Synexa Life Sciences’ Post

Very interesting article entitled “Regulatory landscape and challenges in CAR-T cell therapy development in the US, EU, Japan, and India“ in the August 2024 edition of the European Journal of Pharmaceutics and Biophamaceutics (EJPB), the journal of APV, porposed by our member Louise Rosenmayr-Templeton. The article discusses the requirements and considerations for preclinical studies, clinical trial design, manufacturing standards, safety evaluation, and post-marketing surveillance for CAR-T cell therapy in each of these regulatory regions. It proposes strategies to overcome current development and regulatory hurdles and improve alignment in regulatory requirements to facilitate the global development of these complex therapies. As APV member you can use your EJPB online account to read the article free of costs. #pharmaceutics #APV #EJPB #CARTcell

One of the most painful medical issues we see are degenerative joint conditions. There's typically very little therapy that can be leveraged to relieve patient suffering. The power of cell therapy is hoping to change this. DiscGenics have been working on an allogenic cell therapy for patients experiencing lumbar degenerative disc disease and have been granted approval for a phase 3 study for a single-dose of live, manufactured progenitor cells derived from donated adult human tissue. Unlike autologous cell therapies, there is no need for patient cells to be harvested and economies of scale can be leveraged for a single donor cell line. The phase 2 studies resulted in significant improvement in pain, function, quality of life, and disc volume, suggesting a regenerative effect from the therapy. https://lnkd.in/gFE5i32c

AZ is betting more on cell therapy as it acquires Gracell. This will be interesting to watch as they go through the regulatory review process. More positive momentum to end 2023. With the Gracell FastCAR process, the Novartis T-Charge process, and others in development, is there more reason to develop an expansionless process of your own? https://lnkd.in/eRrG2YQm

The ISPE Guide: Advanced Therapy Medicinal Products – Allogeneic Cell Therapy and the ISPE Guide: Advanced Therapy Medicinal Products – Recombinant AAV Comparability and Lifecyle Management were published earlier this year. These guides add to ISPE’s library of knowledge in the growing and ever-evolving advanced therapy medicinal products (ATMPs) field. Learn more about these two new guides: https://hubs.la/Q02FF8cj0 #ISPE #ATMPs #Allogenic #raav

Gene&Cell Therapy >> FDA offers more clarity on safety testing for cell therapies: The FDA is providing sponsors of cell therapies with a new draft guidance document on the kinds of safety testing they should perform. The 14-page guidance, published late last month, provides sponsors with clarity on how to carry out cell safety testing when submitting an investigational new drug application or a biologics application. The guidance lays out how allogeneic cell therapies do carry some risk, including contamination, or genome changes that can potentially lead to cells developing into tumors. The FDA says sponsors should base safety testing on the potential the cells have to expand, the reagents used to expand the cells and the number of patients capable of being treated. Patrick Hanley Patrick Hanley, chief and director of cell therapy at Children’s National Hospital, told Endpoints News in an interview that the guidance is a sign the FDA is listening to cell therapy sponsors who have been asking the agency for more clarity in this area. He said focusing on allogeneic cell therapies, which involve using cells from a donor to treat the patient, is particularly helpful. He said there wasn’t previously much clarity on the kind of testing that should be done based on the number patients being treated with the therapy. “That’s what this guidance tries to address is what testing needs to be acquired based on the specific properties and treatment potential of the therapy,” he said. Hanley added that it would be helpful if the FDA laid out benchmarks for testing based on the the specific number of patients being treated with a cell therapy, but acknowledged that would be difficult given that each therapy is different. Comments on the guidance are due July 29. The guidance lays out considerations for sponsors using highly expanded cells, as well as cells with limited potential to expand. It also includes a table with a description of types of cell therapies and their corresponding testing recommendations. #lucidquest #genetherapy #celltherapy

Pfizer's termination of a Phase III study on Sickle Cell therapy highlights the difficulty of study recruitment, even for life-changing studies such as this one. To better understand sponsor company approaches regarding "Participant Compensation In Clinical Trials", DIA has assembled a consortium of industry partners, sponsors, and others. The goal is to eliminate financial hurdles that may dissuade participants due to financial reasons. Join us in our efforts to ensure that everyone, regardless of their financial situation, can participate in clinical trials. Please reach out if you are interested in joining our cause. #clinicaltrials #healthcare

