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Advarra's Gene Therapy Ready (GTR) network of research sites and institutions is by far the largest such network in the world. These sites are registered with the NIH Office of Science Policy and prepared by Advarra to undergo Institutional Biosafety Committee (IBC) review for cell and gene therapy research to improve safety and minimize regulatory delays to startup. Covered research includes genetic vaccines (e.g. mRNA vaccines), gene modified cellular therapies (e.g. CAR T cells), genetically engineered microorganisms, gene therapy and gene editing research. #Advarra, #ABSA, #mrnavaccines, #genetherapy, #cellandgenetherapy, #ARM, #SEBSA, #AMEXBIO, #SBA, #clinicaltrials, #clinicalresearch

Cell and gene therapy: 2024's hottest trend in pharma? 🚀 Attention pharma professionals! According to a recent survey by GlobalData, cell and gene therapy (CGT) is poised to be the dominant trend in the industry this year Here's why this matters: ◾ 18% of industry experts surveyed by GlobalData see CGT as the most impactful pharma trend this year. ◾ The global CGT market is expected to reach a whopping $80 billion by 2029, showcasing its immense potential. ◾ Oncology will remain the major focus, but exciting advancements are happening in neurodegenerative, autoimmune, and cardiovascular diseases too. What does this mean for you? ◾ If you're in pharma, pay attention to CGT! It's a rapidly evolving field with immense potential to transform healthcare. ◾ Stay ahead of the curve: Sharpen your knowledge of CGT and its potential applications in your specific field. ◾ Connect with experts and pioneers in the CGT space  ◾ Innovative CGT companies are going to be investor’s favourite. Share your thoughts on CGT's future in the comments below. Tag your colleagues and connections who might be interested in this exciting trend! https://lnkd.in/gZ6KdE2B #TuracozGroup #pharma #biotech #cgtherapy #trends #healthcareinnovation  

Gene&Cell Therapy >> FDA lifts hold on 4DMT's Fabry gene therapy: The FDA lifted an 18-month clinical hold on 4D Molecular Therapeutics’ cardiomyopathy gene therapy for Fabry disease. The biotech said Thursday it expects to resume enrollment in the Phase 1/2 INGLAXA study of 4D-310 before the end of the year. The Emeryville, CA-based company announced the hold in February 2023, a month after pausing enrollment in the trial after three patients on the experimental gene therapy experienced kidney issues. The cases were resolved within four weeks. At the time of the hold, 4DMT CEO David Kirn said the company believed the hold was “an administrative step [and] has no impact on our plans or timing for the 4D-310 program.” Jefferies analyst Kelly Shi wrote in a note on Thursday that 4DMT management indicated the initial focus will be on ex-US sites so they can “collect cardiac biopsies” and “confirm the expected improved safety of rituximab & sirolimus (R/S) regimen + amended protocol to prescreen patients for complement activation.” Several drugmakers have approved Fabry medicines, including Sanofi, Chiesi and Amicus. Sangamo, uniQure and others are developing gene therapies for the disease. Other companies like Freeline and Avrobio have scrapped efforts. Meanwhile, earlier this year, Walking Fish Therapeutics shut down months before planning to request FDA clearance for a trial in Fabry disease. Earlier this summer, Kirn said that the “primary value driver” in 4DMT’s pipeline is its ophthalmology programs, according to an AlphaSense transcript of a presentation at the Goldman Sachs healthcare conference. The company is investigating medicines for wet AMD, diabetic macular edema, X-linked retinitis pigmentosa, choroideremia, geographic atrophy and other indications. It also has an ophthalmology deal with Astellas. Beyond retinal diseases and Fabry, it’s working on candidates for cystic fibrosis, A1AT deficiency and ALS. Jefferies analysts estimate that 4D-310 could receive approval in the US in 2030 and in the EU in 2031. They predict peak worldwide sales of $172 million in 2035 if the drug is approved. #lucidquest #genetherapy #celltherapy

💡Exciting News in Pharma! Genentech and GenEdit have inked a game-changing deal worth up to $644 million for nanoparticle-based gene therapy delivery in autoimmune diseases. GenEdit aims to overcome the limitations and safety concerns of current approaches with its innovative non-viral polymer nanoparticles. These offer: -Payload flexibility -Tissue selectivity -Ability to re-dose while maintaining low immunogenicity This collaboration could potentially reshape treatment paradigms for those battling autoimmune diseases, offering hope for improved outcomes and quality of life. 🗣️ According to Roche Pharma Partnering's global head James Sabry, "Our collaboration with GenEdit holds immense promise, utilizing their cutting-edge polymer nanoparticle platform to address the needs of patients with devastating autoimmune conditions." #automationinhealthcare #pharmaceuticalresearch #Innovation #AutoimmuneDiseases #nanotechnology https://lnkd.in/eh-Vtpzf

