Kapadi’s Post

Adaptimmune’s Tecelra Becomes First FDA-Approved Engineered Cell Therapy for Solid Tumors This approval is monumental milestone for both the cell and gene therapy space and for this rare but potentially fatal disease, metastatic synovial sarcoma. Potentially life-threatening cancers such as synovial sarcoma continue to have a devastating impact, especially for those that have failed standard treatments. Cell and gene therapeutic developers have struggled in demonstrating similar efficacy in solid tumors as seen in hematologic malignancies due to many factors, primarily the immune-hostile tumor microenvironment. The approval of this state-of-the-art immunotherapy technology provides a critical new option for a patient population in need and demonstrates that cell and gene therapies do in fact have a place at the table for solid tumor malignancies.  At Kapadi, a cell and gene focused CRO, we are privileged to continue this fight for innovative, efficacious therapies in the solid tumor space. https://lnkd.in/efCXzQgW

📢 FDA Approves First Gene Therapy to Treat Adults with Metastatic Synovial Sarcoma (Adaptimmune) ➤ Accelerated approval based on multicenter, open-label trial including 44 patients with inoperable and metastatic synovial sarcoma who had received prior systemic therapy and whose tumor expressed the MAGE-A4 tumor antigen. ➤ ORR was 43.2% and the median DOR was six months ➤ The FDA granted Adaptimmune’s Tecelra Orphan Drug, Regenerative Medicine Advanced Therapy and Priority Review designations for this indication. This application was reviewed using a coordinated, cross-agency approach, including CBER, the FDA’s Oncology CoE and the CDRH ➤ Tecelra is also the first FDA-approved T cell receptor (TCR) gene therapy. The product is an autologous T cell immunotherapy composed of a patient’s own T cells. T cells in Tecelra are modified to express a TCR that targets MAGE-A4, an antigen (substance that normally triggers your immune system) expressed by cancer cells in synovial sarcoma. The product is administered as a single intravenous dose #oncology #biotech #biotechnology #regulatoryaffairs #pharma #drugdevelopment #oncologytrials #oncologyresearch #cancer #oncology #regulatoryintelligence #regulatoryprecedent https://lnkd.in/eB2uAMT7

📢 Exciting News in Cancer Treatment😀 !The FDA has approved afamitresgene autoleucel (afami-cel), the first gene therapy for adults with metastatic or unresectable synovial sarcoma. Developed by Adaptimmune, this groundbreaking T cell receptor therapy targets the MAGE-A4 antigen and has shown significant promise in clinical trials. 🔬 FDA Approval Date: August 2024 Clinical Trial Results: 39% overall response rate with a median response duration of 12 months. Significance: First engineered T cell therapy approved for a solid tumor. I assure that this approval brings new hope to patients with advanced synovial sarcoma, offering a potentially life-extending treatment option. I would like to thank for all investigators, study coordinators, CRAs, etc who were involved in all clinicaltrials to commit on getting approval! 🤞 #CancerTreatment #GeneTherapy #FDAApproval #SynovialSarcoma #Adaptimmune #Biotech #CNR #CNRResearch #ClinicalTrials #씨엔알리서치 #임상시험 #유전자치료제 #유럽임상시험 #글로벌CRO https://lnkd.in/gD45dMPM

2023 was a landmark year in CGT and the pace isn't slowing down as we move into Q2... At the Alliance for Regenerative Medicine's (ARM) annual Cell & Gene State of the Industry Briefing in January, it was announced that there were seven therapeutic FDA approvals in 2023, with a further 17 anticipated across the US and EU in 2024. The approvals covered therapies for various conditions from cancer to rare genetic disorders, including Bristol Myers Squibb's Breyanzi® for relapsed or refractory chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia, Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy® for sickle cell disease and beta thalassemia, and Iovance Biotherapeutics’ Amtagvi® for unresectable or metastatic melanoma. BioSpace looks at how the recent approvals fit into the bigger picture of clinical success and patient impact, and what's on the horizon for the near future ⬇️ https://lnkd.in/gbVV9ffV #ManufacturingBrighterFutures #CellTherapy #GeneTherapy #PatientAccess #Biotechnology

I'm thrilled to share our latest research publication titled "Smart Accumulating Dual-Targeting Lipid Envelopes Equipping Oncolytic Adenovirus for Enhancing Cancer Gene Therapeutic Efficacy." In this study, we address a major challenge in cancer gene therapy—enhancing the systemic delivery and therapeutic efficacy of oncolytic adenoviruses (oAd). Our team developed a tumor microenvironment-triggered lipid envelope equipped with both pH-sensitive and EGFR-targeting capabilities to encapsulate the oAd. This innovative approach showed promising results in targeting and accumulating in EGFR-positive breast cancer cells while reducing off-target accumulation, especially in the liver. Key highlights from our findings include: -Higher transduction efficiency in acidic, EGFR-positive environments in vitro. -Enhanced intratumoral accumulation and reduced hepatic accumulation in vivo. -Potential to overcome conventional barriers to effective cancer gene therapy. This breakthrough demonstrates a significant step forward in using gene therapy to combat EGFR-positive cancers safely and effectively. I’m incredibly proud of this work and excited to continue advancing cancer therapies with these novel delivery systems. Feel free to check out the full paper here: https://lnkd.in/gJHFb-8q #CancerResearch #GeneTherapy #OncolyticVirus #Nanomedicine #TargetedTherapies

