Annie Jullien Pannelay’s Post

FDA Grants Accelerated Approval for #Tecelra, the First Gene Therapy to Treat Metastatic Synovial Sarcoma! The FDA has approved #Tecelra (afamitresgene autoleucel) on August 2, 2024, a groundbreaking gene therapy for adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy. This marks a significant advancement in cancer treatment, providing new hope for patients with limited options. About Synovial Sarcoma: Synovial sarcoma is a rare cancer that develops in soft tissues, often in the extremities, and affects around 1,000 people in the U.S. annually, primarily adult males in their 30s or younger. Standard treatments include surgery, radiotherapy, and chemotherapy, but options are limited for advanced stages. Innovative Treatment: Tecelra leverages advanced genetic engineering to reprogram the patient’s own T cells to recognize and attack cancer cells, offering a targeted and effective therapy. #AcceleratedApproval : This expedited process underscores Tecelra's potential to address an unmet medical need, allowing patients faster access to this life-saving therapy. Tecelra is also the first FDA-approved T cell receptor (TCR) gene therapy. #ClinicalEvidence: Clinical trials showed a 43.2% overall response rate with a median duration of response of six months. This demonstrates Tecelra's efficacy and safety for patients with inoperable and metastatic synovial sarcoma. #AdverseReactions : Common side effects include nausea, vomiting, fatigue, infections, and cytokine release syndrome (CRS), among others. A Boxed Warning highlights the risk of CRS, requiring careful patient monitoring. Read more : [FDA Approves First Gene Therapy to Treat Adults with Metastatic Synovial Sarcoma] (https://lnkd.in/dUxh36AY) #GeneTherapy #CancerTreatment #FDAApproval #AcceleratedApproval #MedicalInnovation #Oncology #Healthcare #PharmaceuticalNews #PharmaUpdates #Tecelra #FDA

Interested in Gene Therapy? Check out our review article, "Gene Therapy for Cardiac Diseases: Methods, Challenges, and Future Directions," recently published in Cardiovascular Research. In this comprehensive review, we explore the latest advancements in molecular approaches—ranging from gene replacement to prime editing—and the various vectors used in cardiac gene therapy. We delve into critical strategies to improve genetic constructs, including promoter engineering, miRNA-regulated genetic cassettes, and systems for conditional expression of siRNAs/mRNAs. Additionally, we address the key factors that could facilitate the clinical translation of cardiac gene therapy, emphasizing research that suggests that even a limited percentage of transduced cardiomyocytes may be enough to halt or reverse the progression of certain cardiac genetic diseases. Our focus throughout the review is the progress of gene therapies for inherited arrhythmia syndromes and genetic cardiomyopathies, conditions for which few effective treatment options currently exist. These life-threatening disorders demand urgent therapeutic innovations. A heartfelt thanks to my mentor, Silvia Giuliana Priori, for her guidance, and to my friends and colleagues, Matteo Gianeselli, MD and Rossana Bongianino, who co-authored the manuscript. https://lnkd.in/dy6-bkDP

#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

https://lnkd.in/gn4rkAma Excited to see/share the news that the Metachromatic Leukodystrophy community now has an approved gene therapy! continued effort across a broad group of leaders delivered this therapy and will now be dedicated to maintain/increase access for patients and families in the US. This therapy adds to the growing list of approved agents available to transform the lives of patients around the world. While I know the Therapeutic Development industry is not able to focus on all of the problems within our healthcare systems, delivering approved options, especially in rare diseases, is a critical step. The work will continue and requires all of us to think about how innovations in science, regulatory and payer policy can continue to open the door for transformaitonal therapies....

