replacement of the mouse gene with the human genomic sequence. This approach offers clinically relevant models with improved accuracy. We also provide contract research services in various fields, facilitating genetic disease research and gene therapy drug development. 🐭 Learn more about our services: https://lnkd.in/gdNF9Cbf Fostering Global Collaboration in Constructing Advanced Whole Genomic DNA Humanized Mouse Models, Catalyzing the Pace of Gene Therapy Drug Development. ✔️Full Genomic Coverage of Pathogenic Gene ✔️Efficient Drug Screening ✔️Applicable to Gene Therapy Methods including ASO/CRISPR/siRNA, etc #sciencenews #bio #biotechnology #biotech #science #sciencetechnology #genetherapy #bioscience

Amyotrophic Lateral Sclerosis, aka Lou Gehrig’s disease Affects thousands in the US alone - with no real treatment. But new therapy shows hope in a Phase 3 clinical trial... 🧬 "Researchers made a significant breakthrough in ALS treatment using a new gene therapy, marking a notable slow in disease progression for a patient with an aggressive form of ALS. The patient, treated since early 2020, has maintained much of their physical and social abilities, exceeding typical life expectancy and functionality projections for their condition. This therapy targets the SOD1 gene mutation, reducing levels of a harmful protein and stabilizing the patient’s condition. The findings offer hope for future advancements in ALS treatment and broader applications of gene therapy. The therapy significantly reduces levels of the SOD1 protein, which is linked to ALS, thereby slowing disease progression. Four years post-diagnosis, the patient continues to perform daily activities independently, a rare achievement for those with aggressive ALS. The drug has been approved by the FDA and recommended by the EMA for use in patients with SOD1 gene mutations, indicating its efficacy and potential. Scientists at Umeå University report that the disease progression in a patient with a particularly aggressive form of ALS disease has slowed down considerably with the use of a new gene therapy. After four years on the medication, the patient can still climb stairs, rise from his chair, eat and speak well, and live an active and socially fulfilling life. “I consider this a breakthrough for the research we have conducted for more than 30 years. We have never before seen treatment results as effective as these,” says Peter M. Andersen, a neurologist and professor at the Department of Clinical Sciences at Umeå University. “It is now possible to considerably reduce the levels of the disease-causing SOD1 protein, and measure a clear inhibitory effect on disease progression. “When we diagnosed the patient at the neurology ward in early spring 2020, the patient’s prognosis was 1.5–2 years of survival at best. The patient has far, far exceeded expectation.” The patient is from a family in southern Sweden with a particularly aggressive form of ALS disease caused by a mutation in the SOD1 gene. The patient was a carrier of the disease gene, and after experiencing muscle weakness four years ago, the patient realized that he too was afflicted. The patient was diagnosed with early stage ALS disease. Every four weeks, the patient received the experimental treatment at a university hospital in #Copenhagen in #Denmark. At the time of diagnosis in 2020, the patient’s levels of the substance neurofilament L—a biomarker indicating breakdown of nerve cells—was very high. Now, four years later, the levels are reduced by almost 90%.” Thanks to Neuroscience News - link in the comments! #clinicaltrials #ALS #neuro #neurobiology

The world's first gene therapy #treatment for sickle cell disease has been approved by Britain's #medicines regulator, a development that may provide thousands of people in the #country suffering from the debilitating illness with relief. For patients 12 years of age and older with thalassemia and #sicklecell disease, the agency approved the treatment. Vertex Pharmaceuticals (Europe) Ltd. and CRISPR #therapeutics are the manufacturers of Casgevy. Up until now, the only permanent treatment available has been bone marrow transplants, which are incredibly difficult operations with very unpleasant side effects. "Up until now, the word 'cure' has been incompatible with sickle cell #disease or thalassemia," the statement reads. She also referred to the MHRA's approval of gene therapy as "a positive moment in history." Errors in the haemoglobin gene, which produces sickle cell disease and thalassemia, the oxygen-carrying protein in red blood cells, are the source of these disorders. A genetic mutation that causes sickle cell disease, which is more common in people with African or Caribbean ancestry, causes the cells to take on a crescent shape. This can obstruct blood flow and result in severe pain, organ damage, stroke, and other issues. Severe anaemia can result from the genetic mutation in thalassemia patients. Patients usually need #injections and medications for the rest of their lives, as well as blood transfusions every few weeks. People with Middle Eastern, Southeast Asian, and South Asian ancestry are primarily affected by thalassemia. Targeting the faulty gene in a patient's bone marrow stem cells allows the body to produce haemoglobin that functions as intended. This is how the novel medication Casgevy works. Prior to using stem cells from the patient's bone marrow and genetic editing techniques in a lab to correct the gene, #patients undergo a course of chemotherapy. After that, the patient receives a permanent #treatment when the cells are reinfused. At least two hospital stays are required for patients: one for the collection of stem cells and another for the delivery of the modified cells. The U.S. Food and Drug Administration is presently reviewing casgevy and is anticipated to make a decision early in the upcoming month, after which it will review additional sickle cell gene therapies. According to LaBelle, University of Chicago administrators are "already moving forward to build not only the clinical #infrastructure to get these patients this #treatment , but also the reimbursement infrastructure." www.tycoonstory.com #GeneTherapy #BloodDisorders #UKFirst #MedicalBreakthrough #CRISPR #SickleCellDisease #BetaThalassemia #HopeForPatients #LifechangingTreatment #TransformativeMedicine #HealthcareInnovation #CuringDisease #SavingLives #ChangingTheFuture

