Greenstone Biosciences’ Post

UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data UniQure this week released updated Phase I/II data for its investigational gene therapy AMT-130, which significantly slowed disease progression in patients with Huntington’s disease. With these promising 24-month data, uniQure also announced that the FDA has granted AMT-130 the first-ever Regenerative Medicine Advanced Therapy designation for Huntington’s disease. As of March 31, 2024, 21 patients had evaluable 24-month follow-up data. Twelve of these patients received AMT-130’s lower dose of the gene therapy, while the remaining nine were assigned to the high-dose arm. After 24 months of observation, the higher dose of the gene therapy slowed disease progression by 80% relative to the external controls. https://lnkd.in/gEjK5AeY #aspenalert #biotech #bioprocess

🔬 FDA Approves Pioneering Gene Therapies for Sickle Cell Disease 🌟 In a groundbreaking development, the U.S. FDA has approved two innovative gene therapies, #Casgevy and #Lyfgenia, for treating patients aged 12 and above with #sicklecelldisease (SCD). This marks a significant milestone in gene therapy, with Casgevy becoming the first therapy to utilize CRISPR/Cas9 genome editing technology. #SCD, an inherited blood disorder, affects approximately 100,000 individuals in the U.S. The newly approved treatments, developed through advanced gene-editing techniques, offer hope for those suffering from this debilitating disease, which often leads to severe pain, organ damage, and shortened life expectancy. Casgevy, developed by Vertex Pharmaceuticals, and Lyfgenia, by bluebird bio Inc., both utilize the patient's own modified blood stem cells. This revolutionary approach aims at increasing fetal hemoglobin levels to prevent red blood cell sickling, a key factor in SCD pathophysiology. These approvals underscore the FDA's commitment to facilitating the development of safe and effective treatments, particularly for rare diseases with significant unmet needs. As a firm at the forefront of rare disease research and advocacy, RareMoon Consulting celebrates this landmark achievement. We are committed to supporting the development and accessibility of such transformative therapies, bringing hope to patients and their families. #GeneTherapy #SickleCellDisease #RareDiseases #HealthcareInnovation #RareMoonConsulting

Day 26 of celebrating good news in cell and gene therapies: Another day, another promising indication for cell and gene therapies - researchers at St. Jude Children's Research Hospital have shown good preclinical efficacy for an AAV-based gene therapy for sialidosis! Sialidosis is a rare (we are talking multiple hundred reported cases in the whole world level rare), inherited lysosomal storage disorder that disrupts normal cellular function. In its milder form (late-onset, usually started after 20s) patients may have a relatively normal life expectancy but can experience progressive neurological decline while in its more severe form (early-onset, often in infancy), this can result in significant development issues and reduced life expectancy with many patients not surviving beyond childhood. Currently, there is no cure for sialidosis. Treatment focuses on managing symptoms and improving quality of life. While the therapy is not even human trials yet, it reflects the herculean efforts by researchers around the world to fight rare diseases. This is not only important for patients living with rare diseases, but insights gained from rare disease research frequently translate to therapies for more common conditions. Congratulations to the team on this great discovery and let's celebrate researchers and companies around the world dedicating their efforts towards various rare diseases! 🎊 https://lnkd.in/dX5BGQPP #CGT #genetherapies #rarediseases #celebrate #monthofCGTcelebrations

Today we're celebrating (a little late!) yesterday’s landmark development in medical science – the novel gene therapy, Casgevy, for sickle cell disease by Vertex Pharmaceuticals and CRISPR Therapeutics. 🔬 🎉 This pioneering therapy uses CRISPR-Cas9 gene-editing, a technology that transformed the field of genetic engineering. The roots of this innovation trace back to 2012, when Jennifer Doudna and Emmanuelle Charpentier, made a pivotal discovery. They demonstrated how CRISPR could be harnessed for gene editing, setting the stage for groundbreaking treatments like exa-cel. It took 11 years and hundreds of scientists to develop a working therapeutic for patients – an incredibly fast timeline for a technology as complex as gene therapy. 🧬 At LifeArc, we aim to remove the hurdles that keep promising gene therapies from reaching patients and make that timeline even faster, through: - a network of innovation hubs with the Medical Research Council and the UK Research and Innovation Biotechnology and Biological Sciences Research Council - a £5m Gene Therapy Innovation Fund (https://lnkd.in/eVhT9xvj) #GeneTherapy #SickleCellDisease #CRISPR

