Children's Tumor Foundation’s Post

Rhenman & Partners’ portfolio manager, Kaspar Hållsten, is reporting back from the US where he is attending Piper Sandlers 35th Annual Healthcare Conference. Naturally, a lot of focus has been on Glucagon-like peptide-1 therapies (GLP-1) and assessing potential benefits and challenges across various medical technology areas. The level of innovation, particularly in the development of cancer screening and monitoring techniques, as well as interventional therapies and the importance of gaining regulatory approvals for these innovative approaches have also been in focus during the sessions. Marketing | Capital at risk. | For professional investors. #healthcare #healthcareinnovation

We need to advance CGT manufacturing to make widespread patient access a reality... Through our LightSpeed Early Access Program (LEAP), we've worked with multiple partners, including CTMC, to gather technical feasibility data that demonstrates IRO®'s ability to provide superior biological performance, minimize batch failures, increase throughput, and scale the impact of life-changing therapies. Here are Jason Bock's, Founder and CEO of CTMC, key insights from his direct experience using the platform: 🧫 IRO® delivered higher CAR yields and more efficient transduction (~69% vs ~45%) versus the control. 🧫 Process establishment on IRO® took <5 weeks from kick-off, and met all target release criteria by day 6 across two independent sites. 🧫 Vector copy number remained below the FDA-recommended <5 per transduced cell, meaning IRO® maintained safety and consistency. Read more about IRO®'s technical feasibility data ➡️ https://lnkd.in/eUt_rYfh Interested to see how IRO® could help scale the impact of your therapies? Book a demo today ➡️ https://lnkd.in/eXEXEGZZ #IRO #TheNewStandard #AutomateBetterBiology #AccelerateProductDevelopment #ScaleYourImpact CTMC / A joint venture between Resilience MD Anderson Cancer Center

Our CSO Martin Gosling was recently interviewed by Labiotech for an article discussing how new treatments for #cysticfibrosis (CF) are boosting life expectancy. Just three decades ago, the average life expectancy of people with CF was around 30 years of age. This has now improved to 50, with some patients living into their 80s. Recent advancements in CF research have played a key role in contributing to this. One section of the article discusses how Enterprise Therapeutic’s EDT001 has the potential to meet the needs of people with CF. In preclinical studies, ETD001 boosted the clearance of mucus for longer than 12 hours after a single dose, and Phase 1 studies showed a pharmacokinetic profile consistent with long lung retention. Phase 2 trials are due to begin this month. ETD001 has the potential to treat people with cystic fibrosis not currently eligible or receiving benefit from CFTR modulators, and further improve lung function when administered in combination with CFTR modulators. Read more about new treatments for people with #CF here: https://rb.gy/1jdi5x

😩 #Clinicalpathology labs: struggling to bring out the full potential of #molecularprofiling? Attend our panel discussion on May 16, 1:30 p.m. ET, in which clinicians from leading organizations will present real-world case studies of how they overcame these and other challenges to improve #precisionmedicine-based diagnostics ⬇️

If you're at the meeting, stop by and see my friend, the CEO of Panakeia (Pahini Pandya) and the team at Booth E40 to learn more about their game changing platform in spatial biomarkers for oncology

✴️ Are you working to get advanced therapies to patients in need?✴️ Then, you need to tune in live to 'Advanced therapies - what's next?' on June 4, a live discussion by Cytiva about how to tackle common challenges that come up along the way, including navigating the clinical pipeline, accessing talent, and getting ready for prime time. 📢 During the broadcast, master of ceremonies Dodi Axelson, Vice President of Communications, will bring your questions to Dr. Nathaniel Wang, CEO and Co-Founder of Replicate Bioscience, Inc., and Emmanuel Abate, President of Genomic Medicine, Cytiva. ✨ Don’t miss this opportunity to get expert insight and the answers you need to overcome key obstacles on the journey to market.✨ 👉 Register at https://buff.ly/3QXwN9l #therapy #advancedtherapies #genomic #medicine #clinical #pipeline #patient

What an inspiring #PSI2024 conference! I would never have expected 100 attendees at the Innovation in Dose Escalation trials session - wow! I kicked the session off with methodology to guide dose escalation for modern therapies, illustrating how to model both short- and long-term tolerability under time-varying dose, variable-length regimens and potentially multiple treatment cycles in two case studies. Some of the key takeaways from my talk (submitted together with Sebastian Weber): * Traditional dose escalation safety models were not designed for targeted therapies where efficacy is not due to toxicity (i.e., in contrast to classical cytotoxic therapies) * Long-term tolerability modeling that waits for the full binary data to be available makes trials very slow, even more so under dropout (and considering dropout is more important over longer timescales!) * Time-to-event modeling (and modeling efficacy separately) can deal with this and give a more relevant answer, i.e., on the safety of the therapy. A big thank you to Sebastian Weber, Yunnan Xu and Hans-Jochen Weber for collaborating on the long-term tolerability case study, as well as Andrew Lewandowski, Andrew Stein, Shunguang Wang and Nina Orwitz for helpful discussions on the short-term step-up dosing case study. Many thanks also to the speakers following in the session, with Anaïs Andrillon discussing how to integrate patient-reported outcomes (PROs) into Phase I dose escalation with continuous enrollment, and Helen Barnett as well as Tom Parke discussing benchmarking safety models (model-assisted & model-based) in combination and single drug settings, respectively. Thomas Burnett chaired the session and kept us on track with many interesting questions from the audience. Last, but not least, I was really happy to see so many phones out at the end of my talk to scan the QR codes to the open-source material covering what I presented: * Applied modeling in drug development with {brms}: https://lnkd.in/eEcsGEzV (See chapter 10 on time-to-event modelling in Phase I Oncology) * Making your R code go fast(er) and run it on high-performance compute clusters, e.g., for simulation studies: https://lnkd.in/eByh2ebA #oncology #stats #rstats #stan #brms

Always an exciting and interesting dialogue to explain the broad immune agonist potential of Decoy20 as a novel investigational agent in patients with relapsed solid tumors.

Judging by the results of last week's poll, it seems oncology will be the field to watch over the next few years!   Breakthroughs in any field are exciting to watch, and a great opportunity for all companies and MSLs to join together in celebrating another win for science, and for patients. When science wins, we all win! #MedicalAffairs #MSL #MedicalScienceLiaison #pollresults

Learn how our FDA-cleared PGDx elio tissue complete solution for solid tumor profiling can help address these challenges, empowering your lab to enable access to precision oncology tests. Watch our webinar to learn more.