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Many cell and gene therapies are in clinical development for autoimmune and other diseases beyond cancer. Diseases like multiple sclerosis, rheumatoid arthritis, Type 1 diabetes, Sjogren's syndrome, and systemic lupus erythematosus affect various organs and involve complex immune responses. Despite the excitement for potential new treatments, disease diagnosis remains slow and biomarkers of efficacy for new treatments are greatly needed. By identifying new biomarkers, we can develop more precise and personalized therapies, significantly improving patient outcomes and quality of life. #AutoimmuneDiseases #GeneTherapy #Biomarkers #HealthcareInnovation #ChronicIllness

A significant number of primary immunodeficiency diseases (PIDs) were first reported in patients living in the Middle East. Given the high rate of consanguinity in the region, PIDs are commonplace across the region. According to the Center of Arab Genomic Studies about 2.8 million patients are living with a rare disease in the Middle East. One such PID is the Wiskott-Aldrich syndrome - a rare genetic immunodeficiency which makes it difficult for the bone marrow to produce platelets, making a child prone to bleeding. Treatment options include immunoglobulin (antibody) infusions, platelet transfusions, topical creams for eczema, and steroids or similar medications to control autoimmunity. In severe cases a bone marrow transplant or gene therapy can also be considered. On the occasion of rare disease day, CSL Behring is working relentlessly to provide hope to several patients across the world. #MEA #RareDisease #DrivenbyOurPromise #WiskottAldrichSyndrome

An easy-to-use prediction classification system (the CROSS model) could aid in the early detection patients anti-melanoma differentiation-associated gene 5 positive #dermatomyositis (anti-#MDA5+) who have a high risk to develop rapidly progressive interstitial lung disease (RP-ILD). This model uses clinical variables that could be easily obtained during the routine clinic visit, and based on the CROSS score (range from 0 to 6), anti-MDA5+DM patients were classified as moderate risk (CROSS score = 0-2), high risk (CROSS score = 3-4), and very high risk (CROSS score = 5-6) for developing of RP-ILD, respectively. 🔗https://lnkd.in/dQMHDseY

29 February is #RareDiseaseDay, an opportunity to raise awareness of the urgent needs of over 300 million people globally living with a rare disease. Today, we would like to shine a spotlight on steroid resistant nephrotic syndrome (#SRNS), a rare, therapy-resistant form of nephrotic syndrome, which affects the kidneys’ ability to perform critical filtering functions. Children with SRNS do not respond to steroids, and will likely need dialysis and, ultimately, a kidney transplant, with a serious impact on their quality of life and significant long-term complications. At least a third of children with SRNS have an inherited, or genetic, cause of their disease. There is an urgent need for effective new treatments, but recent data suggest that the podocyte, a specialised cell type that is implicated in many #KidneyDiseases, may be the ideal cell type to target with AAV gene therapy, offering the potential for a long-term correction to the genetic causes of kidney diseases such as SRNS. If you’d like to hear more about what Purespring does to support people with kidney diseases such as SRNS, please get in touch. #RareDiseaseDay #OrphanDisease #LightUpForRare #RareDiseaseDay2024 #ShareYourColours

Geographic atrophy (GA) is a late-stage and severe form of age-related macular degeneration (AMD), the most common cause of irreversible vision loss in older individuals. It causes wavy or blurry vision, visual distortion, loss of central vision, change in the perception of color, and the inability to drive, read, or recognize faces. The only available treatment option is monthly or bimonthly intraocular injections that require recovery time, interrupting a patients’ regular flow of life for several days per year.   While this treatment may help to slow the progression of this severe disease, there is no guarantee it will preserve sight.   At Johnson & Johnson Innovative Medicine, we refuse to settle for this reality. Earlier this year, we announced that we had started enrolling patients in the Phase 2b PARASOL study exploring a revolutionary, one-time, investigational treatment for patients with GA.   GA is a disease landscape that demands innovation – and we will continue to keep pace as we explore and advance novel gene therapies. To learn more about our commitment to find solutions for rare and common retinal diseases like GA, check out my latest piece in STAT.   bit.ly/STATGeneTherapy  #MyCompany #Retina #GeneTherapy

