Flagship Pioneering’s Post

What will it take to advance the first tRNA medicines into the clinic? 🎙️ Tune in to this podcast with RNA Leaders to hear our CEO Michelle Werner share her thoughts about the next steps to support the development of this unique therapeutic modality, which has the potential to scale and accelerate genetic medicines for rare diseases. Michelle also previews what she plans to present at this year's conference in September. Listen here: https://lnkd.in/gPqgNu3R #RNALeaders #RNALeaders24 #tRNA #RNA #tRNAmedicines #tRNAbiology #rarediseases #geneticmedicines #programmablemedicines #machinelearning #AI #StopCodonDisease

Unlocking the Mystery of Disease Connections! 🧬 A new groundbreaking protocol uses gene interaction networks to uncover shared mechanisms between diseases. Discover how this approach sheds light on joint associations between diseases, aiding better diagnosis and therapy development. Interested in exploring disease links? Check out the protocol here: https://ow.ly/PaUF50QFMZZ #Biomedicine #DiseaseResearch #GeneNetworks #UCSD #UCSDHealth #UCSanDiegoHealth #UCSDACTRI #ACTRI

"Philanthropy has had a substantial role in piloting early efforts to develop individualized genetic medicines, supporting collaborations between academics, regulators, disease foundations and families. Scaling these efforts, however, is a substantial challenge. Tens of thousands of patients with ultra-rare diseases could someday be eligible for mutation-specific splice-switching ASO treatments. When other gene therapies and gene editing techniques, such as small interfering RNAs and CRISPR editing of DNA or RNA, are taken into consideration, the number of patients who could be served by individualized genetic therapies increases to the tens of millions." Getting from Mila to Millions will greatly depend on how to pay for individualized genetic medicines. A big thanks to Nature Portfolio/Nature Medicine for publishing our paper on this critical topic, and to my co-authors Julia P., Nana O., Winston Yan, MD PhD, Andrew Lo and Tim Yu. https://lnkd.in/gKq66iY4

The genetic medicine landscape in 2024 remains promising with a combination of scientific and regulatory readouts ready to combat a wide range of rare diseases. Learn more about the 3 critical areas underlying the efficient design and execution of sustainable genetic medicines. Read the latest #SyneosHealth Article: https://lnkd.in/diqxWGAJ #SyneosHealthLife #WorkHereMattersEverywhere #ClinicalCareers #SyneosHealth #ClinicalSolutions #2024HealthTrends 

By when, to whom, and how will you communicate change? Here are the key points: - There are no clear rules on how often genetic testing companies should review their results and update patients. - Labs struggle to find a balance between keeping patients informed and bombarding them with unnecessary updates. - Labs and doctors share responsibility for communicating with patients, but it can be difficult for doctors to track down patients, especially if they move or change doctors. - Some experts believe that the current system is unfair because it places more responsibility on patients who may not have the resources or knowledge to advocate for themselves. #Reanalysis #variant_reclassification #genetic_diagnosis #3billion https://lnkd.in/gu_bFTeF

Cystic fibrosis is a #genetic condition that primarily affects the lungs, pancreas, and other organs in the body, caused by mutation in #CFTR #gene. To detect early disease/ risk factors for parents who may be carriers, #RevvityOmics offers #NBS, CFTR gene #sequencing and #WES panel to screen the CFTR gene. To learn more and start a test order today visit: http://ms.spr.ly/6040Yb5Nu #Revvity #WholeExomeSequencing #Genetictesting #NGS

In the realm of rare diseases, genomic sequencing emerges as a transformative tool, offering swift and precise diagnoses while paving the way for personalized treatments. By unraveling the genetic mysteries underlying these conditions, genomic sequencing not only accelerates the path to diagnosis but also informs targeted therapeutic approaches. With each breakthrough, we move closer to a future where rare diseases are met with hope and the promise of a cure. #RareDiseaseAwareness #GenepoweRx #GeneticTestingIndia

CRISPR unlocks faster genetic disease diagnosis. Researchers in Denmark have used CRISPR technology to activate genes inaccessible cells, like skin and blood, potentially revolutionizing the diagnosis of rare genetic diseases. This breakthrough could lead to faster diagnosis of genetic diseases, addressing challenges in identifying the causes of symptoms. The researchers aim to introduce this technology into clinical diagnostics for improved accuracy and accessibility. Source: https://lnkd.in/dpDm-hJm #crispr #gene #disease #medical #biology

[INNOVATION UPDATE] OCU400 to Treat Retinitis Pigmentosa Retinitis pigmentosa (RP) is a group of rare inherited eye disorders that cause progressive vision loss, leading to disability and diminished quality of life. There are more than 100 genes associated with RP and there is currently an unmet need for treatments that will help patients with a wide range of RP-causing genetic variants. OCU400 is an experimental therapy designed to treat a broad population of patients with RP by delivering a novel, engineered regulatory gene to cells in the retina. Want to learn more about this novel therapy? View this Emerging Health Care Innovation Brief: https://hubs.la/Q02KXRN60 #retinitispigmentosa #ophthalmology #rarediseases #geneticdisorders #patientcare #healthcare #innovation #horizonscanning #ecri

New insights into Multiple Myeloma from the MMRF's Relating Clinical Outcomes study! Authors identified recurrent genetic events, 8 copy number & 12 expression subtypes, & high-risk groups. Excitingly, 25.5% of patients transition to a high-risk subtype with strong immunotherapy target expression, suggesting potential treatment options! #MultipleMyeloma #MMRF #PrecisionMedicine #Research