Joris van Arensbergen’s Post

Are you developing a cell or gene therapy? Make sure you start planning early for your long-term follow-up too! To learn more about the scientific and operational considerations to optimize your #LTFU, check out this white paper developed by me and Delphine Saragoussi, MD, MScPH #RWE #CGT #LTFU https://lnkd.in/eT7UNcWv

Excitement abounds in the field of advanced medicinal therapies, including cell and gene treatments. Yet, it's important to acknowledge and navigate the challenges associated with these groundbreaking innovations. The spotlight ahead is on overcoming limitations, from safety and immunogenicity concerns to targeting intricacies, dosing challenges, and regulatory barriers. The article, "Realizing New Miracles: Gene Therapy Trends in 2024 and Beyond" in the December 2023 issue of Clinical Researcher explores four areas of focus for the year ahead > https://bit.ly/3tRxfgY #ACRP #ClinicalResearcher #ClinicalTrials #Trends #GeneTherapy

Headed to ASGCT this week? Stop by Booth #2235 to learn about our extensive commercial experience and regulatory track record to help advance your gene therapy to patients with more certainty. #ASGCT2024

Adeno-associated virus (AAV) particles are an emerging class of biotherapeutic molecule. They are relatively small, approximately 20 to 25 nm in size, but can carry a nucleotide payload of approximately 4,500 bases. They are nonpathogenic and exist in nature as different serotypes with some serotypes having selectivity to target specific organs in the human body. They are therefore extremely useful as vectors for targeted delivery of cell and gene therapies. However, as with all biotherapeutic molecules, product- or process-related impurities are inevitable and must be characterized. Aggregation is one of the most likely critical quality attributes and is best measured using size exclusion chromatography. Read more: https://lnkd.in/dDAisASm

Navigating the complexities of tangential flow filtration for viral vector-based gene therapies just got easier. Join our upcoming webinar to dive into the differences between hollow fibers and flat sheets, understand AAV stability in TFF systems, and compare TFF steps in the downstream process. Register now >> https://lnkd.in/gpphMqNZ

In 2023 alone, 11 cell and gene therapies (CAGTs) have been approved for 16 indications in the US, but the successful development of CAGTs relies on critical decisions made during the early clinical development phases. In this compelling white paper, our experts delve into the key decisions required in the development of CAGTs, and explore their profound impact on subsequent stages of the therapy’s life cycle. Download now. https://bit.ly/4aLuLAh #CAGT #clinicaldevelopment

Vector shedding represents an important safety risk in the clinical administration of viral vector-based gene therapies.   With state-of-the-art instrumentation and automation to support your vector shedding studies, at #ProtaGene we collaborate closely with clients 🤝 in the design and transfer of studies from inception through validation. Find out more in our Bioanalysis Spotlight. ⬇️ #biopharma #celltherapy #genetherapy

We are thrilled to share positive 24-month follow-up results from our ongoing Phase I/II trial of HORA-PDE6b gene therapy for patients with Retinitis Pigmentosa (RP) caused by bi-allelic mutations in the PDE6b gene.   Our presentation at ARVO 2024 highlighted clinically meaningful benefits in visual functions and a robust safety profile for HORA-PDE6b, reaffirming its potential as a promising treatment option for PDE6b RP patients.   We are now preparing to discuss these encouraging Phase I/II results with health authorities in the US and Europe, aiming to define the optimal path towards making HORA-PDE6b available to patients in need.   For more details and to access our press release, visit: https://lnkd.in/eG9MaH58   #ARVO2024 #RetinitisPigmentosa #GeneTherapy #Coavetx

Navigating the complexities of tangential flow filtration for viral vector-based gene therapies just got easier. Join our upcoming webinar to dive into the differences between hollow fibers and flat sheets, understand AAV stability in TFF systems, and compare TFF steps in the downstream process. Register now >> https://lnkd.in/gitqi4NG

From streamlining your data collection to enhancing patient retention, it's important that you consider various ways to operationalize your gene therapy long-term follow-up clinical trial. Download this insight brief to find out more: https://bit.ly/3SzpwN2 #CAGT #GeneTherapy #biotech