Looking forward to seeing you in Boston José M Carballido! See more information on the 7th ASIT Summit here: https://ter.li/iaozo1 Join Jose and 100+ immune tolerance enthusiasts, decision makers, and innovators at the 7th Antigen-Specific Immune Tolerance Summit to: Identify antigens driving autoimmune diseases & biomarkers of antigen-specific immune tolerance, evaluate tissue-specific tolerogenic approaches & novel delivery systems, & advance durable therapeutics into proof-of-concept trials and beyond 2024 Event Partners include: Novoviah Pharmaceuticals, Barinthus Biotherapeutics, Nykode Therapeutics, Beacon See the full event guide here: https://ter.li/iaozo1 #ASIT #ASIT2024 #immunetolerance #tolerance #tolerizing #antigenspecific #antigen #celiacdisease #type1diabetes #multiplesclerosis #rheumatoidarthritis #drugdevelopment #lnp #mrna #tregs

A systematic review (DOI: 10.1002/ijd3.1182) examines autophagy-related gene polymorphisms and their impact on HBV-associated hepatocellular carcinoma (HCC). Of 422 studies, polymorphisms like rs2241880 in ATG16L1 and rs77859116, rs510432, and rs548234 in ATG5 were significant. These genetic variations were notably associated with an elevated risk of HBV infection, which could lead to HCC. This underscores the genetic underpinnings that may influence HBV infection outcomes and HCC development. #LiverCancer #GeneticRiskFactors #AutophagyGenes #HBVResearch #MedicalGenomics

Today is World Parkinson's Day – a day dedicated to raising public awareness of, and supporting those affected by, Parkinson’s disease (PD). Parkinson's disease is the fastest growing and the second most prevalent neurological condition, affecting an estimated 10 million people worldwide. Despite significant advancements in treatment, there is still no known cure for the disease. Levodopa-induced dyskinesia (LID) is a prevalent and debilitating side effect of chronic levodopa treatment, the current gold-standard for managing PD motor symptoms. Around half of PD patients are affected by LID after 5 years of levodopa treatment and up to 90% after 10 years, with current treatment options providing only partial therapeutic benefit on LID.   We are developing BGT-PD, an investigational AAV2 gene therapy candidate for the treatment of LID in Parkinson’s disease. First preclinical data on our BGT-PD program will be presented at the upcoming American Society of Gene & Cell Therapy 27th Annual Meeting.    Learn more about BGT-PD here: https://lnkd.in/e2tZTHQm #WorldParkinsonsDay #ParkinsonsAwarenessMonth #ASGCT2024 #genetherapy #rarediseases #aav #NeurodegenerativeDisorders #advancedtherapies #Parkinsonsdisease 

#CeliacDiseaseResearch tackles disease management from all angles: immune response modulation, microbiome manipulations, and precision medicine approaches for those with celiac-promoting HLA gene variants. Enzymatic approaches, like our primary asset #latiglutenase, break down gluten into harmless fragments, aiding gluten digestion and reducing immune reactions for all people living with #CeliacDisease. Learn more about the state of celiac disease research, diagnostics, and progress towards treatments: https://bit.ly/3Wbyjq3

Sickle cell disease (SCD) is a rare hematological disease affecting mainly patients of African descent. It is estimated that SCD occurs in 1 out of every 365 Black or African American births. Many physicians are not familiar with the disease pathophysiology, complications, and current standard of care and treatment options. There are several emerging therapies for SCD in the pipeline, and in December 2023, the FDA approved the first cell-based gene therapies for sickle cell disease treatment. It is important for healthcare professionals to know about SCD and its current and future treatment options.   Find out more about sickle cell disease in our latest blog https://lnkd.in/gsrscTni To schedule an update for your medical staff on Sickle Cell Disease, consider contacting our office at 877-505-4777 or email us at info@speakersnetwork.com.   #medicaleducation #cme #patienthealth #medicaleducationspeakersnetwork #training #blog #medicalblog #sicklecelldisease Maria Anderson Elizabeth Sharif