BioSpace’s Post

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

🧬 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

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.

𝐓𝐡𝐞 𝐅𝐃𝐀 𝐡𝐚𝐬 𝐠𝐫𝐚𝐧𝐭𝐞𝐝 𝐟𝐚𝐬𝐭 𝐭𝐫𝐚𝐜𝐤 𝐝𝐞𝐬𝐢𝐠𝐧𝐚𝐭𝐢𝐨𝐧𝐬 𝐭𝐨 𝐭𝐰𝐨 𝐨𝐟 𝐂𝐚𝐫𝐢𝐛𝐨𝐮'𝐬 𝐜𝐥𝐢𝐧𝐢𝐜𝐚𝐥-𝐬𝐭𝐚𝐠𝐞 𝐜𝐚𝐧𝐝𝐢𝐝𝐚𝐭𝐞𝐬 Caribou Biosciences announced yesterday that the FDA has granted Fast Track designations to CB-010 for refractory systemic lupus erythematosus and to CB-012 for relapsed or refractory acute myeloid leukaemia. CB-010 and CB-012 are allogeneic CAR-T cell therapy candidates developed using Caribou's proprietary chRDNA genome-editing platform. The FDA's Fast Track process is designed to facilitate the development and speed up the review of certain drugs to treat serious conditions and fill unmet medical needs. Read the full update here: https://lnkd.in/earVjK8r #crisprmedicinenews #crisprmedicine #crispr #genomeediting #geneediting #crisprtrial

Between 2017 and 2024, nine CAR-T products reached commercialization and this number is continuing to climb. Of these, six are U.S. FDA-approved CAR-T cell therapies, each of which has received approvals in other major healthcare markets as well. The six U.S. FDA approved products include: Kymriah (tisagenlecleucel) Yescarta (axicabtagene ciloleucel) Tecartus (brexucabtagene autoleucel) Breyanzi (lisocabtagene maraleucel) Abecma (idecabtagene vicleucel) Carvykti (ciltacabtagene autoleucel) In addition, two CAR-T therapies have received approval from the Chinese National Medical Products Administration, Relma-cel and Yuanruida, and one CAR-T cell therapy has received approval from the Indian Central Drugs Standard Control Organisation, NexCAR19. Currently, all of the nine marketed CAR-T cell therapy products and nearly three-quarters (75%) of the ongoing clinical trials utilize an autologous treatment approach. Thus, the next frontier for CAR-T cell therapies will be the development of allogeneic CAR-T cell therapies. Solid tumor applications also attracting unprecedented investment. This billion dollar CAR-T cell therapy market would not have been possible without the remarkable efficacy of the early CAR-T therapies in treating several types of blood cancers. Ranging from small start-ups to billion-dollar companies, CAR-T companies are now proliferating in all major healthcare markets worldwide. To learn more about this rapidly evolving market, view the "Global CAR-T Cell Therapy Market – Market Size, Forecasts, Trials & Trends, 2024": https://lnkd.in/eCkW5rn For the next 48 hours only, you can claim this global strategic report for $840 off (20% off). To claim your discount, enter the code "CART20" into the Coupon Box during checkout. #immunotherapy #celltherapy #advancedtherapy #regenerativemedicine

🚨 𝗙𝗗𝗔 𝗖𝗹𝗲𝗮𝗿𝘀 𝗕𝗹𝘂𝗲𝗥𝗼𝗰𝗸 𝗧𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰𝘀' 𝗜𝗡𝗗 𝗳𝗼𝗿 𝗢𝗽𝗖𝗧-𝟬𝟬𝟭 🚨 BlueRock Therapeutics has received FDA clearance for OpCT-001, its investigational iPSC-derived cell therapy targeting primary photoreceptor diseases like retinitis pigmentosa. This marks a major step toward restoring vision for patients suffering from these conditions. “We believe that OpCT-001 has the potential to restore vision in people living with primary photoreceptor diseases,” said Amit Rakhit, MD, MBA, Chief Development and Medical Officer at BlueRock Therapeutics. 𝗟𝗲𝗮𝗿𝗻 𝗺𝗼𝗿𝗲 𝗮𝗯𝗼𝘂𝘁 𝘁𝗵𝗶𝘀 𝗮𝗱𝘃𝗮𝗻𝗰𝗲𝗺𝗲𝗻𝘁: https://lnkd.in/dsw-p-D3 #Ophthalmology #CellTherapy #VisionRestoration #BlueRockTherapeutics #FDA #ClinicalTrials

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

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

**The Future of Cell Therapy: Spotlight on Mesoblast's Advances** 🚀 Cell therapy stands at the forefront of revolutionary treatments, with Mesoblast leading the charge with remestemcel-L for SR-aGVHD. Following a critical FDA meeting, Mesoblast is gearing up to meet regulatory standards with new potency assay data, a move that underscores the rigor and innovation in cell therapy [oai_citation:1,Key Outcomes From FDA Type A Meeting and Mesoblast Next](https://lnkd.in/dyQJSabn) [oai_citation:2,Key Outcomes From FDA Type A Meeting and Mesoblast Next Steps to Achieve RYONCIL Approval](https://lnkd.in/dCZsmxAe). Their proposed trial for adults, in collaboration with the Blood and Marrow Clinical Trials Network (BM CTN), highlights the collaborative effort driving the sector towards new horizons [oai_citation:3,Key Outcomes From FDA Type A Meeting and Mesoblast Next](https://lnkd.in/dyQJSabn). Mesoblast's journey exemplifies the promise of cell therapy to transform patient care. As we embrace the future, let's champion the innovations shaping a new era in medicine. #CellTherapy #Mesoblast #Innovation #HealthcareFuture

And right behind Abecma, Carvykti CAR T cell therapy is also now approved for earlier use in myeloma https://lnkd.in/eYQXrAR3