NOVARTIS

Precision medicine's promise remains unfulfilled due to inadequate diagnostics development. The fragmented ecosystem hinders collaboration between academia, pharma, and diagnostics, leading to inefficient assay transfers and limited scaling. A holistic, collaborative approach is needed to integrate biomarker validation, global deployment, and clinical adoption, addressing challenges in intellectual property, data sharing, and risk-gain balance. This unified effort aims to accelerate diagnostics innovation and realize precision medicine's potential.

REGENXBIO reports Q3 2024 financials and operational updates: RGX-202 for Duchenne Muscular Dystrophy advances in Phase I/II AFFINITY DUCHENNE trial; BLA submission for RGX-121 initiated, expected Q1 2025; positive Phase II data supports bilateral administration of subretinal ABBV-RGX-314; End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy accelerated to Q4 2024; conference call at 4:30 p.m. ET.

FDA approves Caris Life Sciences' MI Cancer Seek as companion diagnostic for multiple cancer drugs across six cancer types, including solid tumours, breast cancer, colorectal cancer, melanoma, non-small cell lung cancer, and endometrial carcinoma. The assay uses whole exome sequencing (WES) and whole transcriptome sequencing (WTS) for molecular profiling of solid tumours.

RNA interference (RNAi) research, despite initial challenges, has seen advancements with six approved medicines and many in clinical testing. New startups believe RNAi is ready for broader use beyond liver diseases, with innovations in delivery techniques to other tissues. Key players include City Therapeutics, Judo Bio, Switch Therapeutics, and Aro Biotherapeutics, backed by significant funding. The potential of RNAi, first discovered in the 1990s, is now seen as a new frontier in genetic medicine.

Beam Therapeutics reported a patient death in a Phase I/II trial for BEAM-101, attributing it to the preconditioning regimen, not the treatment. The FDA allowed the trial to continue without changes. Researchers seek less toxic alternatives to busulfan, crucial in cell and gene therapy protocols.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting progress with WAINUA U.S. launch, olezarsen FDA action date in December 2024, and donidalorsen HAE PDUFA date in August 2025. The company is on track to achieve 2024 financial guidance and increased cash guidance to $2.2 billion. Key pipeline milestones include positive CHMP opinion for WAINUA in the UK, QALSODY approval in China, and ongoing pivotal development programs for olezarsen and donidalorsen.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting successful U.S. launch of WAINUA, upcoming FDA action dates for olezarsen and donidalorsen, and reaffirmed 2024 P&L financial guidance with increased cash guidance to $2.2 billion.