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dGenThera and Nusano Partner to Scale Astatine-211 Production for Next-Generation Cancer Therapy

Precision MedicineOncology
  • dGenThera and Nusano signed a letter of intent to provide reliable access to high-purity astatine-211, a best-in-class alpha-emitting isotope for targeted cancer therapy.
  • Nusano's breakthrough accelerator technology will increase global At-211 supply by 100-fold through a facility with alpha beam current 10 times greater than all comparable systems combined.
  • The partnership enables dGenThera to advance its theranostic molecular pairs platform, which uses covalently-bonded At-211 to create precision radiotherapies that can cross the blood-brain barrier.
  • At-211's unique properties include pure alpha emission with no problematic daughter isotopes and a 7.2-hour half-life ideal for systemic delivery and tumor targeting.

Historical Challenges in NF1-Associated Plexiform Neurofibroma Treatment Drive Development of Targeted Therapies

Targeted TherapyPrecision Medicine
  • Previous therapies for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) have shown limited efficacy and high toxicity, particularly in tumor shrinkage and symptom control.
  • Approximately 50% of individuals with NF1 develop associated plexiform neurofibromas, which are slow-growing tumors involving multiple nerves that rarely allow complete surgical resection.
  • The indolent growth pattern of these tumors has posed longstanding challenges in developing systemic therapies that demonstrate clear therapeutic effects while maintaining long-term tolerability.
  • Tumor heterogeneity and resistance mechanisms have highlighted the need for combination therapies and molecular profiling-based patient selection to improve treatment outcomes.

CNS Oligonucleotide Licensing Deals Surge 339% to $2.81 Billion in 2024

Precision Medicine
  • Licensing agreements for CNS-targeted oligonucleotide therapeutics increased 339% from 2023 to 2024, reaching $2.81 billion in total deal value according to GlobalData's analysis.
  • Antisense oligonucleotides (ASOs) dominated the market with $3.54 billion in cumulative deals from 2021-2025, while siRNAs accounted for $2.51 billion during the same period.
  • Major pharmaceutical companies including AbbVie, Lilly, Roche and Takeda represented 63% of licensing deals, with Ionis Pharmaceuticals leading the field through $13.4 billion in out-licensed assets over the past decade.
  • Arrowhead Pharmaceuticals secured 2025's largest deal worth up to $2.18 billion through an exclusive licensing agreement with Sarepta Therapeutics for rare genetic and CNS disease programs.

Precision Medicine Oncology Pharmacists Emerge as Critical Players in Personalized Cancer Care

Precision MedicineTargeted Therapy
  • Precision medicine oncology pharmacists have become essential members of multidisciplinary cancer care teams, contributing to targeted therapy selection, molecular tumor board discussions, and patient access to novel drug combinations.
  • These specialized pharmacists address significant barriers to precision oncology implementation, including complex medication access pathways, insurance restrictions, and the need for comprehensive patient education on oral anticancer medications.
  • Despite the expanding role of pharmacists in precision medicine, up to 70% remain unaware of institutional precision medicine initiatives, highlighting the need for increased training and institutional support to fully realize their potential in personalized cancer care.

AI Model Reveals 46% Cognitive Decline Reduction in Alzheimer's Trial Reanalysis

AIPrecision Medicine
  • Cambridge researchers used AI to reanalyze a failed Alzheimer's clinical trial, finding the drug lanabecestat slowed cognitive decline by 46% in patients with early-stage, slow-progressing mild cognitive impairment.
  • The AI model stratified patients into slow and rapid progressors with three times greater accuracy than standard clinical assessments using memory tests, MRI scans, and blood tests.
  • This precision medicine approach could accelerate drug discovery by identifying optimal patient populations for treatment, potentially reducing the 95% failure rate and $43 billion spent on unsuccessful dementia research.
  • The findings demonstrate that while the drug cleared beta amyloid protein in both patient groups, only slow-progressing patients showed symptomatic improvements in the AMARANTH trial.

