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RNatives Secures €1.4M Seed Extension to Advance RNA-Based Gene Regulation Platform

Precision Medicine
  • RNatives raised €1.4 million in additional seed funding led by Nordic Science Investments, bringing total seed funding to €2.8 million to accelerate its RNA-based gene regulating platform.
  • The company appointed Jeff Abbey as CEO, bringing over 20 years of biopharmaceutical experience including leadership roles at ImmunOs Therapeutics and Argos Therapeutics.
  • RNatives' RNAIntel™ platform identifies small non-coding RNAs that bind directly to gene promoters to regulate gene expression for treating ocular, CNS, metabolic diseases, and cancer.
  • The funding will support expansion of the company's precision RNA-based gene regulating therapies targeting currently untreatable rare and common diseases.

FDA-Approved FGFR Inhibitors Show Promise for Pediatric Brain Tumors in Preclinical Studies

Precision MedicineTargeted Therapy
  • Researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center found that 8.9% of pediatric gliomas harbor FGFR alterations and may respond to existing FDA-approved FGFR inhibitors.
  • The study developed first-ever preclinical models of FGFR-altered gliomas using neural stem cells and demonstrated tumor sensitivity to FGFR inhibitors in laboratory testing.
  • Retrospective analysis of pediatric patients with FGFR-altered low-grade gliomas showed frequent stable disease after treatment with FGFR inhibitors.
  • The research opens potential pathways for targeted therapy in pediatric gliomas and rare brain tumors like ETMR, addressing an urgent unmet medical need.

JAK Inhibitors Transform Polycythemia Vera Treatment as Novel Therapies Emerge

Precision Medicine
  • Ruxolitinib has established itself as a pivotal second-line therapy for polycythemia vera patients who are resistant or intolerant to hydroxyurea, showing significant improvements in hematocrit control and splenomegaly reduction.
  • Next-generation JAK inhibitors and hepcidin mimetics like rusfertide are emerging as promising treatment options that could complement current therapies and reduce the need for frequent phlebotomy.
  • Personalized treatment approaches using genomic profiling and biomarker-driven strategies are expected to enable more precise therapy selection based on specific mutations beyond JAK2.
  • Combination therapies targeting multiple disease mechanisms simultaneously are likely to become more common, offering enhanced efficacy and better long-term disease control.

Madras Diabetes Research Foundation Partners with Russia's Almazov Centre for AI-Driven Diabetes Research

AIPrecision Medicine
  • The Madras Diabetes Research Foundation (MDRF) has signed a strategic MoU with Russia's Almazov National Medical Research Centre to advance diabetes research through international collaboration.
  • The partnership will focus on developing new diagnostics and treatment strategies for diabetes, with particular emphasis on gestational diabetes mellitus (GDM).
  • Both institutions will explore artificial intelligence and precision medicine applications in diabetes care, marking a significant step toward technology-driven therapeutic approaches.
  • The collaboration includes joint research projects, academic exchanges, training programs, and shared publications between the Chennai-based foundation and the Saint Petersburg medical center.

Abbisko's FGFR4 Inhibitor Irpagratinib Receives China's Breakthrough Therapy Designation for Hepatocellular Carcinoma

Precision MedicineOncologyMonoclonal AntibodyTargeted Therapy
  • Abbisko Therapeutics' irpagratinib (ABSK011) has received Breakthrough Therapy Designation from China's NMPA for treating hepatocellular carcinoma, marking the first precision-targeted therapy using molecularly defined biomarkers for this indication.
  • The highly selective FGFR4 inhibitor targets FGF19 overexpression, which affects approximately 30% of HCC patients worldwide and is associated with significantly worse prognosis.
  • Clinical data from the 2024 ESMO GI Congress showed irpagratinib combined with atezolizumab achieved a 50% objective response rate in FGF19-positive HCC patients previously treated with immune checkpoint inhibitors.
  • The designation will expedite regulatory approval processes, potentially making irpagratinib the first globally approved FGFR4 inhibitor for HCC treatment.