I participated in a panel discussion of an event on "Frontiers in Cell and Gene Therapy for Cardiovascular Diseases and Cancer - From human mini-organs for drug discovery, clinical trials to commercialisation" organised by the DukeNUS medical school, GCT Institute, Mass General Brigham, ASTAR and the National Heart Centre Singapore. Gene therapy was featured in TIME magazine as the future of medicine in the Jan 1999 issue. In the last 2 decades, cell and gene therapy has come a long way. There are 33 approved cell and gene products approved by the US FDA. In 2024, up to 21 cell therapy launches and as many as 31 gene therapy launches are expected, possibly including the first-ever approval of adoptive cell therapy for solid tumors and the first U.S. approval of an allogeneic T-cell therapy. Cell and gene therapies (CGT) hold the promise of treating or even curing some cancers and rare diseases. But these emerging therapies are also among the world’s most expensive treatments—with some priced between $400,000 and $2 million per dose, sometimes more. Still, a potential wave of new gene therapies is building on the horizon. The CGT market is expected to nearly quadruple over the next three years—from $5.3 billion in 2022 to $19.9 billion by 2027. We discussed the issue of allocation of funds to such therapies which will only benefit a small number of the population with rare diseases. It is however enlightening to note that such cell & gene treatments are now developed for the more common cancers and even chronic diseases like heart failures, atherosclerotic heart and autoimmune diseases. Host derived pluripotent stem cells are now successfully directed to healthy and disease-modelled tissues and organoids which allows for gene targets identification and testing of drugs on disease model that recapitulate faithfully the disease phenotypes. Immune therapeutic cells such as CAR-T have now reached the 5th generation that will not only carry more specific TCR to target the diseased cells but also carry co-stimulatory molecules and cytokines to activate, prolong the half-life of such therapeutic cells and moderate other immune functions such as checkpoint inhibitors and JAK inhibitors to enhance efficacy and safety. The greatest challenge is the cost of therapy. The fist 8 gene products are priced above US$1 mill for one treatment. The highest price is US$3.5m for the treatment of Hemophilia B. We discuss affordability and accessibility issue and recommend a change in the reimbursement model. I suggested that the benefit of such treatment should bring quality added life years and a one-off high price is not the ideal reimbursement model. We should deal with the financing aka freehold property where the financial payout should be on a longer term basis much like mortgage for real estate assets. There could also be risk sharing between payors and the CGT pharma companies in that no payment would be made if there is no positive outcome.

Exciting news from in the world of gene therapy! Physicians have collected stem cells from a young boy with sickle cell disease in preparation for the first commercial infusion of Bluebird bio's newly approved gene therapy, Lyfgenia. This personalized treatment involves modifying the patient's own stem cells to produce a corrected form of the defective protein responsible for sickle cell disease. The process, from cell collection to infusion, takes 70-105 days. Bluebird bio is in talks to develop outcomes-based payment models with payers and with aid from the federal government to make Lyfgenia accessible through Medicare. While there is progress being made from access side, challenges remain in the complex and lengthy treatment process, including multiple cell collections, preconditioning chemotherapy, potential side effects like infertility, and complex logistics. However, this development represents a significant milestone in sickle cell treatment, offering hope for a more effective and potentially curative approach. Stay tuned for further advancements in gene therapies like Lyfgenia, revolutionizing the landscape of genetic disease treatment. #GeneTherapy #SickleCellDisease #HealthcareInnovation https://lnkd.in/e6B73-BA

