ICAHN School of Medicine At Mount Sinai
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private, Subsidiary
- Established
- 1963-01-01
- Employees
- 5K
- Market Cap
- -
- Website
- http://www.mssm.edu
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
Mount Sinai's Personalized Vaccine Shows Promise in Bladder Cancer Clinical Trial
• Researchers at Mount Sinai have demonstrated that their personalized cancer vaccine PGV001, when combined with immune checkpoint inhibitor atezolizumab, is safe and generates strong immune responses in bladder cancer patients.
• The phase 1 trial showed vaccine-specific T cell responses in all participants, with only mild injection-site reactions reported, suggesting potential for both metastatic bladder cancer patients and those receiving post-surgical therapy.
• This breakthrough adds to growing evidence that personalized vaccines could enhance standard cancer treatments, particularly for bladder cancer which affects approximately 84,870 Americans annually with 17,420 expected deaths this year.
Novel Contact Force-Sensing Pulsed Field Ablation Catheter Shows Promise in Paroxysmal AF Treatment
• First-in-human data demonstrates 100% acute procedural success with Omnypulse, a novel contact force-sensing pulsed field ablation catheter for paroxysmal atrial fibrillation treatment.
• The catheter showed promising durability with 84.5% of pulmonary veins remaining isolated at 3-month follow-up, with only four non-device-related adverse events reported.
• Comparable technology from Kardium (Globe) demonstrated 78% freedom from treatment failure at 12 months with 95% pulmonary vein isolation durability, suggesting significant advancements in AF ablation technology.
BrightHeart Secures Third FDA Clearance for AI-Powered Fetal Heart Ultrasound Technology
• BrightHeart's B-Right Views has received FDA 510(k) clearance, marking the company's third regulatory approval for AI-powered fetal heart ultrasound assessment technology.
• The new AI software provides automated confirmation of standard fetal heart views during second and third-trimester ultrasounds, helping ensure consistent assessment quality regardless of clinician experience.
• With up to 70% of congenital heart defects going undetected during standard prenatal ultrasounds, BrightHeart's technology aims to improve detection rates through real-time analysis and alerts delivered via cart-side tablet integration.
American Heart Association Awards $1M to Study GLP-1 Medications' Impact on Cardiovascular Risk
• The American Heart Association has funded seven research projects with $1 million to identify which patients with obesity and cardiovascular disease benefit most from GLP-1/GIP medications.
• Research teams from seven prestigious institutions will investigate biological, clinical, and social factors influencing treatment response, using the AHA's Precision Medicine Platform for data analysis.
• Studies will examine how GLP-1/GIP medications affect cardiovascular outcomes across different populations, with aims to develop more personalized treatment approaches and address health disparities.
Declining Childhood Vaccination Rates Raise Alarm for Resurgence of Preventable Diseases
• Recent research published in JAMA predicts millions of preventable disease cases over the next 25 years if childhood vaccination rates continue to decline from their current level of under 93%.
• A study in the American Journal of Public Health found that children of parents who declined COVID-19 vaccines are approximately 25% less likely to receive standard MMR vaccinations.
• Measles is projected to be the first disease to return to endemic levels, with Texas identified as the highest-risk state for outbreaks based on current vaccination trends.
Alarming Gap in Dementia Diagnosis Awareness Reveals Healthcare Disparities
• Over 75% of dementia patients are unaware of their diagnosis despite having access to primary care, with higher unawareness rates among Mexican-Americans (85%) compared to white patients (68%).
• A separate study found that 75% of low-income seniors at community health centers had undiagnosed cognitive issues, with Black patients more than twice as likely as whites to have unrecognized cognitive impairment.
• Medical experts emphasize that timely dementia diagnosis is crucial for proper treatment, support services, and potential interventions that could slow disease progression.
Mount Sinai Launches AI-Powered Center to Revolutionize Small Molecule Drug Discovery
• The Icahn School of Medicine at Mount Sinai has established the AI Small Molecule Drug Discovery Center to accelerate drug development by integrating artificial intelligence with traditional discovery methods.
• The center will focus on three core areas: designing novel drug-like molecules using generative AI, optimizing existing compounds, and predicting drug-target interactions for repurposing known drugs.
• Led by Dr. Avner Schlessinger, the initiative aims to address urgent unmet needs in cancer, metabolic disorders, and neurodegenerative diseases through collaborations with pharmaceutical companies and academic institutions.
Mount Sinai's Personalized Cancer Vaccine PGV001 Shows Promise Across Multiple Tumor Types in Phase 1 Trial
• Mount Sinai researchers demonstrated that PGV001, a personalized multi-peptide neoantigen cancer vaccine, generated strong immune responses across five different cancer types with no serious side effects reported.
• The phase 1 trial showed encouraging long-term outcomes with six of 13 treated patients surviving at five-year follow-up, including three patients who remain tumor-free, suggesting potential correlation with improved survival.
• Based on these promising results, three additional clinical trials of PGV001 have been initiated in glioblastoma, urothelial cancer, and prostate cancer to further evaluate its efficacy alone and in combination with other therapies.
Verastem Oncology to Present New LGSOC Treatment Data at SGO 2025 Annual Meeting
• Verastem Oncology will present additional analyses from the Phase 2 RAMP 201 trial evaluating avutometinib plus defactinib in recurrent low-grade serous ovarian cancer at SGO 2025, building on their FDA Priority Review submission.
• The presentations include new subgroup analyses by KRAS mutational status, supporting the company's ongoing efforts to advance treatment options for RAS/MAPK pathway-driven cancers.
• The company's NDA for the avutometinib-defactinib combination therapy has received Priority Review from the FDA with a PDUFA date of June 30, 2025, for KRAS mutant LGSOC patients.