📅 December 8, 2023: A monumental day in healthcare as the FDA approves two pioneering gene therapies, Casgevy and Lyfgenia, for sickle cell disease in patients aged 12 and older. These are the first cell-based gene therapies for this condition, marking a significant milestone in medical science. ✨ Casgevy, developed by Vertex Pharmaceuticals in partnership with CRISPR Therapeutics, uses the groundbreaking CRISPR/Cas9 genome editing technology. On the other hand, Lyfgenia, created by bluebird bio, represents another advanced approach in gene therapy, using a lentiviral vector to modify cells. Both treatments showcase innovative strides in the field, offering new hope to millions affected by sickle cell disease. 🧬 Sickle cell disease has long been a challenging condition to manage, affecting millions worldwide. The introduction of these therapies opens up new possibilities for patients, offering hope where there was once uncertainty. 💰 However, there's a crucial aspect we must consider: the cost. Casgevy is priced at $2.2 million, and Lyfgenia even higher at $3.1 million. These prices raise important questions about accessibility and affordability for patients. How will these therapies be made accessible to those who need them most, especially in light of their high costs? This is a conversation that needs to be had in tandem with celebrating these advancements. 🌍 As we celebrate this breakthrough, let's acknowledge the impact this will have on healthcare, research, and most importantly, the lives of those affected by sickle cell disease. But while we commend the strides made in gene therapy and the hope it brings to patients with sickle cell disease, we must also address the challenges ahead. Ensuring equitable access to these life-changing treatments is as important as the scientific breakthrough itself. #HealthcareInnovation #GeneTherapy #SickleCellDisease #CRISPR #FDAApproval #MedicalResearch #HealthTech

✨ [RARE DISEASE DAY]   🌏 Globally more than 300 million people, most of them children, are living with one of 7000 rare diseases. Of these 7000 rare diseases, do you know that 80% are genetic diseases and nearly 95% have no #treatments?    🧬 At Genethon, more than 200 of our #scientists and #experts are dedicated from #research to clinical trials to design and develop gene therapy treatments to cure patients with rare diseases. Although gene therapy has proved its worth, winning initial victories, access to these therapeutic innovations remains an obstacle.   🔬 On the occasion of #RareDiseaseDay, we are highlighting our different #strategies to bring gene therapies to patients suffering from rare diseases such as Limb Girdle Muscular Dystrophies (LGMD), #Crigler Najjar syndrome and Duchenne muscular dystrophy. Find out more 👉 https://urlz.fr/pIDw AFM-Téléthon Rare Disease Day #genethon #RareDiseaseDay2024 #development #strategies #rarediseaseday #duchenne #LGMD #JIMR

Scientists at CMRI have pioneered successful testing of AAV gene therapies in whole human livers in the lab. This breakthrough leverages a normothermic liver perfusion system to evaluate early-stage gene therapies directly within the human liver, addressing the challenge of replicating human physiological conditions for preclinical models. The study also demonstrates potential for assessing gene therapeutics' function, effective dosing, and identifying toxic side effects, offering promising advancements in treating inherited diseases. #GeneTherapy #LiverResearch #AAVVectors #MedicalResearch #HealthTech #InheritedDiseases #PreclinicalTesting #TranslationalResearch #NormothermicPerfusion #ClinicalOutcomes https://lnkd.in/gNaiDBWv

