3D Executive Search Partners’ Post

+++ Pipeline #News +++ Fast Track Designation for Heart Failure Gene Therapy Asklepios BioPharmaceutical, Inc. (AskBio)'s gene therapy program for congestive heart failure has been granted U.S. FDA Fast Track Designation, emphasizing the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure. Learn more: https://lnkd.in/ekY-krjA #TeamBayer

Precision's Anshul Mangal weighs in on what to expect in 2024 regarding biopharma manufacturing and supply-related issues in this Scrip Asks Part 3 series. "Cell and gene therapies will continue to dominate the innovative clinical pipeline with the largest number of treatments in clinical trials (589 gene therapies and 568 cell therapies). The recent historic landmark of the first CRISPR technology gaining regulatory approval signals confidence in this approach. Additionally, as the adoption of radioligand therapy increases, it continues to show profound potential. One of the most promising aspects of radioligand therapy lies in the potential to create unique combinations of radioactive atoms and targeting molecules tailored to specific tumor types." Read the article here (subscription required): https://hubs.ly/Q02jhS1j0 #precisionmedicine #biopharma #clinicaltrials

#Webinar now available on demand! How to apply quality by design principles to cell and gene therapy release testing In this webinar Audrey Chang (Executive Director, WuXi Advanced Therapies) will discuss how to apply quality by design principles to cell and #genetherapy release testing and how to develop and use #QCassays. Watch this webinar to learn about: ·       Concerns surrounding adventitious agent and product characterization that must be addressed with cell and #genetherapyproducts ·       Why manufacturing and testing of cell and gene therapy products should not be regarded as a series of discrete events, but rather as a continuum of interdependent activities ·       Developing and using QC assays that can facilitate both product and process understanding and control

We survey verified gene therapy outsourcers to inform drug developers and CDMOs alike so they can make the most of their partnerships. This experiential data is from real #biopharma professionals who have worked on gene therapy projects and will help readers gain a better understanding of the current state of outsourced gene therapy manufacturing: https://lnkd.in/eNdveDtK AGC Biologics Charles River Laboratories FUJIFILM Diosynth Biotechnologies Novartis Global Biotech Cooperations (CDMO) Resilience Center for Breakthrough Medicines #genetherapy #pharmaceuticalmanufacturing #marketresearch

Precision's Anshul Mangal weighs in on what to expect in 2024 regarding biopharma manufacturing and supply-related issues in this Scrip Asks Part 3 series. "Cell and gene therapies will continue to dominate the innovative clinical pipeline with the largest number of treatments in clinical trials (589 gene therapies and 568 cell therapies). The recent historic landmark of the first CRISPR technology gaining regulatory approval signals confidence in this approach. Additionally, as the adoption of radioligand therapy increases, it continues to show profound potential. One of the most promising aspects of radioligand therapy lies in the potential to create unique combinations of radioactive atoms and targeting molecules tailored to specific tumor types." Read the article here (subscription required): https://hubs.ly/Q02jkmfX0 #precisionmedicine #biopharma #clinicaltrials

Check out the July issue of Pharmaceutical Technology to explore recent work conducted to advance the manufacturing and development process for emerging therapies, particularly gene therapy.  Read our issue here: https://lnkd.in/eVMHS8mm

New promising option in #HF for the next future

📢 Exciting News in Haemophilia A Treatment! 📢 Pfizer has announced top-line results from their pivotal phase 3 study of an investigational haemophilia A gene therapy, giroctocogene fitelparvovec, developed with Sangamo Therapeutics, Inc. This condition affects about 25 in every 100,000 male births globally, leading to low factor VIII levels. In the AFFINE study, the therapy showed superiority in reducing the annualised bleeding rate over routine treatment from week 12 to 15 months post-infusion. Impressively, 84% of patients maintained factor VIII activity above 5%, with most at least 15%. The therapy was generally well tolerated. 🌍🔬 This marks a major advance in gene therapy, potentially transforming the lives of those with haemophilia A and reducing treatment burdens. The fast-track and orphan drug designations from the FDA and EU add further optimism. 🌟🩺 #HaemophiliaA #GeneTherapy #Pfizer #InnovativeHealthcare #MedicalResearch

**AAV GENE THERAPY FOR PARKINSON'S** You may or may not remember my post about the Doudna Lab publishing non-AAV delivery & uptake of CRISPR RNP complexes by simply injecting the RNP straight into rat brains. I said something about it being one way of targeting delivery without viral vectors, but it was an early first step. Well, the good folks at Asklepios BioPharmaceutical, Inc. (AskBio) just hit a Phase 1b trial milestone (tolerance in patients) by directly injecting their AAV vector directly into patient brains (the red section in the diagram). Zero adverse events across all patients, too. Huge news for gene therapy for Parkinson's Disease, and for my people at AskBio! https://lnkd.in/gJ8PWbuS Press release: https://lnkd.in/gCf4V5SM #cgt #aav #parkinsonsdisease #askbio #crispr

Thoughts on this? >> Indian President Droupadi Murmu launches first homegrown gene therapy for cancer - WION >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #healthcare #biotech #pharma #competitivemarketing