🔬 Exploring FDA-Approved Cell and Gene Therapies 🔬 Did you know that the field of cell and gene therapies is rapidly expanding, with numerous innovative treatments gaining regulatory approval? 🌟 Today, I'm sharing a snapshot of the current landscape, highlighting the number of approved therapies in each category. 🧫 Cell Therapies: With 17 FDA-approved treatments, these therapies harness the power of living cells to replace or repair damaged tissues. From blood cell transplants derived from the umbilical cord and placenta to pancreatic islet cell transplants, they offer hope for a wide range of conditions. 🧬 Gene Therapies: There are 9 FDA-approved treatments focusing on modifying genetic material to treat or prevent diseases, offering promising avenues for medical innovation. From correcting genetic defects in hemophilia to treating specific types of Duchenne muscular dystrophy, these therapies address various genetic disorders with immense promise. 🧫 🧬 Gene modified cell therapy (ex-vivo gene therapy): There are 11 FDA-approved cell and gene therapies. Combining the strengths of both cell and gene therapies, treatments range from CAR-T therapy targeting specific cancers, such as large B-cell lymphoma, to addressing genetic defects in sickle cell disease. These innovative approaches hold significant promise for a diverse range of conditions. As we celebrate the growing number of approved therapies, it's essential to recognise the impact these advancements are having on patient care and the broader healthcare landscape. With ongoing research and development, we can expect to see continued growth in the field, with even more innovative therapies on the horizon. 📊 Check out the accompanying Venn diagram for a visual representation of the approved cell and gene therapies. Stay tuned for more posts. Feel free to share your thoughts and questions in the comments below. #CellTherapy #GeneTherapy #PrecisionMedicine #HealthcareInnovation #FutureOfMedicine

#GeneTherapies #Sicklecelldisease #CRISPRCas9 #Casgevy #Lyfgenia #FDA #PriorityReview #OrphanDrug, #FastTrack #RegenerativeMedicine #AdvancedTherapy Sickle cell disease is a group of inherited disorders caused by mutations in HBB, which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 and 400,000 neonates globally each year, the majority in sub-Saharan Africa. Haemoglobin molecules that include mutant sickle β-globin subunits can polymerize; erythrocytes that contain mostly haemoglobin polymers assume a sickled form and are prone to haemolysis Common acute complications are acute pain events, acute chest syndrome and stroke; chronic complications can damage all organs Today treatments are Hydroxycarbamide, blood transfusions and haematopoietic stem cell transplantation taht can reduce the severity of the disease Yesterday the FDA Approves First #GeneTherapies to Treat Patients with Sickle Cell Disease #Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology. Patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology #Lyfgenia is a cell-based gene therapy. Lyfgenia uses a lentiviral vector for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events. With Lyfgenia, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease. Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, a patients’ own stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy). Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness. The Vertex-CRISPR therapy has a U.S. list price of $2.2 million, while bluebird's is $3.1 million. Both therapies, pitched as one-time treatments, will be available in early 2024. Both the Casgevy and Lyfgenia applications received #PriorityReview, #OrphanDrug, #FastTrack and #RegenerativeMedicine #AdvancedTherapy designations More information: https://lnkd.in/gBE9jEFp Rocket Program, Biochem123, KONEXIO BIOTECH

A gene therapy cure for sickle cell disease could soon be available, but it’s not clear whether insurance companies will balk at the cost and deny coverage. The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy. Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African Americans and, while less prevalent, also affects Hispanic Americans. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape. “Arguably” the gene therapy does not appear as cost-effective as the grinding medical care that sickle cell patients now receive— Gene therapy applied just once to a sickle cell patient costs as much as $2.8 million, compared to an estimated average cost of about $1.2 million for the lifelong waves of hospitalizations, blood transfusions, and in some cases bone marrow transplants that now constitute standard care. https://lnkd.in/g_M4K5Uc

#GeneTherapies #Sicklecelldisease #CRISPRCas9 #Casgevy #Lyfgenia #FDA #PriorityReview #OrphanDrug, #FastTrack #RegenerativeMedicine #AdvancedTherapy Yesterday, the U.S. Food and Drug Administration approved two milestone treatments, #Casgevy and #Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy Both the Casgevy and Lyfgenia applications received #PriorityReview, #OrphanDrug, #FastTrack and #RegenerativeMedicine #AdvancedTherapy designations