✅FDA Gives Green Light to First Gene Therapies to Treat Patients with Sickle Cell Disease.✅ 👉 The FDA has greenlit two groundbreaking gene therapies, Casgevy and Lyfgenia, marking the first-ever cell-based gene treatments for sickle cell disease (SCD) in patients aged 12 and older. 🩸Sickle cell disease, impacting about 100,000 people in the U.S, predominantly affects African Americans, with a lesser but significant impact on Hispanic Americans. 💉 Casgevy stands out as the first FDA-approved therapy leveraging CRISPR/Cas9, a cutting-edge genome editing technology, to modify patients' hematopoietic stem cells. CRISPR/Cas9 technology enables precise DNA editing, fostering increased production of fetal hemoglobin (HbF) that prevents the sickling of red blood cells, a hallmark of SCD. 🌈 Lyfgenia utilizes a lentiviral vector for genetic modification, aiming to produce HbAT87Q, a gene-therapy derived hemoglobin, reducing the risk of sickling and blood flow obstruction. 🩸 Both therapies are crafted from the patients' own blood stem cells, ensuring compatibility and are administered through a one-time, single-dose infusion. 📊 A comprehensive long-term study will monitor the safety and effectiveness of Casgevy and Lyfgenia, providing crucial insights into their sustained impact. 🩺 In trials, Casgevy demonstrated impressive outcomes, with 93.5% of patients achieving freedom from severe vaso-occlusive crises for at least 12 consecutive months. ⚠️ Common side effects include low blood cell levels, mouth sores, and nausea. However, vigilant monitoring is essential due to potential risks, particularly hematologic malignancy with Lyfgenia. Source: https://lnkd.in/dpDtiwKy . #crisprcas9 #happening #geneediting .

CALLING ALL RARE DISEASE ADVOCATES! ONLY 1 WEEK TO GO! TELL FDA TO INCORPORATE PATIENT PERSPECTIVES ON INDIVIDUALIZED CELL & GENE THERAPIES! Personalized cell & #genetherapies, which are specifically tailored to an individual patient (or a very small group of patients) based on specific genetic markers or other unique traits, are the only hope for a cure for many rare disease patients. These individualized cell & genetherapies are also extremely expensive and difficult to make. To move this promising field forward, FDA is asking the public for information about current challenges and opportunities in the development of individualized cell & gene therapies. Specifically, FDA would like input about the following issues: - How to streamline chemistry manufacturing and controls (CMC) processes to make these therapies faster, easier, and cheaper to manufacture with consistent quality and in very small batches for individual patients. - How to best leverage non-clinicial studies in animal models, in-vitro studies, computational studies, etc. to make these therapies faster, easier, and cheaper to develop - with less risk of R&D failure. - How to effectively & efficiently design clinical studies including vital safety surveillance for these very small patient populations. - How to effectively leverage collaboration and best practices to propel this field forward. - How to appropriately apply regulatory flexibility for these therapies. RARE DISEASE ADVOCATES, MAKE YOUR VOICE HEARD! How are you thinking about the benefit-risk trade-offs for these products - and what level of regulatory flexibility should FDA apply in reviewing these products? How can patient groups, sponsors, and researchers collaborate effectively to move this field forward? How can patient-centric study designs improve clinical studies in this field and maximize enrollment and retention in the clinical studies? What are your perspectives on data from animal models, computational simulations, or tissue-culture in lieu of or in addition to some clinical data? And what can patient groups do to help prioritize investments in CMC to streamline processes and make these products faster, cheaper, and more reliable to manufacture? What else should FDA be thinking about in regulating peronalized cell & genetherapies? FDA needs to hear from you! Submit comments here: https://lnkd.in/gvA7ctrk and see our webinar for more background on cell and gene therapies.

#BreakingNews: The FDA on Friday approved a pair of #genetherapies for #sicklecelldisease, including the first treatment based on the breakthrough #CRISPR #geneediting technology. The agency approved #Lyfgenia from bluebird bio (BLUE.O), and a separate treatment called #Casgevy by partners Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN). Both the therapies were approved for people aged 12 years and older. The #Vertex/CRISPR gene therapy uses the breakthrough gene editing technology that won its inventors the Nobel Prize in 2020. Makers of both the therapies have pitched them as one-time treatments, but data on how long their effect lasts is limited. The only longer-term treatment for sickle cell disease is a bone marrow transplant. "I actually am very reticent to call them a cure. I prefer to call them a transformative therapy because patients will still have sickle cell disease on the other side of gene therapy," said Dr Sharl Azar, medical director of the Comprehensive Sickle Cell Disease Treatment Center at Massachusetts General Hospital. #Bluebirdbio's sickle cell therapy is designed to work by inserting modified genes into the body through disabled viruses to help the patient's red blood cells produce normal hemoglobin. For Vertex's therapy, patients must have stem cells harvested from their bone marrow. The cells are then sent to manufacturing facilities where they are edited using CRISPR/Cas9 technology. Once the cells are incubated, they are infused back into the patient during a month-long hospital stay. Both gene therapies can take several months and involve high-dose chemotherapy, but this has potential risks of infertility. Read the full story at Reuters 👇🏼