Dapagliflozin Combination Therapy Shows Promise for Adolescents with Type 1 Diabetes in Clinical Trial

Precision Medicine
  • A multicenter clinical trial found that dapagliflozin combined with insulin reduced kidney disease risk and improved health outcomes in 98 adolescents with type 1 diabetes over 16 weeks.
  • The combination therapy demonstrated significant improvements including 8.8 ml/min reduction in glomerular filtration rate, 0.47% decrease in HbA1c, and 2.8 kg weight reduction compared to placebo.
  • Results showed similar safety profiles between treatment groups with only one mild case of diabetic ketoacidosis in the dapagliflozin group, suggesting the therapy could inform new early intervention strategies.
  • The study addresses a critical gap in adolescent diabetes research, as this age group is often underrepresented in clinical trials despite facing unique challenges with hormonal changes and treatment management.

Adolescent Type 1 Diabetes Treatment With SGLT2i for hyperglycEMia & hyPerfilTration Trial

NCT04333823Active, Not RecruitingPhase 3
The Hospital for Sick Children
Posted 12/11/2020

Pattern Computer Advances AI-Discovered Drug for Multiple Hard-to-Treat Cancers to Testing Phase

Precision Medicine
  • Pattern Computer has developed a novel AI-discovered drug using CRISPR technology that targets a protein not expressed in healthy adult tissue but present in aggressive early-onset colorectal cancer patients.
  • The company's Pattern Discovery Engine identified a genetic signature that increases colorectal cancer risk by 200% in individuals under 45 years old, leading to both a new diagnostic tool and therapeutic target.
  • Initial testing will focus on early-onset colorectal cancer, with potential applications across multiple hard-to-treat cancers including ovarian, lung, breast, pancreatic, and brain cancers.
  • The drug is designed with genome-wide specificity for a single target and aims to block metastasis while reducing tumor growth with minimal toxicity.

MD Anderson Study Identifies Three B-Cell Lymphoma Subgroups with Distinct CD19 CAR-T Response Patterns

CAR-TPrecision Medicine
  • Researchers at MD Anderson Cancer Center analyzed 232 patients with large B-cell lymphoma to identify three distinct tumor microenvironment subgroups with different responses to CD19 CAR-T therapy.
  • The study profiled over 1.8 million cells and validated findings using ZUMA-7 Phase III trial data, revealing that the lymph node subgroup benefits most from CAR-T therapy while the T cell exhausted group shows no significant benefit.
  • The findings provide a precision medicine framework to guide treatment selection and identify patients who may need alternative therapeutic approaches beyond CAR-T cell therapy.
  • The research opens opportunities for targeted therapies in development to address the needs of patients in subgroups with poorer CAR-T outcomes.

Actio Biosciences Raises $66M Series B to Advance Rare Disease Programs Targeting Ion Channel Disorders

Rare DiseaseOrphan DrugPrecision Medicine
  • Actio Biosciences secured $66 million in Series B funding co-led by Regeneron Ventures and Deerfield Management to advance two lead programs targeting rare genetic diseases.
  • The company's most advanced program, ABS-0871, is currently in Phase 1 trials for Charcot-Marie-Tooth disease type 2C, with potential expansion to overactive bladder treatment.
  • ABS-1230, targeting KCNT1-related epilepsy affecting approximately 2,500 individuals in the U.S., received FDA rare pediatric and orphan drug designations and is expected to enter Phase 1 trials in late 2025.
  • The company's "inclusive precision medicine" approach aims to develop treatments that address multiple mutations within the same genetic pathway, potentially benefiting broader patient populations.

City of Hope Receives $23.7 Million ARPA-H Grant to Map Immunotherapy Resistance in Lung Cancer

Precision Medicine
  • City of Hope has been awarded up to $23.7 million by ARPA-H to create a biomap of tumor changes causing immunotherapy resistance in advanced non-small cell lung cancer.
  • The six-year clinical trial will enroll 535 patients and use real-time monitoring techniques including liquid biopsies and single-cell sequencing to track tumor evolution.
  • Researchers aim to improve progression-free survival by 50% in at least one patient group by adapting treatments as resistance develops.
  • The project addresses the limited reliability of current biomarkers, with existing immunotherapy response rates below 40% in NSCLC patients.

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