Starting off the week with an FDA approval! Orchard Therapeutics' one-time gene therapy Lenmeldy becomes the first FDA-approved treatment option for children with metachromatic leukodystrophy (MLD).  MLD is caused by an arylsulfatase A (ARSA) enzyme deficiency, which leads to a buildup of sulfatides that can cause damage to the central and peripheral nervous system. Hematopoietic (blood) stem cells from the patients are modified to produce the ARSA enzyme to effectively halt disease progression. This is a milestone for the treatment of rare diseases and offers new hope to patients and families affected by this debilitating disease. #genetherapy #raredisease https://lnkd.in/epcpJtrT

Parkinson’s disease continues to pose significant challenges as a neurodegenerative disorder, necessitating novel therapeutic strategies. Gene therapy has emerged as a promising contender, with AAV2-GDNF (AB-1005) at the forefront. Presented at the 2024 American Academy of Neurology conference by Asklepios BioPharmaceutical, Inc. (AskBio), the Phase Ib trial data of AAV2-GDNF provides valuable insights into its safety and initial effectiveness in individuals with mild to moderate Parkinson's Disease. Explore the potential of gene therapies in parkinson’s disease treatment @ https://lnkd.in/gkwg_eVY #ParkinsonsDisease #PD #NeurodegenerativeDisorder #GeneTherapy #AAV2GDNF #AB1005 #NeurologyConference #PhaseIbTrial #Safety #Efficacy #AsklepiosBioPharmaceutical

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

🌟 Very good news for a rare central nervous system disease: The US FDA has given the green light to Lenmeldy™ (atidarsagene autotemcel), a first-of-its-kind gene therapy for early-onset metachromatic leukodystrophy (MLD) 💉💰. This one-time treatment is designed to halt or slow the progression of this life-altering neurological disorder in which myelin in the brain and spinal cord is destroyed (much like in Multiple Sclerosis) 🧬👶. #GeneTherapy #FDAApproval #MLDTreatment #InnovativeMedicine #HealthcareBreakthrough Source: https://lnkd.in/eDcZpEjR

August has seen some great successes for the cell and gene therapy space, with significant milestones achieved across various diseases, showcasing the potential of innovative treatments to address critical unmet medical needs. Ocugen OCU400 gene therapy for retinitis pigmentosa (RP) received FDA approval for an Expanded Access Program, providing more patients access to this potential one-time treatment ahead of full approval. Sangamo Therapeutics, Inc. partnered with Genentech to develop genomic medicines for neurodegenerative diseases, using advanced zinc finger repressors and AAV capsid technology, with potential milestone payments of up to $1.9 billion. IN8bio received FDA guidance for advancing INB-100, an allogeneic gamma-delta T-cell therapy, to a registrational Phase 2 trial for acute myeloid leukemia (AML). Early results from Phase 1 trials show promising relapse-free survival in patients, with plans to initiate the Phase 2 trial next year. 4D Molecular Therapeutics resumed its Phase 1/2 trial for 4D-310, a gene therapy for Fabry disease, after the FDA lifted its clinical hold. The therapy continues to show promising cardiac improvements with no new significant adverse events. TG Therapeutics, Inc. received FDA clearance to initiate a Phase 1 trial for azercabtagene zapreleucel (azer-cel), an allogeneic CAR-T therapy targeting progressive multiple sclerosis (MS), marking a significant step forward in using cell therapy for autoimmune diseases. These advancements underscore the rapid progress and potential impact of cell and gene therapies in transforming patient care. #CellTherapy #GeneTherapy #CGT

Exciting news from the Myology 2024 conference! The international gene therapy trial for Duchenne Muscular Dystrophy (DMD) using GNT0004 has unveiled promising findings. Led by Professor @Francesco Muntoni, the trial combines phases I to III with a dose escalation stage, followed by a pivotal phase. Enrolling ambulant boys aged 6 to 10 with DMD, the trial has received approval from French and UK authorities. Five patients have been administered GNT0004, demonstrating good tolerability and immunological response. Initial efficacy results for the patient receiving the higher dose (3×10^13 vg/kg) after one year showed clinical improvement, including an inflection in the North Star Ambulatory Assessment score. Other functional assessments also displayed positive trends. The safety and efficacy data have enabled Genethon, the trial’s sponsor, to prepare the pivotal European phase with the European Medicines Agency (EMA). GNT0004 is an AAV8 gene therapy that targets key tissues like skeletal and cardiac muscles through a single intravenous injection. DMD is a rare genetic disease that affects muscle function, and its absence leads to muscle degeneration and complications such as cardiomyopathy and loss of mobility. The trial’s progress represents a significant step towards potential therapeutic options for DMD.