YEASEN BIOTECHNOLOGY ’s Post

Cell and gene therapies (CGT) are one of the hottest sectors in the pharmaceutical industry. Quite a few companies in the field are making remarkable advancements toward potentially curative treatments for challenging diseases using groundbreaking science. Despite a steady stream of regulatory approvals and promising growth prospects, the high manufacturing costs associated with these therapies often render them inaccessible to the majority of patients. To address this issue, companies are actively striving to streamline and optimise the highly intricate and labour-intensive process of cell and gene therapy manufacturing. Let’s explore further at https://lnkd.in/gtqtgRa5 #asiamedtechs #medtechs #biospectrumasia #healthcare #cell #gene

According to GlobalData Plc, 19 gene therapies and gene-modified cell therapies have been marketed in the US. Of those 19 treatments, Casgevy is one of seven gene therapies and gene-modified cell therapies to have launched on the US market with an average price per unit that is above $1m. With Casgevy and Lyfgenia now being among some of the most expensive treatments on the market, questions have arisen regarding how these large price tags may create barriers to access for patients in a disease space that already sees limited treatment options. Read the full article below on Pharmaceutical-Technology: #globaldata #pharmaceuticaltechnology #raredisease #CRISPR #cellbased #genetherapy #sicklecelldisease #pharmaceuticalnews #analystcomment https://lnkd.in/e5NRhqg6

As the true potential of cell and gene therapies comes to light, companies looking to break into this sector must find ways to overcome their associated development and commercialisation challenges. At this year's Cell and Gene Therapy Summit organised by Charles River Laboratories, experts Franz Gerner, Nina Kotsopoulou and Richard Fagan discussed the importance of potency assays in the process. #CGT #cellandgenetherapy #potencyassays #biopharma #biotherapeutics #CGTmanufacturing #CGTdevelopment

Sangamo Therapeutics and Pfizer announced positive results from the Phase 3 AFFINE trial for giroctocogene fitelparvovec, a gene therapy for hemophilia A. The therapy met its primary goal of reducing annualized bleeding rates (ABR) compared to routine Factor VIII prophylaxis. Key findings include: - A significant reduction in mean total ABR post-infusion (1.24 vs. 4.73). - 84% of participants maintained FVIII activity >5% at 15 months post-infusion. - The mean treated ABR showed a 98.3% reduction from pre-infusion to post-infusion. The therapy was generally well tolerated, with serious adverse events reported in 20% of participants, most of which were treatment-related but resolved with clinical management. Sangamo stands to earn up to $220 million in milestone payments and royalties from Pfizer if the therapy is approved and commercialized. The full dataset analysis is ongoing, with plans to present additional data at future medical meetings. With Pfizer's approval of Beqvez in Hemophilia B as well as Roctavian's slow uptake in Hemophilia A, Pfizer is well positioned to become a leader in the gene therapy space for this indication! https://lnkd.in/euRyVyR8

𝑪𝒂𝒏 𝒂 𝑫𝒓𝒖𝒈 𝒃𝒆 𝒂𝒑𝒑𝒓𝒐𝒗𝒆𝒅 𝒊𝒇 𝒕𝒉𝒆 𝒑𝒓𝒊𝒎𝒂𝒓𝒚 𝒆𝒏𝒅𝒑𝒐𝒊𝒏𝒕 𝒊𝒔 𝒏𝒐𝒕 𝒔𝒕𝒂𝒕𝒊𝒔𝒕𝒊𝒄𝒂𝒍𝒍𝒚 𝑺𝒊𝒈𝒏𝒊𝒇𝒊𝒄𝒂𝒏𝒕? In late June, the FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy | FDA despite the pivotal trial failing to meet its Primary Efficacy Endpoint. According to the FDA's review documents, some internal reviewers, including the Statistical Review, opposed the approval due to insufficient evidence. Nonetheless, Dr. Peter Marks, head of the FDA CBER division, overruled these reviewers to approve this gene therapy. In fact, the statistical reviewer noted: “These results do not suggest there is substantial evidence to support the effectiveness of SRP-9001 for the expanded indication to all DMD patients and do not support the conversion of accelerated to traditional approval.” While this is a rare exception rather than the rule, and have raised controversy this also high-lights the complexity of Drug Development and Regulatory decision making - which under certain circumstances can take an intriguing turn! What do you guys thinks about this? https://lnkd.in/ezxmbSTi

What`s in a word - peer reviewed article supporting scientific discussion on Gene Therapy definition I’m thrilled to share that our recent publication is now out! Jointly with BioNTec, Curevac and Cencora, we've taken a deep dive into the evolving discussion around gene therapy definition. One of the key points we highlight is that approved mRNA medicines, like the COVID-19 vaccines, don’t modify the human genome. However, the current classification, shaped before mRNA vaccines existed, may lead to confusion and impact public trust in these groundbreaking therapies. This paper offers a science-based perspective on how updated definitions can help streamline regulations and, ultimately, foster public acceptance of these innovative medicines. I’m excited about the potential impact this discussion could have on the future of mRNA technology! Currently presented at the GPRAS conference in Brussels. 📖 Check it out here: https://lnkd.in/dR_8kN2W Thanks to everyone who contributed to this project—I’m proud to be part of such an important conversation. #GeneTherapy #mRNA #Innovation #PublicHealth #Pharma

Haemophilia B Breakthrough: European Medicines Agency Recommends Conditional Approval for Durveqtix Gene Therapy The European Medicines Agency has recommended conditional marketing authorization for Durveqtix in the European Union to treat severe and moderately severe haemophilia B in adults without factor IX inhibitors and no detectable antibodies to variant adeno-associated virus serotype Rh74. Developed by Pfizer Europe MA EEIG, this gene therapy is administered as a single infusion to enable the body to produce factor IX, reducing bleeding episodes. The most common side effect was increased liver enzyme levels. Supported by European Medicines Agency's PRIority MEdicines scheme, Durveqtix's approval marks a major advancement in haemophilia B treatment, pending European Commission approval for European Union-wide marketing. For more details please click the link! https://lnkd.in/gTNgNDfy #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

🌟 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 🌍

Thoughts on this? >> Fabry disease gene therapy 4D-310 improves heart health after 1,2... - Fabry Disease News >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #healthcare #biotech #pharma #competitivemarketing

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Insights, Clinical Trials, Treatment Drugs, and Companies 2024: DelveInsight has released its latest report titled “AAV Vectors in Gene Therapy Pipeline Insight 2024″ offering extensive insights into over 70 companies and more than 235 pipeline drugs within the AAV vectors gene therapy landscape. This comprehensive report includes detailed profiles … Continue reading → #PharmaceuticalsBiotech