A Phase 2, Open-Label Study of ABSK-011 Combined Atezolizumab in HCC Patients

NCT05441475RecruitingPhase 2
Abbisko Therapeutics Co, Ltd
Posted 12/30/2021

A Study Evaluating the Efficacy and Safety of Multiple Immunotherapy-Based Treatment Combinations in Patients With Advanced Liver Cancers (Morpheus-Liver)

NCT04524871RecruitingPhase 1
Hoffmann-La Roche
Posted 11/1/2020

A Study Evaluating Atezolizumab and Bevacizumab, With or Without Tiragolumab, in Participants With Untreated Locally Advanced or Metastatic Hepatocellular Carcinoma (IMbrave152)

NCT05904886Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 9/14/2023

Pfizer's BRAFTOVI Combination Therapy Halves Death Risk in BRAF-Mutant Metastatic Colorectal Cancer

Targeted TherapyPrecision MedicineOncology
  • Pfizer announced that its BRAFTOVI® combination regimen demonstrated a 50% reduction in death risk for patients with BRAF V600E-mutant metastatic colorectal cancer.
  • The targeted therapy represents a major advance in treating this specific genetic subtype of colorectal cancer.
  • This breakthrough could significantly improve survival outcomes for patients harboring the BRAF V600E mutation in metastatic disease.

Caris Life Sciences Files for $300 Million IPO to Advance AI-Powered Precision Oncology Platform

Precision MedicineAIOncology
  • Caris Life Sciences has filed for an initial public offering aiming to raise up to $300 million, with plans to list on Nasdaq under the ticker symbol "CAI".
  • The company has developed an AI-powered precision medicine platform for oncology, built on a massive dataset of over 6.5 million tests from 849,000 cases, generating measurements of more than 38 billion molecular markers.
  • Caris' commercial portfolio includes MI Profile, a tissue-based molecular profiling solution, and Caris Assure, a blood-based profiling solution launched in Q1 2024 for therapy selection.

Roche Partners with Broad Clinical Labs to Advance SBX Sequencing for Critically Ill Newborns

Precision Medicine
  • Roche and Broad Clinical Labs have formed a strategic collaboration to develop applications using Roche's next-generation Sequencing By Expansion (SBX) technology, initially focusing on whole genome sequencing for critically ill newborns.
  • The partnership aims to establish SBX technology as a routine component of clinical care in neonatal intensive care units (NICUs), enabling faster diagnosis of genetic disorders like cystic fibrosis and sickle cell disease.
  • Beyond neonatal applications, the collaboration will explore SBX technology for RNA sequencing, including bulk and single-cell approaches, to unlock novel molecular insights into disease mechanisms and potential therapeutic targets.

Ulefnersen Shows Unprecedented Recovery in Young Patients with Rare FUS-ALS

Rare DiseasePrecision MedicineNeurology
  • Columbia University researchers report that ulefnersen, an experimental drug, demonstrated remarkable efficacy in treating FUS-ALS, a rare genetic form of ALS affecting young people.
  • In a small case series of 12 patients, two showed exceptional responses, including one young woman who regained the ability to walk and breathe independently after treatment.
  • The therapy reduced neurofilament light, a biomarker of nerve damage, by up to 83% after six months, suggesting potential for not just slowing but reversing functional losses in early intervention.

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

NCT04512235Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 11/3/2020

A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis

NCT04973137TerminatedPhase 3
Prothena Biosciences Ltd.
Posted 8/30/2021

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

NCT04504825Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 2/2/2021

Servier to Present Phase 3 Data on IDH-Mutated Cancer Therapies at ASCO 2025

Precision MedicineTargeted TherapyOncology
  • Servier will showcase its precision medicine research for IDH-mutated cancers at the 2025 ASCO Annual Meeting in Chicago, focusing on chondrosarcoma, cholangiocarcinoma, and myelodysplastic syndrome.
  • The company, which allocates over 65% of its R&D budget to oncology, will present updates on several Phase 3 programs aimed at developing new treatment options for patients with IDH-mutated cancers.
  • As a global leader in IDH-mutant targeted therapies, Servier's presentations will reinforce its commitment to advancing precision medicine through mutation identification and understanding cancer progression mechanisms.

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