Pharmaron US Lab Services’ Post

The development of chimeric antigen receptor (CAR) T-cell therapy for hematological malignancies is a promising therapeutic advancement in the cell and gene therapies. Our latest article talks about the heightened interest in applying CAR T-cell therapy to various hematologic malignancies over the last decade. https://bit.ly/3s2rlGH #cartcelltherapy #cartcell #cartcells #celltherapy #hematology #genetherapy #pharmaceutical #pharma #drugdevelopment #clinicaltrials #drugsafety #pharmacovigilance 

RNA-based therapeutics, exemplified by ASOs, siRNAs, and miRNAs, have emerged as pivotal players in the era of precision medicine. Their ability to selectively modulate gene expression holds transformative potential across a spectrum of diseases. https://lnkd.in/gDUnu96i #drugdiscovery #drugdevelopment #rna #rnatherapeutics #rnai

Are you designing an early-phase clinical trial for your cell & gene therapy (CGT) product? Unsure of how to approach the trial design? Early phase trial designs for CGT products depend on the product's distinctive features, previous clinical experience and manufacturing logistics. Inevitably, these are significantly different (and may be more complex） than those of small molecules. It is important for sponsors to consider clinical safety issues, preclinical issues, and CMC related issues for CGT products. Read our blog that covers the key considerations when designing early-phase clinical trials for CGT products https://lnkd.in/dRzXEzcx Discuss your regulatory requirements for designing CGT clinical trials 🤝 Meet us at BIO International 2024: https://lnkd.in/gJt7Vxwv 🤝 Meet us at DIA Global 2024: https://lnkd.in/grrwWGU6 #cell #gene #therapy #clinical #trial #regulatory #BIO2024 #DIAGlobal #pharma #biologic #CMC #preclinical #safety

2024 has started with a BANG, CGT deals worth over $1.6billion, Here's 3 exciting new partnerships 👇 1. Voyager Therapeutics, Inc. has entered a licensing deal with Novartis, resulting in a potential $1.2 billion agreement. Novartis will pay $100 million upfront for access to Voyager's 𝘙𝘕𝘈-𝘣𝘢𝘴𝘦𝘥 𝘴𝘤𝘳𝘦𝘦𝘯𝘪𝘯𝘨 𝘱𝘭𝘢𝘵𝘧𝘰𝘳𝘮 and the development of a gene therapy candidate targeting Huntington's disease. Voyager's shares have surged by 35%. 2. MaxCyte, Inc. and Lion TCR have formed a strategic partnership for Lion TCR's global expansion of TCR-T cell therapy. Lion TCR will use MaxCyte, Inc.'s technology for solid tumour and viral-related disease therapies. Lion gains global rights and MaxCyte receives fees/revenue. 3. 4D Molecular Therapeutics and Arbor Biotechnologies have joined forces to co-develop AAV-delivered CRISPR/Cas-based genetic medicines for CNS diseases. The collaboration focuses on up to 𝘴𝘪𝘹 𝘱𝘳𝘰𝘥𝘶𝘤𝘵 𝘤𝘢𝘯𝘥𝘪𝘥𝘢𝘵𝘦𝘴, with a 50:50 profit share. -- 𝐓𝐡𝐞 𝐢𝐧𝐢𝐭𝐢𝐚𝐥 𝐟𝐨𝐜𝐮𝐬 is on ALS, with additional targets for diseases with high unmet needs. 4DMT contributes customised AAV vectors and a robust development engine, while Arbor provides a proprietary AAV-compatible gene editing toolbox and CNS expertise. #genetherapy #celltherapy #CGTweekly #biotech

𝐈𝐧𝐭𝐞𝐥𝐥𝐢𝐚 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐏𝐮𝐛𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧 𝐨𝐟 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐈𝐧𝐭𝐞𝐫𝐢𝐦 𝐏𝐡𝐚𝐬𝐞 1 𝐃𝐚𝐭𝐚 𝐟𝐨𝐫 𝐍𝐓𝐋𝐀-2002 𝐢𝐧 𝐇𝐞𝐫𝐞𝐝𝐢𝐭𝐚𝐫𝐲 𝐀𝐧𝐠𝐢𝐨𝐞𝐝𝐞𝐦𝐚 Intellia Therapeutics, Inc. has shared interim clinical results from the Phase 1 portion of the ongoing Phase 1/2 trial of NTLA-2002 in hereditary angioedema (HAE). NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for HAE, and the results were were published online in the New England Journal of Medicine. https://lnkd.in/eDPEHfDJ #crisprmedicine #crisprmedicinenews #crispr #geneediting #crisprtrial #raredisease