Nanoscope Therapeutics Inc. President and CSO Samarendra Mohanty, PhD will chair the 4th Annual Gene Therapy Comparability Summit this week in Boston. In his presentation and in a session, Dr. Mohanty will offer insights into navigating process modifications and forward-thinking strategies in transitioning to commercially viable processes while ensuring comparability. Details of the presentations are: Session: Innovations in Gene Therapy Process Development to Aid Comparability Title: Fireside Chat: Navigating Process Changes and the Assurance of Comprehensive Testing to Minimize Challenges in Gene Therapy Comparability Date: Wednesday, February 14, 2024 Time: 3:30 p.m. ET Session: Delving into Analytical Challenges to Overcome Comparability Issues Title: Navigating Challenges in CMC Package Submissions Date: Thursday, February 15, 2024 Time: 11:30 a.m. ET Nanoscope remains committed to pushing the boundaries of gene therapy to elevate patient outcomes and eagerly anticipates stimulating discussions with esteemed peers at this meeting. Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases for which no cure exists. The company’s lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy. #genetherapy #ophthalmology #retina https://lnkd.in/gtQSQMBd

There was great news for rare disease patients this week as several gene therapy agreements took shape. 🏥Reimbursement agreement reached in the UK for CRISPR gene editing therapy to treat patients with transfusion-dependent beta thalassemia 🤝License agreement announced to develop genomic medicines for neurodegenerative diseases Read the full stories below and subscribe to our Sector Vector newsletter for more weekly cell and gene therapy clinical, business, and manufacturing news. 📩 https://lnkd.in/eD7ZNaU This week, Vertex Pharmaceuticals announced it entered into a reimbursement agreement with NHS England for patients eligible for its gene editing therapy to treat transfusion-dependent beta thalassemia. https://lnkd.in/eNbMYtjN Sangamo Therapeutics, Inc. announced it entered into a license agreement with Genentech to develop genomic medicines to treat certain neurodegenerative diseases using capsid delivery platforms and epigenetic regulation. https://lnkd.in/dyKGsN_n #Genetherapy #CRISPR

2024 is brimming with potential for cell and gene therapy 🧬 CGTLive has summarized the latest developments in CGT so far this year, including: 1️⃣ The FDA has approved Kite Pharma’s manufacturing change for Yescarta®, which reduces turnaround time – from leukapheresis to product release – from 16 to 14 days 2️⃣ Janssen and Legend Biotech’s Carvykti® therapy has moved forward for FDA review, potentially expanding its use in multiple myeloma 3️⃣ Guidelines for the development of CAR-T and genome editing products have been finalized by the FDA 4️⃣ Astellas and Mass General Brigham have embarked on a 5-year strategic collaboration in oncology, rare disease and cell and gene therapy to advance novel therapies toward commercialization It's encouraging that we are seeing developments across the CGT development, including a focus on manufacturing and scalability. We look forward to seeing further progress and impact through the rest of 2024. Read the full report ⬇️ https://lnkd.in/eBdrEKD9 #CellTherapy #GeneTherapy #AroundTheHelix #ManufacturingBrighterFutures #Biotechnology

🌟 Big News in Gene Therapy! PTC Therapeutics' Upstaza Receives FDA Priority Review for AADC Deficiency 🌟 PTC Therapeutics, Inc. has reached a major milestone in the quest to treat aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA has granted priority review to Upstaza, PTC Therapeutics, Inc.'s gene therapy candidate designed to address this rare and devastating genetic disorder. AADC deficiency typically manifests early in life, leading to severe disability and suffering. Upstaza offers hope as a one-time gene replacement therapy for patients aged 18 months and older with confirmed severe AADC deficiency. By delivering a functioning human DDC gene directly into the brain using a recombinant adeno-associated virus serotype 2 (AAV2), Upstaza aims to correct the underlying genetic defect and restore dopamine production crucial for neurological function. The BLA acceptance marks a significant step forward, with a target regulatory action date set for November 13, 2024. PTC Therapeutics, Inc. CEO Matthew Klein, MD, MS, FACS expressed excitement about bringing this transformative therapy to patients in the United States. Clinical trials and compassionate use programs have validated Upstaza's safety and efficacy, showing transformative neurological improvements. While common side effects include initial insomnia, irritability, and dyskinesia, the therapy's potential impact is profound. Administering Upstaza requires a stereotactic surgical procedure conducted by qualified neurosurgeons at specialized centers, ensuring precise delivery of the gene therapy. This news underscores PTC Therapeutics, Inc. commitment to advancing innovative treatments for rare diseases and offers hope to patients and families affected by AADC deficiency. Stay tuned for further updates as we await the FDA's decision. Discover more about this here: https://lnkd.in/eF4ATVXj For the latest in healthcare innovation, follow World Pharmaceutical Frontiers. 🧬💡 #PTCTherapeutics #Upstaza #GeneTherapy #RareDisease #WorldPharmaceuticalFrontiers 🌍