Anna Rose Welch’s Post

I love this for a few reasons: 1) Elegant solution to a BIG problem. Coronary artery disease still affecting millions. 2) Addresses the main pain points with medications to lower LDL - specifically medication adherence and the side effects of statins and cost of PCSK9 inhibitors. 3) Would like to see this expanded and a randomized controlled trial ultimately performed. 4) Glad to see a cardiac CT image used 😀

Super exciting early clinical data from my friends Verena Karsten & team at Verve Tx from there first in human #CRISPR based base editing study Heart-1. Using #LNP to deliver the base editing machinery that changes one base in the PCSK9 gene aiming at permanently lowering LDL-C. The 10 patients in this study all had interventions due to coronary artery disease, half were post MI and all had failed prior cholesterol lowering drugs. They were able to lower LDL-C of up to 55% in those patients. This is a breakthrough for #LNP based #invivo #gene editing

#Groundbreaking topical #gene #therapy for #rare disease! This is fourth FDA approval of a gene therapy for rare diseases since last fall. May 19, 2023 the U.S. FDA approved the first-of-its-kind topical gene treatment #Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based #gene therapy, for the treatment of wounds in patients 6 months of age and older with #dystrophic #epidermolysis #bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. “Vyjuvek is the first FDA-approved gene therapy treatment for DEB, a #rare and #serious #genetic skin disorder,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s action demonstrates the FDA’s ongoing commitment to supporting the development and evaluation of new treatments that address unmet needs for rare diseases or conditions.” #genetherapy #development #research #skindisease #raredisease #dystrophicepidermolysisbullosa https://lnkd.in/eh3wfN5j https://lnkd.in/e-guyQcB

Hemophilia B patients could have a new reason to hope. CSL Behring recently dosed its first patient in the Phase III trial of Hemgenix, a gene therapy aiming to transform the treatment of this debilitating disease. If successful, the therapy could significantly reduce the need for regular clotting factor infusions. We at RareMoon Consulting are always intrigued by advancements in the gene therapy arena. It's such advancements that inspire our work with orphan drug companies. Find out more in the linked article: https://lnkd.in/g3DHAwBH #HemophiliaB #GeneTherapy #ClinicalTrials #RareMoonConsulting

🔬 Exciting News for the Pharmaceutical Industry! 🌟 📚 The article below sheds light on the exciting advancements in cell and gene therapy, which are revolutionizing the field of medicine. With the FDA anticipating a surge in therapy submissions, it's an opportune time for Réalta Technologies to offer our expertise and support to pharmaceutical companies navigating this evolving landscape. ✨ At Réalta Technologies, we are supporting our clients by driving automation and digital solutions. Our cutting-edge technologies empower organizations to streamline processes, enhance operational efficiency, and ultimately improve patient outcomes. 💼 In my role, I am privileged to collaborate with forward-thinking companies, helping them leverage automation and digitalization to unlock their full potential. With cell and gene therapies poised to reshape the healthcare industry, Réalta Technologies is uniquely positioned to provide tailored solutions that enable our clients to adapt and thrive. #cellandgenetherapy #pharmaceuticalindustry #automationsolutions #digitalization #healthcareinnovation #RéaltaTechnologies #automation #genetherapy

🌟 𝐁𝐢𝐠 𝐍𝐞𝐰𝐬 𝐢𝐧 𝐇𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚 𝐁 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭! 🌟 🔬 Pfizer has announced a "paradigm shift" in the treatment of hemophilia B with the FDA approval of Beqvez, their groundbreaking gene therapy. This innovative treatment promises to significantly reduce the burden of regular Factor IX infusions for adults with moderate to severe hemophilia B. 👥 As we embrace this advancement, key decision-makers and healthcare systems will need to familiarize themselves with the transformative potential of gene therapies like Beqvez. However, the price tag of $3.5 million mirrors that of its predecessor, Hemgenix, posing potential challenges for widespread adoption within our healthcare systems. 💡 Beqvez delivers a healthy Factor IX gene via an adeno-associated virus vector, offering a potential long-term solution for patients who currently depend on regular infusions to manage their condition. 🏥 With the commercial rollout beginning in the US and plans for EU market approval, Pfizer is also introducing a warranty program to enhance payer confidence and improve patient access, reflecting a proactive approach to addressing cost concerns. #GeneTherapy #HemophiliaB #HealthcareInnovation #Pfizer https://lnkd.in/eXSyw_PT

Want to learn more about how adeno-associated viral vector-based gene therapy can help with debilitating and life-threatening diseases? Read this white paper co-authored by Certara expert Raj K. to learn why innovative MIDD is the key to the approval of these drugs. https://ow.ly/W2Kq50OXkS9

According to Rare Disease Advisor, “2024 will be a breakout year in addressing key challenges to developing cell and gene therapies, especially for rare disorders.”   Sadly, science continues to outpace the commercialization of innovative therapies while the pharmaceutical industry remains slow to change. As a result, patients are getting lost in the shuffle. At MPATHIK, we’re tackling these shifts head-on with empathy and collaboration.   Learn more about what’s expected in 2024 for gene therapies: https://lnkd.in/eWPSz2Jk

After Kyowa Kirin buyout, Orchard scores FDA approval for first MLD gene therapy in the US Orchard’s Lenmeldy, also known as atidarsagene autotemcel, is the first U.S.-approved gene therapy for kids with certain types of metachromatic leukodystrophy (MLD) MLD, is a rare genetic disease that affects around one in every 40,000 people in the U.S. . The condition arises from a deficiency of the enzyme known as arylsulfatase A (ARSA), which causes a buildup of fatty substances in a person’s cells, in turn dealing damage to the central and peripheral nervous systems. Orchard’s latest approval in the U.S. marks a win for Kyowa Kirin, too. Last October, the Japan-based company shelled out $387.4 million upfront to take over Orchard and get its hands on the biotech’s gene therapy, still coded at the time as OTL-200. https://lnkd.in/dNAWYUZS

Gene Therapies Are Still Hampered By Substantial Delays Between Approval And Launch Gene therapies hold promise for treating a wide range of diseases. Innovative companies such as bluebird bio and BioMarin are at the forefront of clinical development. Yet, firms face numerous challenges, in all phases, from pre-clinical through Phase 3. Furthermore, what is sometimes underappreciated is the fact that even when gene therapies obtain marketing authorization, they’re still often hampered by substantial delays between their date of approval and the time they launch. And it isn’t simply a matter of not having a payment or reimbursement system in place. Manufacturing and patient preparation delays figure prominently, too. https://lnkd.in/eWhNhEPS #development #manufacturing