MedTech Strategist’s Post

Cell and gene therapies (CGT), also known as “Advanced Therapy Medicinal Products” (ATMP), address diseases by directly repairing genetic defects within the body or introducing tissues or cells with new functions. These innovative therapies can correct or cure a wide range of conditions. The development and manufacture of these medicines are complex and novel, and they have only recently started gaining traction in the commercial sphere over the past decade. What sets them apart is their remarkable potential to offer permanent cures rather than temporary symptom reduction. Get an overview in the latest article by VTU experts Cornelia Haas and Birgit Krenn, which appeared in the magazine “botenstoff” of the Human.technology Styria GmbH (February 2024 issue, Precision Medicine special edition). #VTU #CellandGeneTherapy

#CellTherapy and #GeneTherapy are complex modalities and every program is truly unique. Does your commercial strategy take into account nuances specific to your drug?

We are honored to share that Andelyn has been selected by Armatus Bio, Inc. to accelerate manufacturing of their gene therapy treatment for Charcot-Marie-Tooth Type 1A (CMT1A), a rare genetic neurological disease associated with independence-limiting disability and risk of fatal complications that has no approved therapies today.   Charcot-Marie-Tooth disease type 1A (CMT1A) is a type of inherited neurological disorder that affects the peripheral nerves. People with this disease experience weakness and wasting (atrophy) of the muscles of the lower legs beginning in adolescence; later they can also have hand weakness and sensory loss. CMT1A is caused by having an extra copy (a duplication) of the PMP22 gene (NIH).   We are truly grateful to play an important role in directly transforming patient lives. https://lnkd.in/e3WBkyYU   #GeneTherapy #RareDisease #CDMO #aav

What IF... together, we could overcome the challenges of LNP encapsulation at high mRNA concentrations? High mRNA levels are essential for therapies like protein replacement and gene therapy. At Innoforce, our advanced LNP technology delivers outstanding encapsulation efficiency, even at elevated mRNA concentrations. 💡For example, with mRNA concentrations of 2.5 mg/mL, we achieve over 97% encapsulation efficiency, keeping empty capsids below 6%. Explore how Innoforce can help you meet the challenges across a wide range of mRNA concentrations. ☞https://lnkd.in/gQRhKU-U #InnoforcePharma #PlasmidDNA #RNA #LNP #ViralVectors #CellTherapy

Cell and gene therapies are shaping the future of medicine. However, it is crucial to recognize that the CGT landscape is dynamic and therapeutic success requires careful consideration. Here is our infographic that covers the latest CGT trends: https://ow.ly/Mi2V50QkC4p.

Gene therapy has the potential to transform the lives of individuals with haemophilia, but should it be accessible to everyone? During #EHA2024, I attended an engaging debate where experts presented compelling arguments both in favour of and against expanding access to these innovative treatments. Here are some of my key takeaways from both sides of the debate, illustrating the many factors that must be weighed when considering these cutting-edge therapies for patients. Please reach out if you would like to learn more about our insights on gene therapy in rare blood disorders.

We are quickly approaching an inflection point with respect to using cell therapies to treat #autoimmune disease. While we have increasing confidence that cell therapies will play a pivotal role in the treatment of autoimmune disorders, sponsors will have to overcome significant barriers to deliver on this promise: reducing manufacturing costs, expanding treatment infrastructure, improving the risk/benefit profile, to name just a few. Curious to learn more? Check out our blog post and presentation at #ISCT2024 by Health Advances Cell and Gene Therapy practice co-leader Ned Wydysh PhD, and Jamie Pierson of Parexel. Read our blog here: https://bit.ly/3yZzelC ISCT, International Society for Cell & Gene Therapy #CGT #CellandGene

Gene therapies represent an exciting approach to treatment across the length and breadth of the IMD field. In this new article, Lee et al consider what it takes to develop a new therapy in an inherited disorder of neurotransmission using their own work in succinic semialdehyde dehydrogenase deficiency to illustrate the move from bench to bedside. Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency Henry Lee, Itay Tokatly Latzer, Mariarita Bertoldi, Guangping Gao, Phillip Pearl, Mustafa Sahin, and Alexander Rotenberg https://lnkd.in/ekt74-vD

🧬 Exciting advancements in gene therapy are revolutionizing treatment options for patients with factor deficiency, offering a potential one-time solution to prevent bleeding episodes. The current therapies on the market provide hope for a future with minimal treatment burden. More innovations are on the horizon, shaping a promising landscape for improved patient care. 🌟 Learn more and earn FREE #CME in the complete CME Snack with Maya Bloomberg, MSN, APRN and Guy Young, MD! #GeneTherapy #MedicalInnovations #HealthcareAdvancements

Launching revolutionary cell and gene therapies (CGT) from lab to patient is a complex but rewarding challenge. 🧬💡 Understanding the BLA filing process and navigating clinical trials are pivotal steps in this quest. 🔍 Dive into the key considerations for BLA filing, ensuring your groundbreaking CGT advancements are set for success. It explains the complexities and breakthroughs in cell and gene therapy development. Ready to learn more? Click the link here ➡️ https://lnkd.in/dc-dqA-Q Have questions about navigating clinical trials? 🤔 LaSalle Group is here to help! #CellGeneTherapy #ClinicalTrials #BiotechInnovation #HealthcareProgress