The FDA approved Tecelra (afamitresgene autoleucel), a gene therapy indicated for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen. Tecelra is an engineered T-cell receptor (TCR) therapy. It's the first TCR therapy approved for cancer. The product is an autologous T-cell immunotherapy composed of a patient’s own T cells. T cells in Tecelra are modified to express a TCR that targets MAGE-A4, an antigen expressed by cancer cells in synovial sarcoma. The product is administered as a single intravenous dose. 

Personalized medicine is soon shaping up to be the future of healthcare. Gene 🧬 therapy is a branch of personalized medicine and it is revolutionizing the way we treat genetic disorders. Here is a comprehensive list of FDA-approved gene therapies Ex-vivo gene therapies, where cells/tissues are modified outside the body of the patient and then reinfused: 1️⃣ CAR T-cell therapies ✅ Kymriah by Novartis to treat acute lymphoblastic leukemia in pediatric and young adults and adults with relapsed or refractory B-cell lymphoma. ✅ Yescarta and Tecartus by Kite Pharma for adults with relapsed or refractory B-cell lymphoma and mantle cell lymphoma, respectively. ✅ Breyanzi by Juno Therapeutics, Inc. for adults with relapsed or refractory B-cell lymphoma. ✅ Abecma by bluebird bio and Bristol Myers Squibb for adult patients with relapsed or refractory multiple myeloma. 2️⃣ Hematopioetic Stem Cell Gene Therapies ✅ Zynteglo by bluebird bio for all patients with beta-thalassemia. ✅ Libmeldy by Orchard Therapeutics - U.S. for children with early-onset metachromatic leukodystrophy. In-vivo therapies, where cells are modified in the body. All currently approved in-vivo therapies use viral vectors for gene delivery. 1️⃣ Luxturna developed by Spark Therapeutics, Inc. for treating RPE65 mutation associated retinal dystrophy. 2️⃣ Zolgensma by Novartis for spinal muscular dystrophy (SMA). 3️⃣ Hemgenix by CSL Behring B.V. for Hemophilia B. As a biomaterials and formulation expert, I cannot wait to see how lipid nanoparticles will shape the next generation of in-vivo gene therapies, and be added onto this list. #gene #genetherapy #personalized #medicine #healthcare

This week on Care for Rare, we're delving into treatment approaches for curing Fanconi Anemia. Currently, hematopoietic stem cell transplantation (HSCT) is the primary option, but challenges like donor availability and secondary cancers hinder its effectiveness. However, promising alternatives such as gene therapy and CRISPR-based gene editing offer precise corrections, bringing hope for targeted treatments. Excitingly, recent advancements in mRNA technology open new avenues for FA treatment, offering potential as replacement or cell therapy. Yet, challenges like repeated administration and precise tissue targeting persist. As May comes to an end, let's continue the momentum of Fanconi Anemia Awareness Month with optimism for further advancements in finding a cure and advocating for those affected by FA. #FanconiAnemiaAwareness #HopeForACure #CareForRare 📁Subscribe to get the weekly free newsletter on Metabolic Diseases, Disease Disruptors, and Care for Rare delivered directly to your inbox at https://lnkd.in/gBN7dVsD ✍️Care For Rare Team Malini Gupta, Ph.D., Author Jyothi Inampudi PhD, & Anusha Jayaraman, PhD, Managing Editors Ananya Rakshit, Ph.D., & Himanshi Agarwal, PhD, Social Media Managers

In the dynamic field of oncology, recent advancements in gene therapy offer a promising solution for hepatocellular carcinoma (HCC). The limitations of conventional treatments have spurred the exploration of innovative approaches, with adeno-associated viruses (AAVs) emerging as potential game-changers. AAVs, known for their efficient gene delivery, show promise for targeted HCC gene therapy. Despite the absence of specific clinical trials in this area, AAVs' proven safety and efficacy in diverse diseases underscore their potential in liver cancer treatment. https://lnkd.in/g8NHfZT2 #genetherapy #oncology #aav

Remarkable work by my colleagues Alexander Renner, Dominik Schmiedel and team from Fraunhofer IZI was recently published at the journal Gene Therapy. The publication discusses the development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer in non dividing cells. By achieving high transduction rates of freshly isolated immune cells before expansion, the approach enables a streamlined and cost-effective automated production of off-the-shelf cell therapeutics in less than 1 day, requiring fewer viral particles and less manufacturing steps. This holds the potential to significantly reduce the time and resources required for producing e.g. NK cell therapeutics. https://lnkd.in/dfWN6miH