CURE ID’s Post

C-Path's Cure Drug Repurposing Collaboratory, and the CURE ID team will be onsite at the International Drug Repurposing Conference 2024 (#iDR24): Bridging boundaries for Innovative Drug Repurposing this week where key leaders in drug repurposing will unite. Co-organized by REMEDi4ALL, Beacon for Rare Diseases and MeRIT, the conference showcases how patient communities, researchers, industry and health authorities can benefit from cutting-edge drug repurposing. The agenda follows three topical paths in parallel: · Cancer · Rare Disease · Methodologies Find out more about the speakers, location, details here: https://lnkd.in/eRtYcAmZ Smith Heavner, PhD, RN | Marco Schito | Heather Stone | Shira Strongin | Keyla Tumas #CPath #REMEDi4ALL #Beacon #MeRIT #drugrepurposing #patientadvocacy

Making rare disease drug development happen, and happen faster, by looking at rare disease drug development paradigms, and how to shift them. All explained in Volv Global SA's talk at the World Orphan Drug Congress Europe 2024, next Wednesday - see you there!

I am pleased to share a report from Copenhagen Economics that Matthias Heck and I inputted to, alongside experts from EURORDIS and Takeda. The data presents some very interesting implications for the concept of High Unmet Medical Need (HUMN) as a proposed incentive for orphan drug exclusivity under the revised General Pharmaceutical Legislation; namely the continued need for incremental innovation for people living with a rare disease, the startling lack of research underway for the 90% of rare diseases without an authorised treatment, and the need for realistic criteria for determining HUMN. Innovation must be encouraged, not disincentivised through modulations that are not readily achievable, while a significant increase in public funding toward basic research must be provided if we are to truly accelerate new discovery.

In light of the European Commission’s recent proposal to revamp the incentive system for orphan medicinal product (#OMP) development, we delve into the implications of the new High Unmet Medical Need (#HUMN) category, which aims to bring transformative treatments for rare diseases that currently lack effective therapies. The introduction of the HUMN category represents a paradigm shift in how we incentivize OMP development. From today’s system, where incentives make the investment case for innovation in OMPs possible, to a reward-based system that introduces a separate category for HUMN treatments reducing incentives anywhere else.   While the reward-based approach may seem attractive, we show that transformative treatments tend to emerge as the results of many incrementally innovative treatments that serve to build up the knowledge base that allows transformative treatment to be developed. When incentives for incremental treatments are reduced, the pathway to transformative treatments weakens. This may in fact lead to fewer transformative treatments being developed, thereby working against the ambitions of the Commission.   This publication has been co-authored by EURORDIS-Rare Diseases Europe, Copenhagen Economics, Alexion AstraZeneca Rare Disease, Takeda, and in collaboration with Orphanet. Read it here: https://lnkd.in/deEajTkd   #copenhageneconomics #raredisease #EU #innovation #healthcare Yann Le Cam, Simone Boselli, Christian Jervelund, Malwina Mejer, Colin O'Donnell, Matthias Heck, Luana Banu, Toon Digneffe 

Ultra rare was topic of a roundtable session at #ISCT2024 organized by Mo Heidaran and Michael Mendicino, Ph.D. and esteemed panelists included Nicole Verdun Julie Douville and Donald B. Kohn. In my opinion the commercial viability of highly transformational therapies for ultra rare diseases requires a new approach but one that is within framework of existing regulatory requirements. I found n=1 clinical trials conducted by n-lorem foundation as possibly a blueprint for future success. I am curious what your thoughts are on this topic. #celltherapy #genetherapy #cellandgenetherapy #nih #ncat #fda

🔬 Exciting findings from the ENVISION trial: UBX1325 shows promise in advanced #wetAMD cases. Despite not meeting primary endpoint, it's safe and well-tolerated. Dr. raj maturi shares insights on its unique mechanism targeting senescent cells. Significant proportion of patients didn't require anti-VEGF treatment through 24 weeks. Secondary analysis hints at efficacy in longer disease duration patients. Ongoing exploration of combination therapy potential in Part B of ENVISION study. 👉 Learn more in this article from Healio by Michela Cimberle, fact checked by Christine Klimanskis #agerelatedmaculardegeneration #maculardegeneration #cataract #ophthalmology #ophthalmologynews

A new partnership between RCSI and Irish diagnostics company, Serosep Ltd. is set to improve the quality of life for those living with ulcerative colitis by introducing personalised treatments.   The organisations will work to develop technology to identify ulcerative colitis patients who are likely to progress in their disease, ensuring that people get the most effective care for their disease, and sparing people treatments where they are not likely to be of benefit to them. The project will accelerate the development of new tests to help predict disease progression in ulcerative colitis, a form of inflammatory bowel disease that damages the lining of the gut over time. “Currently there are no biomarkers which allow prediction of disease progression in patients with ulcerative colitis,” explained Dr Sudipto Das, Lecturer and Principal Investigator at RCSI School of Pharmacy and Biomolecular Sciences. “Having such biomarkers could augment decision-making for clinicians about whether the patient could benefit from an escalation of treatment at an early stage of the disease.” Read more about the project here: bit.ly/47QfX1A The partnership between Serosep and RCSI is supported by the RCSI Innovation team and is co-funded by Serosep and the Enterprise Ireland Innovation Partnership Programme. #RCSIdiscover

Did you miss a session yesterday? The recording is up! Find the presentations from Bernard Siegel, Cory R. Nicholas, PhD and Peter Marks and the discussions following with Anders Lindahl, Elias Uhlin and Maria Lüttgen, respectively. Session 1: How to use the 'advocates toolbox' to engage public sentiment...."with public sentiment, nothing can fail; without it nothing can succeed" Abraham Lincoln. How can we advocate for effective ATMP treatments for healthcare to enable people to not only live longer but live healthier longer. Session 2: Is it possible that cell therapies to the brain will not invoke immune responses? Will this be an organ where immune matching will not be necessary? How can we expedite development by using delivery routes and specialist clinics already exisiting in the indication? What are the safety implications at clinical trial for ATMPs that are less invasive than current treatments. Session 3: How is the FDA supporting rare disease gene therapies? Great discussion here on 'platform' approvals specific to a single company and 'process' standardisation to support developers in sufficiently defining their products to enable successful technology transfer. Even global regulatory harmonisation to simplify processes for global treatment of rare disease patients. https://lnkd.in/ggS_UR6V

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