Citeline Clinical’s Post

Increased awareness and collaboration are key to innovation. Orphan drug innovation has gone from strength to strength, propelled by advances in genetic medicine, such as mRNA therapies. However, challenges remain. In this article, Will Maier, VP and Head of Centre for Rare Disease, ICON Biotech, explores the challenges and the necessary solutions to advance rare disease drug development in today’s dynamic landscape. Read it now: https://ow.ly/pitn50RxcHN. #RareDisease #OrphanDrug #DrugDevelopment #HealthcareInnovation #RareDiseaseAwareness

Increased awareness and collaboration are key to innovation. Orphan drug innovation has gone from strength to strength, propelled by advances in genetic medicine, such as mRNA therapies. However, challenges remain. In this article, Will Maier, VP and Head of Centre for Rare Disease, ICON Biotech, explores the challenges and the necessary solutions to advance rare disease drug development in today’s dynamic landscape. Read it now: https://ow.ly/9gmi50RxcJ1. #RareDisease #OrphanDrug #DrugDevelopment #HealthcareInnovation #RareDiseaseAwareness

Increased awareness and collaboration are key to innovation. Orphan drug innovation has gone from strength to strength, propelled by advances in genetic medicine, such as mRNA therapies. However, challenges remain. In this article, Will Maier, VP and Head of Centre for Rare Disease, ICON plc, explores the challenges and the necessary solutions to advance rare disease drug development in today’s dynamic landscape. Read it now: https://ow.ly/Mrvx50RxcJI. #RareDisease #OrphanDrug #DrugDevelopment #HealthcareInnovation #RareDiseaseAwareness

Patients… patients… patients. That’s who we work with and for at GeneDx . Listen to Katherine Stueland talk about how working with patients, clinicians, advocacy organizations, researchers, and BioPharma all matter when we talk about better healthcare. #RareDisease #Genetics #Exome #GeneDx

The PHARMO Institute and Lumanity are partnering to deliver research insights into amyloidosis and its hard-to-identify subtypes, such as light chain (AL), amyloid (AA), and transthyretin amyloidosis (ATTR), and we invite you to put our data to the test. Our data is well-suited/ ready for conducting amyloidosis research with: - >10,000 patients with an amyloidosis related hospital admission or ambulatory consultation - Unique linkages to clinical labs and pathology data to uncover specific blood-based test results, biopsy, and gene sequences that point to clinical subtypes - Ability to address a range of RWE use cases from drug safety, treatment patterns, comparativeness effectives, clinical outcomes, and beyond Learn more about our expertise in Amyloidosis at https://buff.ly/42UVVlZ and contact us to discuss your research needs in amyloidosis. #amyloidosis #amyloidosisresearch #RWE #realworldevidence #drugsafety #ATTR

Unlocking the Potential of RNA Editing: A Promising New Frontier in Genetic Medicine: In a remarkable scientific breakthrough, RNA-editing drugs are rapidly advancing from the preclinical stage into early-stage clinical trials, offering new hope for patients with genetic disorders. This emerging field of genetic medicine holds immense promise, as these innovative therapies harness the power of RNA editing to directly address the underlying genetic defects, rather than merely managing symptoms. The article in Nature highlights several promising RNA-editing drug candidates that have entered clinical trials, targeting a diverse range of genetic conditions, including Duchenne muscular dystrophy, cystic fibrosis, and other rare disorders. The successful development and approval of these therapies could pave the way for a new era in personalized genetic medicine, where targeted treatments become the norm, transforming the lives of countless individuals affected by genetic disorders. As these RNA-editing drugs progress through clinical trials, the scientific community and the broader public eagerly await the outcomes. The potential of these groundbreaking therapies to address the root cause of genetic conditions and provide new treatment options for patients with limited or no effective therapies available is truly exciting. The future of genetic medicine has never been brighter. #Cancer #rna #genetics #dna #oncology #vaccine #clinicaltrials #drugdiscovery #innovation #genetherapy #medicine #genomics

Today is Rare Disease Day. Millions of people are affected by often overlooked and under-researched rare diseases. The path to future treatments is complex, and many scientific challenges remain. But I and many others believe that RNA therapeutics hold the promise of precise, targeted treatments. At Abzu, we are acutely aware of the scientific challenges that impede the progress of RNA therapeutics. Our technology enables a deeper understanding of biological interactions, paving the way for more effective, successful, and fast drug development. We are proud to be at the forefront of accelerating the development of innovative RNA-based drugs. Last year, a record 10% of all new drugs were RNA-based short oligos. In 5 years, I see no reason why it shouldn't be 50%, making a real dent in the sad list of currently untreatable diseases.

Orphan drug innovation has been propelled by advances in genetic medicine. The fact is that challenges remain. In this article, Will Maier, VP and Head of Centre for Rare Disease, ICON Biotech, explores the challenges and the necessary solutions to advance rare disease drug development in today’s dynamic landscape. Topics include identifying patient populations, the role of patient advocacy organizations, scalable containment, regulatory complexity, reimbursement concerns, and CRO partnerships. Read it now: https://ow.ly/kKyL50Rwev5. #RareDisease #OrphanDrug #DrugDevelopment #HealthcareInnovation #RareDiseaseAwareness

Orphan drug innovation has been propelled by advances in genetic medicine. The fact is that challenges remain. In this article, Will Maier, VP and Head of Centre for Rare Disease, ICON Biotech, explores the challenges and the necessary solutions to advance rare disease drug development in today’s dynamic landscape. Topics include identifying patient populations, the role of patient advocacy organizations, scalable containment, regulatory complexity, reimbursement concerns, and CRO partnerships. Read it now: https://ow.ly/nrQp50RRy6i. #RareDisease #OrphanDrug #DrugDevelopment #HealthcareInnovation #RareDiseaseAwareness

Pharmacogenomics examines how genes influence medication responses, helping to identify risks for adverse drug reactions (ADRs), toxicity, or reduced effectiveness. This is particularly valuable for individuals on multiple medications. Using pharmacogenomics, doctors can tailor prescriptions to your genetic profile, reducing side effects, minimising ADR adverse drug reaction risks, and avoiding ineffective treatments. This approach can also lower healthcare costs by preventing unnecessary medication use and hospitalisations. A review of 108 studies found that 71% show pharmacogenomics testing is cost-effective or cost-saving, especially for cardiac medications, antiepileptics, and antidepressants. PharmaGene goes beyond testing by offering a live portal for doctors to check drug interactions and side effects, enabling personalized and optimized treatment plans that save time and money. Read the full blog: https://ow.ly/a0X550SjBMW For more information on PharmaGene, click here: https://ow.ly/wln650SjBMX