Avidity Biosciences

A new blood test developed by Johns Hopkins researchers could revolutionize the diagnosis and treatment of postpartum depression by identifying biological markers in Extracellular Vesicles (EVs) carrying genetic material from the brain. This test aims to predict who will develop depression and when, potentially treating new mothers like Brooke before their babies are born.

FDA accepts new drug application for donidalorsen, a prophylactic RNA-targeting drug to prevent hereditary angioedema attacks, with an action date set for Aug. 21, 2025.

A genetic test, originally developed for spinal fluid, now adapted for respiratory infections, can rapidly detect and identify almost any disease-causing microorganism, including viruses, bacteria, fungi, and parasites, within 12 to 24 hours, potentially revolutionizing infection diagnosis and treatment.

India's cardiac care has advanced significantly, shifting from open-heart surgeries to minimally invasive procedures and digital therapeutics. Innovations like AI-driven diagnostics, robotic-assisted surgeries, and digital therapeutics are revolutionizing the field, improving patient outcomes and reducing recovery times. The future of cardiac care in India is promising, with personalized, genomic-based therapeutics and minimally invasive approaches expected to dominate.

Advances in microscopy and live-cell imaging are crucial for understanding dynamic cellular processes, moving beyond static imaging to observe real-time changes. This shift is essential for developing more accurate models of disease and drug discovery, with technologies like light sheet microscopy and genetically encoded calcium indicators enhancing our ability to study cellular behavior without harming cells. The industry is also transitioning from 2D to 3D cell cultures to better mimic in vivo conditions, and phenotypic screening is regaining importance as a method to identify first-in-class drugs. Label-free imaging and sophisticated software are further advancing live-cell analysis, enabling more detailed and less invasive studies.

New CRISPR-Cas13 targets noncoding RNAs, revealing nearly 800 essential for cell survival/growth, with potential as biomarkers/therapeutic targets in personalized cancer treatments.

Depixus launches MAGNA One for biomolecular interaction analysis, PacBio reduces HiFi human genome sequencing cost to under $500, Jackson Laboratory and Allentown partner for remote mouse monitoring, PumasAI releases AI tools for healthcare data analysis, VALANX Biotech and Fina Biosolutions collaborate on conjugate vaccine development, iotaSciences validates isoPick™ platform for single-cell lipidomics, CalciMedica raises $10.2M in public offering, Biosynth opens new site in Suzhou, Orion Corporation halts ODM-111 development, Innomost appoints Tiina Bensky as CEO, and PLL Therapeutics names Souad Kechairi as Chief Medical Officer.

NVIDIA's BioNeMo AI toolkit, featuring models like DNABERT and scBERT, is revolutionizing drug discovery by enabling analysis of DNA sequences, protein interactions, and cell functions. Over 100 companies, including Cadence and Iambic Therapeutics, are integrating these AI tools to accelerate molecular simulations and drug design, reducing reliance on physical experiments.

Avidity Biosciences initiates biomarker cohort in Phase 1/2 FORTITUDE trial of delpacibart braxlosiran (del-brax) for FSHD, targeting DUX4 gene. Enrollment expected in 1H 2025, with del-brax 2 mg/kg administered every six weeks. Initial data showed significant reductions in DUX4-regulated genes and biomarkers, with trends of functional improvement.

RNAscence Biotechnology's RNA patch technology targets skin's healing processes to address hyperpigmentation, eczema, and wrinkles, offering non-invasive solutions for common skin conditions.