Purple Biotech Launches Phase 2 Trial of NT219 for Drug-Resistant Head and Neck Cancer
• Purple Biotech initiates Phase 2 trial of NT219, a novel dual-pathway inhibitor, in combination with pembrolizumab or cetuximab for treating recurrent/metastatic head and neck cancer.
• The study, led by Dr. Antonio Jimeno at University of Colorado, will evaluate NT219's potential to overcome tumor resistance in a $5 billion market where current treatments show only 15-20% response rates.
• NT219's unique mechanism targeting IRS1/2 and STAT3 pathways showed promising results in preclinical studies, demonstrating ability to reverse immunotherapy resistance in tumors.
ACC.25 Highlights Novel Therapies and Strategies in Cardiovascular Care
• The SOUL trial demonstrated that oral semaglutide significantly reduces cardiovascular events in type 2 diabetes patients with atherosclerotic cardiovascular and/or chronic kidney disease.
• The RIVAWAR trial investigated the efficacy of rivaroxaban versus warfarin in patients with acute left ventricular thrombus following myocardial infarction.
• The FAIR-HF2 trial assessed the impact of ferric carboxymaltose on morbidity and mortality in patients with iron deficiency and chronic heart failure.
• The ALPACA Phase 2 trial of Lepodisiran showed promising results for an extended duration small-interfering RNA targeting Lipoprotein(a).
FDA Approves Spevigo: First-Ever Treatment for Generalized Pustular Psoriasis Flares
Boehringer Ingelheim's spesolimab (Spevigo) has received FDA approval as the first dedicated treatment for generalized pustular psoriasis (GPP) flares. In the pivotal EFFISAYIL 1 trial, 54% of patients treated with spesolimab showed complete clearance of pustules within one week, marking a significant advancement in the management of this rare, life-threatening skin condition.
BioNTech Advances Oncology Pipeline with BNT327/PM8002 and mRNA Immunotherapies
• BioNTech is progressing BNT327/PM8002, a bispecific antibody, into global clinical trials for first-line small cell lung cancer and non-small cell lung cancer.
• The company plans to initiate additional trials combining BNT327/PM8002 with antibody-drug conjugates (ADCs) in 2025, expanding its combination strategy.
• BioNTech's mRNA cancer immunotherapy, autogene cevumeran, is being evaluated in a Phase 2 trial for muscle-invasive urothelial carcinoma in combination with nivolumab.
• Clinical data readouts are expected in 2025 and 2026 from multiple randomized trials of personalized and off-the-shelf mRNA cancer immunotherapy candidates.
Durvalumab Gains Priority Review for Muscle-Invasive Bladder Cancer Treatment
• The FDA has granted priority review to durvalumab (Imfinzi) for muscle-invasive bladder cancer (MIBC), potentially expediting its approval.
• Phase 3 NIAGARA trial data supports the application, showing a 32% reduction in disease progression or recurrence with perioperative durvalumab.
• The Imfinzi regimen also demonstrated a 25% reduction in the risk of death, offering a significant survival benefit.
• Regulatory decisions are anticipated in the second quarter of 2025, with potential implications for MIBC treatment standards.
Genetic Signature Predicts Venclexta Response in Multiple Myeloma
• A six-gene pattern can predict which multiple myeloma patients will respond favorably to Venclexta (venetoclax) therapy, according to a new study.
• The genetic test could help customize treatments, improving success chances by identifying the best candidates for venetoclax treatment.
• Researchers found Venclexta's effectiveness is enhanced when combined with a CDK7 inhibitor, promoting natural cell death in cancer cells.
• Future studies will validate findings in larger cohorts and test Venclexta-CDK7 inhibitor combinations in clinical trials for personalized treatment plans.
Transferrin Receptor-Targeted ASOs Show Promise in Crossing the Blood-Brain Barrier
• Researchers have developed antisense oligonucleotides (ASOs) conjugated with a transferrin receptor (TfR)-targeting antibody fragment for enhanced brain delivery.
• Intravenous administration of these TfR-ASO conjugates in mice demonstrated effective ASO distribution throughout the brain.
• The TfR-ASO conjugates led to significant target mRNA reduction in the brain, showcasing their potential for treating neurological disorders.
• This targeted delivery approach may improve the efficacy and reduce systemic exposure of ASO therapeutics for CNS diseases.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
Early Heart Valve Replacement Shows Superior Outcomes in Aortic Stenosis Patients
• A new study suggests early heart valve replacement with minimally invasive procedures is more effective for patients with severe aortic stenosis.
• Early surgery halved the risk of hospitalization for heart problems compared to the traditional watch-and-wait approach.
• The Early TAVR study showed that early transcatheter aortic-valve replacement (TAVR) significantly reduced the incidence of death, stroke, or unplanned hospitalization.
• The Triscend II trial demonstrated that transcatheter tricuspid valve replacement improved outcomes in patients with tricuspid valve regurgitation.
Fibrinogen-to-Albumin Ratio Predicts Preeclampsia Risk in Laboring Women
• A new study reveals that the fibrinogen-to-albumin ratio (FAR) can predict preeclampsia risk in women upon hospital admission for labor.
• Women with an elevated FAR, specifically above 0.1, showed a 24% increased risk, while those above 0.3 had over a 41% increased risk of preeclampsia.
• The FAR assessment could provide anesthesiologists and obstetricians with a valuable tool for early identification and management of preeclampsia risks.
• Further research is needed to establish a universally accepted FAR range for preeclampsia prediction and its integration into routine prenatal care.
© Copyright 2025. All Rights Reserved by MedPath