🧬 𝐎𝐛𝐣𝐞𝐜𝐭𝐢𝐯𝐞𝐥𝐲 𝐞𝐯𝐚𝐥𝐮𝐚𝐭𝐢𝐧𝐠 𝐢𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐯𝐞 𝐜𝐞𝐥𝐥 𝐚𝐧𝐝 𝐂𝐀𝐑 𝐓 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 Kristin Yarema, Ph.D. - the president and CEO of Poseida Therapeutics, Inc. discussed the importance of objectively evaluating innovative cell therapies and CAR-T treatments in oncology. ⚆ The real-world effectiveness of innovative therapies is frequently limited due to infrastructural constraints such as manufacturing or timing ⚆ 'Intent to Treat (ITT)' analysis vs 'As Treated' analysis – by including all patients originally intended for treatment, regardless of whether they receive it or not, allows for a better reflection of the value and risk/benefit ratio of the innovative therapies and overall response rates (ORR) ⚆ The allogeneic CAR-T therapies, when viewed through the lens of ITT analysis, may offer advantages over autologous CAR-T with potentially more competitive pricing and availability 𝘞𝘩𝘢𝘵 𝘰𝘵𝘩𝘦𝘳 𝘧𝘢𝘤𝘵𝘰𝘳𝘴 𝘴𝘩𝘰𝘶𝘭𝘥 𝘸𝘦 𝘢𝘤𝘤𝘰𝘶𝘯𝘵 𝘧𝘰𝘳 𝘸𝘩𝘦𝘯 𝘢𝘯𝘢𝘭𝘺𝘴𝘪𝘯𝘨 𝘵𝘩𝘦 𝘰𝘶𝘵𝘤𝘰𝘮𝘦𝘴 𝘰𝘧 𝘵𝘩𝘦𝘴𝘦 𝘵𝘩𝘦𝘳𝘢𝘱𝘪𝘦𝘴? Full article: https://lnkd.in/d4rvFb-h #pharma #cart #celltherapy #innovation #business #biotech #womeninscience #drjojo

Final FDA guidance on CAR-T and its derivative therapies. This follows up recent FDA recommendation to have a label on CAR-T therapy products. This has stirred the cell therapy community. Enclosed a link to the latest news of FDA final industry guidance for CAR-T developers. Noteworthy are their guidance that extends to related therapies such as NK-T and TCR-based modified patient cells, and FDA recognition of differences between autologous and allogenic therapies. Please comment. https://lnkd.in/e52qzvc3

"Fortunately, in our view the field is approaching the tipping point where allogeneic cell therapy will come into its own. When that happens, those players with the right approach, the right technology and the right focus will quickly be recognized and rewarded." #DahliaConsulting #BiopharmaceuticalIndustry #CommercialLifeSciences #ManagementConsultant #LifeSciencesBusinessStrategy #GlobalPharma #GlobalBioTech #SupplychainOperations #CommercialOperations #CGTcommercialization #Manufacturing #QualityAssurance #OperationalExcellence #FinancialAnalysis #OpModelDesign #LifeScience #BioTechnology #Bioprocessing #Biosimilars #BioPharmaIndustry #MedicalResearch #CDMO #GMP #GMPbatch #GMPmanufacturing #GenomeEditing #GeneTherapy #CellTherapy #Car_T #mRNA #cGMP #ClinicalGradeManufacturing #ClinicalGradeProduction #BiologicsLicenseApplication #BLA #Pharmaceuticals #RegenerativeMedicine #CellAndGene #Allogeneic #Oncology #PoseidaTherapeutics #BispecificAntibodies #CellChoice #CRISPR #TALENs

Exosomes are a novel modality to deliver medicines to avoid allergic responses and adverse events. For the past 5+ years, hundreds of millions of $$ is poured into exosome-based therapeutics. Currently, 100 trials are running involving exosomes. It is studied as a safer alternative to cell therapy. But is the field moving too quickly? Experts are divided, and regulatory bodies are cautious. If holds true, using exosome to deliver drugs has a value in taking therapeutics forward despite existing knowledge gaps to fulfill many unmet needs for diseases such as stroke and ALS.