The latest announced long-term data from the ongoing phase 1 study of NTLA-2002, developed by Intellia Therapeutics, Inc., provides strong evidence that it could be a one-time, potentially functional cure for Hereditary Angioedema. On June 2, Intellia Therapeutics announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002, an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE). This rare genetic condition leads to potentially life-threatening swelling attacks. Results highlights: ✔Eight of 10 patients remain completely attack-free following the 16-week primary observation period through the latest follow-up, including patients with the most severe disease ✔Single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate, with an average follow-up of over 20 months across all patients ✔100% of patients who discontinued prophylaxis treatment after NTLA-2002 remain free of chronic prophylaxis treatment ✔Favorable safety and tolerability profile observed at all dose levels Plasma kallikrein inhibition is among the current treatment approaches to HAE. NTLA-2002 is designed to knock out the target gene kallikrein B1 (KLKB1) in hepatocytes. NTLA-2002 is administered intravenously as a single dose of Cas9 mRNA and gRNA via lipid nanoparticles. Guide RNA targeting human- and cynomolgus monkey-specific KLKB1 were identified and formulated using Intellia’s modular CRISPR/Cas9 LNP platform technology. #crispr #crisprcas9 #geneediting #genetherapy #invivo #mRNA #LNP #raredisease Resource from: Intellia Therapeutics

FDA has issued a new guidance that assists sponsors in developing Chimeric Antigen Receptor (CAR) T cell products, a form of human gene therapy. It covers recommendations on chemistry, manufacturing, control (CMC), pharmacology, toxicology, and clinical study design, with specific notes for autologous or allogeneic CAR-T cell products. Analytical comparability studies are also addressed. This guidance, while tailored for CAR-T cell therapy products, offers applicable insights for other genetically modified lymphocyte products like CAR Natural Killer cells or T cell receptor modified T cells. #CAR-T #GeneTherapy #FDAGuidance #cmc #clinicalpharmacology #fda #pharmaproconsulting To learn more use the link below: https://lnkd.in/gJZ3aGBX

What's Going On With Pfizer On Thursday? Pharma Giant's Shares Tick Lower https://ift.tt/dMV7Hjf Pfizer Inc. reported that its Phase 3 CIFFREO study on Duchenne muscular dystrophy (DMD) gene therapy did not meet its primary effectiveness goal. Despite efforts to improve motor functions in boys aged 4 to 7 using a modified human dystrophin gene, the trial showed no significant progress, affecting Pfizer's quest for new treatments as it diversifies beyond its COVID-19 portfolio. read more via Benzinga - Stock Market Quotes, Business News, Financial News, Trading Ideas, and Stock Research by Professionals https://ift.tt/9yc4Wza June 13, 2024 at 10:18AM

Research Update! Sarepta Therapeutics, Inc. today provided an update on the regulatory progress of ELEVIDYS (delandistrogene moxeparvovec-rokl), its gene therapy for DMD. FDA Approves Expanded Label for ELEVIDYS Research Update! The U.S. Food and Drug Administration (FDA) has approved an expansion of the labelled indication for ELEVIDYS to include patients aged 4 and above who have a confirmed mutation in the DMD gene. This is a significant step forward in making ELEVIDYS available to a broader range of DMD patients. "Representing many years of dedicated research, development, investment and creative energy, the expansion of the ELEVIDYS label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science." Doug Ingram, President and Chief Executive Officer, Sarepta Find out more on our website: https://lnkd.in/e-Rh3_DD

Here's a great blog by Kevin Robb on lessons learned from market-approved MSCs based on a presentation at ISCT, International Society for Cell & Gene Therapy. Stempeutics Research Private Limited and Takeda both have approved MSC products. Kevin shares tips from Pawan Gupta and Eleuterio Lombardo on unconventional manufacturing strategies, cell doses, patient selection, selecting the target disease, potency assays, and more. Read it here: https://lnkd.in/dHZwarEB #clinicaltrials, #regulatoryapproval